149 results on '"Muppidi S"'
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2. Aerothermal Analysis and Thermal Protection System (TPS) Design of the Mars Sample Retrieval Lander (SRL) Concept
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Muppidi, S, Mcguire, M. K, Kinney, D. J, Saunders, D. A, and Bensassi, K
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Fluid Mechanics And Thermodynamics - Abstract
This poster provides a glimpse of the aerothermal analysis and TPS design work for the Mars Sample Retrieval Lander (SRL), part of the Mars Sample Return (MSR) architecture.
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- 2019
3. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
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Mantegazza, R., Wolfe, G. I., Muppidi, S., Wiendl, H., Fujita, K. P., O'Brien, F. L., Booth, H. D. E., Howard, J. F., Claudio Gabriel Mazia, Miguel, Wilken, Fabio, Barroso, Juliet, Saba, Marcelo, Rugiero, Mariela, Bettini, Marcelo, Chaves, Gonzalo, Vidal, Alejandra Dalila Garcia, Jan De Bleecker, Guy Van den Abeele, Kathy de Koning, Katrien De Mey, Rudy, Mercelis, Délphine, Mahieu, Linda, Wagemaekers, Philip Van Damme, Annelies, Depreitere, Caroline, Schotte, Charlotte, Smetcoren, Olivier, Stevens, Sien Van Daele, Nicolas, Vandenbussche, Annelies, Vanhee, Sarah, Verjans, Jan, Vynckier, Ann, D'Hont, Petra, Tilkin, Alzira Alves de Siqueira Carvalho, Igor Dias Brockhausen, David, Feder, Daniel, Ambrosio, Gabor Lovasamela César, Ana Paula Melo, Renata Martins Ribeiro, Rosana, Rocha, Bruno Bezerra Rosa, Thabata, Veiga, Luiz Augusto da Silva, Murilo Santos Engel, Jordana Gonçalves Geraldo, Maria da Penha Ananias Morita, Erica Nogueira Coelho, Gabriel, Paiva, Marina, Pozo, Natalia, Prando, Debora Dada Martineli Torres, Cristiani Fernanda Butinhao, Gustavo, Duran, Tomás Augusto Suriane Fialho, Tamires Cristina Gomes da Silva, Luiz Otavio Maia Gonçalves, Lucas Eduardo Pazetto, Luciana Renata Cubas Volpe, Luciana Souza Duca, Maurício AndréGheller Friedrich, Alexandre, Guerreiro, Henrique, Mohr, Maurer Pereira Martins, Daiane da Cruz Pacheco, Luciana, Ferreira, Ana Paula Macagnan, Graziela, Pinto, Aline de Cassia Santos, Acary Souza Bulle Oliveira, Ana Carolina Amaral de Andrade, Marcelo, Annes, Liene Duarte Silva, Valeria Cavalcante Lino, Wladimir, Pinto, Natália, Assis, Fernanda, Carrara, Carolina, Miranda, Iandra, Souza, Patrícia, Fernandes, Zaeem, Siddiqi, Cecile, Phan, Jeffrey, Narayan, Derrick, Blackmore, Ashley, Mallon, Rikki, Roderus, Elizabeth, Watt, Stanislav, Vohanka, Josef, Bednarik, Magda, Chmelikova, Marek, Cierny, Stanislava, Toncrova, Jana, Junkerova, Barbora, Kurkova, Katarina, Reguliova, Olga, Zapletalova, Jiri, Pitha, Iveta, Novakova, Michaela, Tyblova, Ivana, Jurajdova, Marcela, Wolfova, Henning, Andersen, Thomas, Harbo, Lotte, Vinge, Susanne, Krogh, Anita, Mogensen, John, Vissing, Joan, Højgaard, Nanna, Witting, Anne Mette Ostergaard Autzen, Jane, Pedersen, Juha-Pekka, Erälinna, Mikko, Laaksonen, Olli, Oksaranta, Tuula, Harrison, Jaana, Eriksson, Csilla, Rozsa, Melinda, Horvath, Gabor, Lovas, Judit, Matolcsi, Gedeonne, Jakab, Gyorgyi, Szabo, Brigitta, Szabadosne, Laszlo, Vecsei, Livia, Dezsi, Edina, Varga, Monika, Konyane, Antonini, Giovanni, Antonella Di Pasquale, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Fionda, Laura, Amelia, Evoli, Paolo Emilio Alboini, Valentina, D'Amato, Raffaele, Iorio, Inghilleri, Maurizio, Frasca, Vittorio, Elena, Giacomelli, Gori, MARIA CRISTINA, Diego, Lopergolo, Onesti, Emanuela, Maria, Gabriele, Francesco, Saccà, Alessandro, Filla, Teresa, Costabile, Enrico, Marano, Angiola, Fasanaro, Angela, Marsili, Giorgia, Puorro, Carlo, Antozzi, Silvia, Bonanno, Giorgia, Camera, Alberta, Locatelli, Lorenzo, Maggi, Maria, Pasanisi, Angela, Campanella, Akiyuki, Uzawa, Tetsuya, Kanai, Naoki, Kawaguchi, Masahiro, Mori, Yoko, Kaneko, Akiko, Kanzaki, Eri, Kobayashi, Hiroyuki, Murai, Katsuhisa, Masaki, Dai, Matsuse, Takuya, Matsushita, Taira, Uehara, Misa, Shimpo, Maki, Jingu, Keiko, Kikutake, Yumiko, Nakamura, Yoshiko, Sano, Kimiaki, Utsugisawa, Yuriko, Nagane, Ikuko, Kamegamori, Tomoko, Tsuda, Yuko, Fujii, Kazumi, Futono, Yukiko, Ozawa, Aya, Mizugami, Yuka, Saito, Makoto, Samukawa, Hidekazu, Suzuki, Miyuki, Morikawa, Sachiko, Kamakura, Eriko, Miyawaki, Meinoshin, Okumura, Soichiro, Funaka, Tomohiro, Kawamura, Masayuki, Nakamori, Masanori, Takahashi, Namie, Taichi, Tomoya, Hasuike, Eriko, Higuchi, Hisako, Kobayashi, Kaori, Osakada, Hirokazu, Shiraishi, Teiichiro, Miyazaki, Masakatsu, Motomura, Akihiro, Mukaino, Shunsuke, Yoshimura, Shizuka, Asada, Seiko, Yoshida, Shoko, Amamoto, Tomomi, Kobashikawa, Megumi, Koga, Maeda, Yasuko, Kazumi, Takada, Mihoko, Takada, Masako, Tsurumaru, Yumi, Yamashita, Yasushi, Suzuki, Tetsuya, Akiyama, Koichi, Narikawa, Ohito, Tano, Kenichi, Tsukita, Rikako, Kurihara, Fumie, Meguro, Yusuke, Fukuda, Miwako, Sato, Tomihiro, Imai, Emiko, Tsuda, Shun, Shimohama, Takashi, Hayashi, Shin, Hisahara, Jun, Kawamata, Takashi, Murahara, Masaki, Saitoh, Shuichiro, Suzuki, Daisuke, Yamamoto, Yoko, Ishiyama, Naoko, Ishiyama, Mayuko, Noshiro, Rumi, Takeyama, Kaori, Uwasa, Ikuko, Yasuda, Anneke van der Kooi, Marianne de Visser, Tamar, Gibson, Byung-Jo, Kim, Chang Nyoung Lee, Yong Seo Koo, Hung Youl Seok, Hoo Nam Kang, Hyejin, Ra, Byoung Joon Kim, Eun Bin Cho, Misong, Choi, Hyelim, Lee, Ju-Hong, Min, Jinmyoung, Seok, Jieun, Lee, Da Yoon Koh, Juyoung, Kwon, Sangae, Park, Eun Haw Choi, Yoon-Ho, Hong, So-Hyun, Ahn, Dae Lim Koo, Jae-Sung, Lim, Chae Won Shin, Ji Ye Hwang, Miri, Kim, Seung Min Kim, Ha-Neul, Jeong, Jinwoo, Jung, Yool-Hee, Kim, Hyung Seok Lee, Ha Young Shin, Eun Bi Hwang, Miju, Shin, Carlos, Casasnovas, Maria Antonia Alberti Aguilo, Christian, Homedes-Pedret, Natalia Julia Palacios, Laura Diez Porras, Valentina Velez Santamaria, Ana, Lazaro, Josep Gamez Carbonell, Pilar, Sune, Maria Salvado Figueras, Gisela, Gili, Gonzalo, Mazuela, Isabel, Illa, Elena Cortes Vicente, Jordi, Diaz-Manera, Luis Antonio Querol Gutiérrez, Ricardo Rojas Garcia, Nuria, Vidal, Elisabet, Arribas-Ibar, Exuperio Diez Tejedor, Pilar Gomez Salcedo, Mireya, Fernandez-Fournier, Pedro Lopez Ruiz, Francisco Javier Rodriguez de Rivera, Maria, Sastre, Fredrik, Piehl, Albert, Hietala, Lena, Bjarbo, Ihsan, Sengun, Arzu, Meherremova, Pinar, Ozcelik, Bengu, Balkan, Celal, Tuga, Muzeyyen, Ugur, Sevim, Erdem-Ozdamar, Can Ebru Bekircan-Kurt, Nazire Pinar Acar, Ezgi, Yilmaz, Yagmur, Caliskan, Gulsah, Orsel, Husnu, Efendi, Seda, Aydinlik, Hakan, Cavus, Ayse, Kutlu, Gulsah, Becerikli, Cansu, Semiz, Ozlem, Tun, Murat, Terzi, Baki, Dogan, Musa Kazim Onar, Sedat, Sen, Tugce Kirbas Cavdar, Adife, Veske, Fiona, Norwood, Aikaterini, Dimitriou, Jakit, Gollogly, Mohamed, Mahdi-Rogers, Arshira, Seddigh, Giannis, Sokratous, Gal, Maier, Faisal, Sohail, Saiju, Jacob, Girija, Sadalage, Pravin, Torane, Claire, Brown, Amna, Shah, Sivakumar, Sathasivam, Heike, Arndt, Debbie, Davies, Dave, Watling, Anthony, Amato, Thomas, Cochrane, Mohammed, Salajegheh, Kristen, Roe, Katherine, Amato, Shirli, Toska, Nicholas, Silvestri, Kara, Patrick, Karen, Zakalik, Jonathan, Katz, Robert, Miller, Marguerite, Engel, Dallas, Forshew, Elena, Bravver, Benjamin, Brooks, Mohammed, Sanjak, Sarah, Plevka, Maryanne, Burdette, Scott, Cunningham, Megan, Kramer, Joanne, Nemeth, Clara, Schommer, Tierney, Scott, Vern, Juel, Jeffrey, Guptill, Lisa, Hobson-Webb, Janice, Massey, Kate, Beck, Donna, Carnes, John, Loor, Amanda, Anderson, Robert, Pascuzzi, Cynthia, Bodkin, John, Kincaid, Riley, Snook, Sandra, Guingrich, Angela, Micheels, Vinay, Chaudhry, Andrea, Corse, Betsy, Mosmiller, Andrea, Kelley, Doreen, Ho, Jayashri, Srinivasan, Michal, Vytopil, Jordan, Jara, Nicholas, Ventura, Cynthia, Carter, Craig, Donahue, Carol, Herbert, Stephanie, Scala, Elaine, Weiner, Sharmeen, Alam, Jonathan, Mckinnon, Laura, Haar, Naya, Mckinnon, Karan, Alcon, Kaitlyn, Mckenna, Nadia, Sattar, Kevin, Daniels, Dennis, Jeffery, Miriam, Freimer, Joseph Chad Hoyle, John, Kissel, Julie, Agriesti, Sharon, Chelnick, Louisa, Mezache, Colleen, Pineda, Filiz, Muharrem, Chafic, Karam, Julie, Khoury, Tessa, Marburger, Harpreet, Kaur, Diana, Dimitrova, James, Gilchrist, Brajesh, Agrawal, Mona, Elsayed, Stephanie, Kohlrus, Angela, Ardoin, Taylor, Darnell, Laura, Golden, Barbara, Lokaitis, Jenna, Seelbach, Neelam, Goyal, Sarada, Sakamuri, Yuen, T So, Shirley, Paulose, Sabrina, Pol, Lesly, Welsh, Ratna, Bhavaraju-Sanka, Alejandro Tobon Gonzalez, Lorraine, Dishman, Floyd, Jones, Anna, Gonzalez, Patricia, Padilla, Amy, Saklad, Marcela, Silva, Sharon, Nations, Jaya, Trivedi, Steve, Hopkins, Mohamed, Kazamel, Mohammad, Alsharabati, Liang, Lu, Kenkichi, Nozaki, Sandi, Mumfrey-Thomas, Amy, Woodall, Tahseen, Mozaffar, Tiyonnoh, Cash, Namita, Goyal, Gulmohor, Roy, Veena, Mathew, Fatima, Maqsood, Brian, Minton, H James Jones, Jeffrey, Rosenfeld, Rebekah, Garcia, Laura, Echevarria, Sonia, Garcia, Michael, Pulley, Shachie, Aranke, Alan Ross Berger, Jaimin, Shah, Yasmeen, Shabbir, Lisa, Smith, Mary, Varghese, Laurie, Gutmann, Ludwig, Gutmann, Nivedita, Jerath, Christopher, Nance, Andrea, Swenson, Heena, Olalde, Nicole, Kressin, Jeri, Sieren, Richard, Barohn, Mazen, Dimachkie, Melanie, Glenn, April, Mcvey, Mamatha, Pasnoor, Jeffery, Statland, Yunxia, Wang, Tina, Liu, Kelley, Emmons, Nicole, Jenci, Jerry, Locheke, Alex, Fondaw, Kathryn, Johns, Gabrielle, Rico, Maureen, Walsh, Laura, Herbelin, Charlene, Hafer-Macko, Justin, Kwan, Lindsay, Zilliox, Karen, Callison, Valerie, Young, Beth, Disanzo, Kerry, Naunton, Michael, Benatar, Martin, Bilsker, Khema, Sharma, Anne, Cooley, Eliana, Reyes, Sara-Claude, Michon, Danielle, Sheldon, Julie, Steele, Rebecca, Traub, Manisha, Chopra, Tuan, Vu, Lara, Katzin, Terry, Mcclain, Brittany, Harvey, Adam, Hart, Kristin, Huynh, Said, Beydoun, Amaiak, Chilingaryan, Victor, Doan, Brian, Droker, Hui, Gong, Sanaz, Karimi, Frank, Lin, Krishna, Polaka, Akshay, Shah, Anh, Tran, Salma, Akhter, Ali, Malekniazi, Rup, Tandan, Michael, Hehir, Waqar, Waheed, Shannon, Lucy, Michael, Weiss, Jane, Distad, Susan, Strom, Sharon, Downing, Bryan, Kim, Tulio, Bertorini, Thomas, Arnold, Kendrick, Henderson, Rekha, Pillai, Liu, Ye, Lauren, Wheeler, Jasmine, Hewlett, Mollie, Vanderhook, Richard, Nowak, Daniel, Dicapua, Benison, Keung, Aditya, Kumar, Huned, Patwa, Kimberly, Robeson, Irene, Yang, Joan, Nye, and Hong, Vu
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Adult ,Male ,0301 basic medicine ,medicine.medical_specialty ,Clinical Neurology ,Antibodies, Monoclonal, Humanized ,Placebo ,Article ,Antibodies ,Post-intervention ,law.invention ,03 medical and health sciences ,0302 clinical medicine ,Randomized controlled trial ,Refractory ,law ,Internal medicine ,Monoclonal ,Myasthenia Gravis ,Medicine and Health Sciences ,Humans ,Medicine ,Humanized ,Science & Technology ,business.industry ,Middle Aged ,Eculizumab ,Complement Inactivating Agents ,Female ,Treatment Outcome ,EFFICACY ,medicine.disease ,Myasthenia gravis ,Clinical trial ,030104 developmental biology ,SAFETY ,Neurosciences & Neurology ,Neurology (clinical) ,business ,Life Sciences & Biomedicine ,COMPLEMENT INHIBITOR ECULIZUMAB ,030217 neurology & neurosurgery ,medicine.drug - Abstract
ObjectiveTo evaluate whether eculizumab helps patients with anti–acetylcholine receptor–positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension.MethodsPatients who completed the REGAIN randomized controlled trial and continued into the open-label extension were included in this tertiary endpoint analysis. Patients were assessed for the MGFA post-intervention status of improved, unchanged, worse, MM, and pharmacologic remission at defined time points during REGAIN and through week 130 of the open-label study.ResultsA total of 117 patients completed REGAIN and continued into the open-label study (eculizumab/eculizumab: 56; placebo/eculizumab: 61). At week 26 of REGAIN, more eculizumab-treated patients than placebo-treated patients achieved a status of improved (60.7% vs 41.7%) or MM (25.0% vs 13.3%; common OR: 2.3; 95% CI: 1.1–4.5). After 130 weeks of eculizumab treatment, 88.0% of patients achieved improved status and 57.3% of patients achieved MM status. The safety profile of eculizumab was consistent with its known profile and no new safety signals were detected.ConclusionEculizumab led to rapid and sustained achievement of MM in patients with AChR+ refractory gMG. These findings support the use of eculizumab in this previously difficult-to-treat patient population.ClinicalTrials.gov IdentifierREGAIN, NCT01997229; REGAIN open-label extension, NCT02301624.Classification of EvidenceThis study provides Class II evidence that, after 26 weeks of eculizumab treatment, 25.0% of adults with AChR+ refractory gMG achieved MM, compared with 13.3% who received placebo.
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- 2020
4. Distributed Computing Environment (DCE) Porting Tool
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Muppidi, S., Krawetz, N., Beedubail, G., Marti, W., Pooch, U., Schill, Alexander, editor, Mittasch, Christian, editor, Spaniol, Otto, editor, and Popien, Claudia, editor
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- 1996
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5. Consolidation and maintenance therapy with lenalidomide, bortezomib and dexamethasone (RVD) in high-risk myeloma patients
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Nooka, A K, Kaufman, J L, Muppidi, S, Langston, A, Heffner, L T, Gleason, C, Casbourne, D, Saxe, D, Boise, L H, and Lonial, S
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- 2014
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6. Favorable impact of pre-transplant ATG on outcomes of reduced-intensity hematopoietic cell transplants from partially mismatched unrelated donors
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Langston, A A, Prichard, J M, Muppidi, S, Nooka, A, Lechowicz, M J, Lonial, S, Sinha, R, Graiser, M, Kaufman, J L, Khoury, H J, Flowers, C R, and Waller, E K
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- 2014
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7. ‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab
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Vissing J., Jacob S., Fujita K. P., O'Brien F., Howard J. F., Mazia C. G., Wilken M., Barroso F., Saba J., Rugiero M., Bettini M., Chaves M., Vidal G., Garcia A. D., DeBleecker J., Vanden Abeele G., deKoning K., DeMey K., Mercelis R., Mahieu D., Wagemaekers L., VanDamme P., Depreitere A., Schotte C., Smetcoren C., Stevens O., VanDaele S., Vandenbussche N., Vanhee A., Verjans S., Vynckier J., D'Hont A., Tilkin P., Alves deSiqueira Carvalho A., DiasBrockhausen I., Feder D., Ambrosio D., Cesar P., Melo A. P., MartinsRibeiro R., Rocha R., Rosa B. B., Veiga T., daSilva L. A., SantosEngel M., GoncalvesGeraldo J., daPenha Ananias Morita M., NogueiraCoelho E., Paiva G., Pozo M., Prando N., MartineliTorres D. D., Butinhao C. F., Duran G., SurianeFialho T. A., Gomes daSilva T. C., MaiaGoncalves L. O., Pazetto L. E., CubasVolpe L. R., SouzaDuca L., GhellerFriedrich M. A., Guerreiro A., Mohr H., PereiraMartins M., daCruz Pacheco D., Ferreira L., Macagnan A. P., Pinto G., deCassia Santos A., Souza BulleOliveira A., Amaral deAndrade A. C., Annes M., DuarteSilva L., CavalcanteLino V., Pinto W., Assis N., Carrara F., Miranda C., Souza I., Fernandes P., Siddiqi Z., Phan C., Narayan J., Blackmore D., Mallon A., Roderus R., Watt E., Vohanka S., Bednarik J., Chmelikova M., Cierny M., Toncrova S., Junkerova BarboraKurkova J., Reguliova K., Zapletalova O., Pitha J., Novakova I., Tyblova M., Jurajdova I., Wolfova M., Andersen H., Harbo T., Vinge L., Krogh S., Mogensen A., Hojgaard J., Witting N., Mette OstergaardAutzen A., Pedersen J., Eralinna J. -P., Laaksonen M., Oksaranta O., Harrison T., Eriksson J., Rozsa C., Horvath M., Lovas G., Matolcsi J., Szabo G., Jakab G., Szabadosne B., Vecsei L., Dezsi L., Varga E., Konyane M., Antonini G., DiPasquale A., Garibaldi M., Morino S., Troili F., Fionda L., Sacca F., previous, Filla A., sub-investigators, Costabile T., Marano E., Fasanaro A., Marsili A., Puorro G., Mantegazza R., Antozzi C., Bonanno S., Camera G., Locatelli A., Maggi L., Pasanisi M., Campanella A., Evoli A., Alboini P. E., D'Amato V., Iorio R., Inghilleri M., Frasca V., Giacomelli E., Gori M., Lopergolo D., Onesti E., Gabriele M., Uzawa A., Kanai T., Kawaguchi N., Mori M., Kaneko Y., Kanzaki A., Kobayashi E., Murai H., Masaki K., Matsuse D., Matsushita T., Uehara T., Shimpo M., Jingu M., Kikutake K., Nakamura Y., Sano Y., Utsugisawa K., Nagane Y., Kamegamori I., Tsuda T., Fujii Y., Futono K., Ozawa Y., Mizugami A., Saito Y., Samukawa M., Suzuki H., Morikawa M., Kamakura S., Miyawaki E., Shiraishi H., Mitazaki T., Motomura M., Mukaino A., Yoshimura S., Asada S., Yoshida S., Amamoto S., Kobashikawa T., Koga M., Maeda Y., Takada K., Takada M., Tsurumaru M., Yamashita Y., Suzuki Y., Akiyama T., Narikawa K., Tano O., Tsukita K., Kurihara R., Meguro F., Fukuda Y., Sato M., Okumura M., Funaka S., Kawamura T., Makamori M., Takahashi M., Taichi N., Hasuike T., Higuchi E., Kobayashi H., Osakada K., Imai T., Tsuda E., Shimohama S., Hayashi T., Hisahara S., Kawamata J., Murahara T., Saitoh M., Suzuki S., Yamamoto D., Ishiyama Y., Ishiyama N., Noshiro M., Takeyama R., Uwasa K., Yasuda I., Kim B. -J., Lee C. N., Koo Y. S., Seok H. Y., Kang H. N., Ra H. J., Kim B. J., Cho E. B., Choi M. S., Lee H. L., Min J. -H., Seok J., Lee J. E., Koh D. Y., Kwon J. Y., Park S. A., Choi E. H., Hong Y. -H., Ahn S. -H., Koo D. L., Lim J. -S., Shin C. W., Hwang J. Y., Kim M., Kim S. M., Jeong H. -N., Jung J. W., Kim Y. -H., Lee H. S., Shin H. Y., Hwang E. B., Shin M., van derKooi A., deVisser M., Gibson T., Casasnovas C., AlbertiAguilo M. A., Homedes-Pedret C., Palacios N. J., DiezPorras L., VelezSantamaria V., Lazaro A., DiezTejedor E., GomezSalcedo P., Fernandez-Fournier M., LopezRuiz P., Rodriguez deRivera F. J., Sastre M., GamezCarbonell J., Sune P., SalvadoFigueras M., Gili G., Mazuela G., Illa I., CortesVicente E., Diaz-Manera J., QuerolGutierrez L. A., RojasGarcia R., Vidal N., Arribas-Ibar E., Piehl F., Hietala A., Bjarbo L., Sengun I., Meherremova A., Ozcelik P., Balkan B., Tuga C., Ugur M., Erdem-Ozdamar S., Bekircan-Kurt C. E., Acar N. P., Yilmaz E., Caliskan Y., Orsel G., Efendi H., Aydinlik S., Cavus H., Kutlu A., Becerikli G., Semiz C., Tun O., Terzi M., Dogan B., Onar M. K., Sen S., KirbasCavdar T., Veske A., Norwood F., Dimitriou A., Gollogly J., Mahdi-Rogers M., Seddigh A., Sokratous G., Maier G., Sohail F., Sadalage G., Torane P., Brown C., Shah A., Sathasivam S., Arndt H., Davies D., Watling D., Amato A., Cochrane T., Salajegheh M., Roe K., Amato K., Toska S., Wolfe G., Silvestri N., Patrick K., Zakalik K., Katz J., Miller R., Engel M., Forshew D., Bravver E., Brooks B., Sanjak M., Plevka S., Burdette M., Cunningham S., Kramer M., Nemeth J., Schommer C., Tinerney S., Juel V., Guptill J., Hobson-Webb L., Massey J., Beck K., Carnes D., Loor J., Anderson A., Pascuzzi R., Bodkin C., Kincaid J., Snook R., Guinrich S., Micheels A., Chaudhry V., Corse A., Mosmiller B., Kelley A., Ho D., Srinivasan J., Vytopil M., Jara J., Ventura N., Carter C., Donahue C., Herbert C., Scala S., Weiner E., Alam S., McKinnon J., Haar L., McKinnon N., Alcon K., McKenna K., Sattar N., Daniels K., Jeffery D., Freimer M., Hoyle J. C., Kissel J., Agriesti J., Chelnick S., Mezache L., Pineda C., Muharrem F., Karam C., Khoury J., Marburger T., Kaur H., Dimitrova D., Gilchrist J., Agrawal B., Elsayed M., Kohlrus S., Andoin A., Darnell T., Golden L., Lokaitis B., Seelbach J., Muppidi S., Goyal N., Sakamuri S., So Y. T., Paulose S., Pol S., Welsh L., Bhavaraju-Sanka R., TobonGonzalez A., Dishman L., Jones F., Gonzalez A., Padilla P., Saklad A., Silva M., Nations S., Trivedi J., Hopkins S., Kazamel M., Alsharabati M., Lu L., Nozaki K., Mumfrey-Thomas S., Woodall A., Mozaffar T., Cash T., Roy G., Mathew V., Maqsood F., Minton B., Jones H. J., Rosenfeld J., Garcia R., Echevarria L., Garcia S., Pulley M., Aranke S., Berger A. R., Shah J., Shabbir Y., Smith L., Varghese M., Gutmann L., Jerath N., Nance C., Swenson A., Olalde H., Kressin N., Sieren J., Barohn R., Dimachkie M., Glenn M., McVey A., Pasnoor M., Statland J., Wang Y., Liu T., Emmons K., Jenci N., Locheke J., Fondaw A., Johns K., Rico G., Walsh M., Herbelin L., Hafer-Macko C., Kwan J., Zilliox L., Callison K., Young V., DiSanzo B., Naunton K., Benatar M., Bilsker M., Sharma K., Cooley A., Reyes E., Michon S. -C., Sheldon D., Steele J., Howard J., Traub R., Chopra M., Vu T., Katzin L., McClain T., Harvey B., Hart A., Huynh K., Beydoun S., Chilingaryan A., Doan V., Droker B., Gong H., Karimi S., Lin F., Polaka K., Tran A., Akhter S., Malekniazi A., Tandan R., Hehir M., Waheed W., Lucy S., Weiss M., Distad J., Strom S., Downing S., Kim B., Bertorini T., Arnold T., Henderson K., Pillai R., Liu Y., Wheeler L., Hewlett J., Vanderhook M., Nowak R., Dicapua D., Keung B., Kumar A., Patwa H., Robeson K., Yang I., Nye J., Vu H., Vissing, J., Jacob, S., Fujita, K. P., O'Brien, F., Howard, J. F., Mazia, C. G., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Garcia, A. D., Debleecker, J., Vanden Abeele, G., Dekoning, K., Demey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., Vandamme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., Vandaele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hont, A., Tilkin, P., Alves deSiqueira Carvalho, A., Diasbrockhausen, I., Feder, D., Ambrosio, D., Cesar, P., Melo, A. P., Martinsribeiro, R., Rocha, R., Rosa, B. B., Veiga, T., Dasilva, L. A., Santosengel, M., Goncalvesgeraldo, J., daPenha Ananias Morita, M., Nogueiracoelho, E., Paiva, G., Pozo, M., Prando, N., Martinelitorres, D. D., Butinhao, C. F., Duran, G., Surianefialho, T. A., Gomes daSilva, T. C., Maiagoncalves, L. O., Pazetto, L. E., Cubasvolpe, L. R., Souzaduca, L., Ghellerfriedrich, M. A., Guerreiro, A., Mohr, H., Pereiramartins, M., daCruz Pacheco, D., Ferreira, L., Macagnan, A. P., Pinto, G., deCassia Santos, A., Souza BulleOliveira, A., Amaral deAndrade, A. C., Annes, M., Duartesilva, L., Cavalcantelino, V., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Siddiqi, Z., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova BarboraKurkova, J., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Andersen, H., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Hojgaard, J., Witting, N., Mette OstergaardAutzen, A., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., Dipasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Sacca, F., Previous, Filla, A., sub-investigators, Costabile, T., Marano, E., Fasanaro, A., Marsili, A., Puorro, G., Mantegazza, R., Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Evoli, A., Alboini, P. E., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Murai, H., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Utsugisawa, K., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Samukawa, M., Suzuki, H., Morikawa, M., Kamakura, S., Miyawaki, E., Shiraishi, H., Mitazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Okumura, M., Funaka, S., Kawamura, T., Makamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., Kim, B. -J., Lee, C. N., Koo, Y. S., Seok, H. Y., Kang, H. N., Ra, H. J., Kim, B. J., Cho, E. B., Choi, M. S., Lee, H. L., Min, J. -H., Seok, J., Lee, J. E., Koh, D. Y., Kwon, J. Y., Park, S. A., Choi, E. H., Hong, Y. -H., Ahn, S. -H., Koo, D. L., Lim, J. -S., Shin, C. W., Hwang, J. Y., Kim, M., Kim, S. M., Jeong, H. -N., Jung, J. W., Kim, Y. -H., Lee, H. S., Shin, H. Y., Hwang, E. B., Shin, M., van derKooi, A., Devisser, M., Gibson, T., Casasnovas, C., Albertiaguilo, M. A., Homedes-Pedret, C., Palacios, N. J., Diezporras, L., Velezsantamaria, V., Lazaro, A., Dieztejedor, E., Gomezsalcedo, P., Fernandez-Fournier, M., Lopezruiz, P., Rodriguez deRivera, F. J., Sastre, M., Gamezcarbonell, J., Sune, P., Salvadofigueras, M., Gili, G., Mazuela, G., Illa, I., Cortesvicente, E., Diaz-Manera, J., Querolgutierrez, L. A., Rojasgarcia, R., Vidal, N., Arribas-Ibar, E., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C. E., Acar, N. P., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Onar, M. K., Sen, S., Kirbascavdar, T., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Sanjak, M., Plevka, S., Burdette, M., Cunningham, S., Kramer, M., Nemeth, J., Schommer, C., Tinerney, S., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guinrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Carter, C., Donahue, C., Herbert, C., Scala, S., Weiner, E., Alam, S., Mckinnon, J., Haar, L., Mckinnon, N., Alcon, K., Mckenna, K., Sattar, N., Daniels, K., Jeffery, D., Freimer, M., Hoyle, J. C., Kissel, J., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Andoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelbach, J., Muppidi, S., Goyal, N., Sakamuri, S., So, Y. T., Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., Tobongonzalez, A., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Mozaffar, T., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., Jones, H. J., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Berger, A. R., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Barohn, R., Dimachkie, M., Glenn, M., Mcvey, A., Pasnoor, M., Statland, J., Wang, Y., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., Disanzo, B., Naunton, K., Benatar, M., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Howard, J., Traub, R., Chopra, M., Vu, T., Katzin, L., Mcclain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Polaka, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Henderson, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Nowak, R., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., Vu, H., ANS - Neuroinfection & -inflammation, and Neurology
- Subjects
0301 basic medicine ,Male ,Myasthenia gravi ,Gastroenterology ,0302 clinical medicine ,Quality of life ,Activities of Daily Living ,CYCLOPHOSPHAMIDE ,Medicine and Health Sciences ,Medicine ,Receptors, Cholinergic ,Acetylcholine receptor ,Myasthenia gravis ,Original Communication ,Eculizumab ,Minimal symptom expression ,Refractory ,Middle Aged ,Acetylcholine receptor antibody ,Tolerability ,Neurology ,Female ,Life Sciences & Biomedicine ,COMPLEMENT INHIBITOR ECULIZUMAB ,medicine.drug ,Adult ,medicine.medical_specialty ,Clinical Neurology ,Placebo ,Antibodies, Monoclonal, Humanized ,03 medical and health sciences ,Double-Blind Method ,Internal medicine ,Humans ,Immunologic Factors ,Patient Reported Outcome Measures ,Generalized myasthenia ,Aged ,Autoantibodies ,Science & Technology ,business.industry ,Confidence interval ,030104 developmental biology ,Quality of Life ,Neurology (clinical) ,Neurosciences & Neurology ,business ,030217 neurology & neurosurgery - Abstract
Background The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension. Methods Attainment of ‘minimal symptom expression’ was evaluated using patient-reported outcome measures of gMG symptoms [MG activities of daily living scale (MG-ADL), 15-item MG quality of life questionnaire (MG-QOL15)] at the completion of REGAIN and during the open-label extension. ‘Minimal symptom expression’ was defined as MG-ADL total score of 0–1 or MG-QOL15 total score of 0–3. Results At REGAIN week 26, more eculizumab-treated patients achieved ‘minimal symptom expression’ versus placebo [MG-ADL: 21.4% vs 1.7%; difference 19.8%; 95% confidence interval (CI) 8.5, 31.0; p = 0.0007; MG-QOL15: 16.1% vs 1.7%; difference 14.4%; 95% CI 4.3, 24.6; p = 0.0069]. During the open-label extension, the proportion of patients in the placebo/eculizumab group who achieved ‘minimal symptom expression’ increased after initiating eculizumab treatment and was sustained through 130 weeks of open-label eculizumab (MG-ADL: 1.7 to 27.8%; MG-QOL15: 1.7 to 19.4%). At extension study week 130, similar proportions of patients in the eculizumab/eculizumab and placebo/eculizumab groups achieved ‘minimal symptom expression’ (MG-ADL: 22.9% and 27.8%, respectively, p = 0.7861; MG-QOL15: 14.3% and 19.4%, respectively, p = 0.7531). The long-term tolerability of eculizumab was consistent with previous reports. Conclusions Patients with AChR+ refractory gMG who receive eculizumab can achieve sustained ‘minimal symptom expression’ based on patient-reported outcomes. ‘Minimal symptom expression’ may be a useful tool in measuring therapy effectiveness in gMG. Trial registration ClinicalTrials.gov NCT01997229, NCT02301624.
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- 2020
8. Consistent improvement with eculizumab across muscle groups in myasthenia gravis
- Author
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Mantegazza, R., O'Brien, F. L., Yountz, M., Howard, J. F., Gabriel Mazia, C., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Dalila Garcia, A., De Bleecker, J., Van den Abeele, G., de Koning, K., De Mey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., Van Damme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., Van Daele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hont, A., Tilkin, P., Alves de Siqueira Carvalho, A., Dias Brockhausen, I., Feder, D., Ambrosio, D., Cesar, P., Paula Melo, A., Martins Ribeiro, R., Rocha, R., Bezerra Rosa, B., Veiga, T., Augusto da Silva, L., Santos Engel, M., Goncalves Geraldo, J., da Penha Ananias Morita, M., Nogueira Coelho, E., Paiva, G., Pozo, M., Prando, N., Torres, D. D. M., Fernanda Butinhao, C., Duran, G., Augusto Suriane Fialho, T., Gomes da Silva, T. C., Goncalves, L. O. M., Eduardo Pazetto, L., Renata Cubas Volpe, L., Souza Duca, L., Friedrich, M. A. G., Guerreiro, A., Mohr, H., Pereira Martins, M., da Cruz Pacheco, D., Ferreira, L., Paula Macagnan, A., Pinto, G., de Cassia Santos, A., Souza Bulle Oliveira, A., Amaral de Andrade, A. C., Annes, M., Duarte Silva, L., Cavalcante Lino, V., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Siddiqi, Z., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova, J., Kurkova, B., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Andersen, H., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Vissing, J., Hojgaard, J., Witting, N., Mette Ostergaard Autzen, A., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., Di Pasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Pasquale, A., Evoli, A., Emilio Alboini, P., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Sacca, F., Filla, A., Costabile, T., Marano, E., Fasanaro, A., Marsili, A., Puorro, G., Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Murai, H., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Utsugisawa, K., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Samukawa, M., Suzuki, H., Morikawa, M., Kamakura, S., Miyawaki, E., Okumura, M., Funaka, S., Kawamura, T., Nakamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Shiraishi, H., Miyazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., van der Kooi, A., de Visser, M., Gibson, T., Kim, B. -J., Nyoung Lee, C., Seo Koo, Y., Youl Seok, H., Nam Kang, H., Ra, H., Joon Kim, B., Bin Cho, E., Choi, M., Lee, H., Min, J. -H., Seok, J., Lee, J., Koh, D. Y., Kwon, J., Park, S., Haw Choi, E., Hong, Y. -H., Ahn, S. -H., Lim Koo, D., Lim, J. -S., Won Shin, C., Ye Hwang, J., Kim, M., Min Kim, S., Jeong, H. -N., Jung, J., Kim, Y. -H., Seok Lee, H., Young Shin, H., Bi Hwang, E., Shin, M., Casasnovas, C., Antonia Alberti Aguilo, M., Homedes-Pedret, C., Julia Palacios, N., Diez Porras, L., Velez Santamaria, V., Lazaro, A., Gamez Carbonell, J., Sune, P., Salvado Figueras, M., Gili, G., Mazuela, G., Illa, I., Cortes Vicente, E., Diaz-Manera, J., Antonio Querol Gutierrez, L., Rojas Garcia, R., Vidal, N., Arribas-Ibar, E., Diez Tejedor, E., Gomez Salcedo, P., Fernandez-Fournier, M., Lopez Ruiz, P., Rodriguez de Rivera, F. J., Sastre, M., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C., Pinar Acar, N., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Kazim Onar, M., Sen, S., Kirbas Cavdar, T., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Jacob, S., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Sanjak, M., Plevka, S., Burdette, M., Cunningham, S., Kramer, M., Nemeth, J., Schommer, C., Scott, T., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guingrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Carter, C., Donahue, C., Herbert, C., Scala, S., Weiner, E., Alam, S., Mckinnon, J., Haar, L., Mckinnon, N., Alcon, K., Mckenna, K., Sattar, N., Daniels, K., Jeffery, D., Freimer, M., Chad Hoyle, J., Kissel, J., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Ardoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelbach, J., Muppidi, S., Goyal, N., Sakamuri, S., Y. T., So, Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., Tobon Gonzalez, A., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Mozaffar, T., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., James Jones, H., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Ross Berger, A., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Barohn, R., Dimachkie, M., Glenn, M., Mcvey, A., Pasnoor, M., Statland, J., Wang, Y., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., Disanzo, B., Naunton, K., Benatar, M., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Traub, R., Chopra, M., Vu, T., Katzin, L., Mcclain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Polaka, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Henderson, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Nowak, R., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., Vu, H., Mantegazza, R., O'Brien, F. L., Yountz, M., Howard, J. F., Gabriel Mazia, C., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Dalila Garcia, A., De Bleecker, J., Van den Abeele, G., de Koning, K., De Mey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., Van Damme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., Van Daele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hont, A., Tilkin, P., Alves de Siqueira Carvalho, A., Dias Brockhausen, I., Feder, D., Ambrosio, D., Cesar, P., Paula Melo, A., Martins Ribeiro, R., Rocha, R., Bezerra Rosa, B., Veiga, T., Augusto da Silva, L., Santos Engel, M., Goncalves Geraldo, J., da Penha Ananias Morita, M., Nogueira Coelho, E., Paiva, G., Pozo, M., Prando, N., Torres, D. D. M., Fernanda Butinhao, C., Duran, G., Augusto Suriane Fialho, T., Gomes da Silva, T. C., Goncalves, L. O. M., Eduardo Pazetto, L., Renata Cubas Volpe, L., Souza Duca, L., Friedrich, M. A. G., Guerreiro, A., Mohr, H., Pereira Martins, M., da Cruz Pacheco, D., Ferreira, L., Paula Macagnan, A., Pinto, G., de Cassia Santos, A., Souza Bulle Oliveira, A., Amaral de Andrade, A. C., Annes, M., Duarte Silva, L., Cavalcante Lino, V., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Siddiqi, Z., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova, J., Kurkova, B., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Andersen, H., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Vissing, J., Hojgaard, J., Witting, N., Mette Ostergaard Autzen, A., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., Di Pasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Pasquale, A., Evoli, A., Emilio Alboini, P., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Saccà, Francesco, Filla, Alessandro, Costabile, T., Marano, E., Fasanaro, A., Marsili, Angela, Puorro, Giorgia, Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Murai, H., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Utsugisawa, K., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Samukawa, M., Suzuki, H., Morikawa, M., Kamakura, S., Miyawaki, E., Okumura, M., Funaka, S., Kawamura, T., Nakamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Shiraishi, H., Miyazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., van der Kooi, A., de Visser, M., Gibson, T., Kim, B. -J., Nyoung Lee, C., Seo Koo, Y., Youl Seok, H., Nam Kang, H., Ra, H., Joon Kim, B., Bin Cho, E., Choi, M., Lee, H., Min, J. -H., Seok, J., Lee, J., Koh, D. Y., Kwon, J., Park, S., Haw Choi, E., Hong, Y. -H., Ahn, S. -H., Lim Koo, D., Lim, J. -S., Won Shin, C., Ye Hwang, J., Kim, M., Min Kim, S., Jeong, H. -N., Jung, J., Kim, Y. -H., Seok Lee, H., Young Shin, H., Bi Hwang, E., Shin, M., Casasnovas, C., Antonia Alberti Aguilo, M., Homedes-Pedret, C., Julia Palacios, N., Diez Porras, L., Velez Santamaria, V., Lazaro, A., Gamez Carbonell, J., Sune, P., Salvado Figueras, M., Gili, G., Mazuela, G., Illa, I., Cortes Vicente, E., Diaz-Manera, J., Antonio Querol Gutierrez, L., Rojas Garcia, R., Vidal, N., Arribas-Ibar, E., Diez Tejedor, E., Gomez Salcedo, P., Fernandez-Fournier, M., Lopez Ruiz, P., Rodriguez de Rivera, F. J., Sastre, M., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C., Pinar Acar, N., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Kazim Onar, M., Sen, S., Kirbas Cavdar, T., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Jacob, S., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Sanjak, M., Plevka, S., Burdette, M., Cunningham, S., Kramer, M., Nemeth, J., Schommer, C., Scott, T., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guingrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Carter, C., Donahue, C., Herbert, C., Scala, S., Weiner, E., Alam, S., Mckinnon, J., Haar, L., Mckinnon, N., Alcon, K., Mckenna, K., Sattar, N., Daniels, K., Jeffery, D., Freimer, M., Chad Hoyle, J., Kissel, J., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Ardoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelbach, J., Muppidi, S., Goyal, N., Sakamuri, S., So, Y. T., Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., Tobon Gonzalez, A., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Mozaffar, T., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., James Jones, H., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Ross Berger, A., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Barohn, R., Dimachkie, M., Glenn, M., Mcvey, A., Pasnoor, M., Statland, J., Wang, Y., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., Disanzo, B., Naunton, K., Benatar, M., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Traub, R., Chopra, M., Vu, T., Katzin, L., Mcclain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Polaka, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Henderson, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Nowak, R., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., Vu, H., Neurology, and Amsterdam Neuroscience - Neuroinfection & -inflammation
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0301 basic medicine ,Malalties neuromusculars ,Activities of daily living ,Autoimmune diseases ,Severity of Illness Index ,Complement inhibitor ,0302 clinical medicine ,CYCLOPHOSPHAMIDE ,Activities of Daily Living ,Outcome Assessment, Health Care ,Medicine and Health Sciences ,Research Articles ,Malalties autoimmunitàries ,General Neuroscience ,Eculizumab ,myasthenia ,Neuromuscular diseases ,Life Sciences & Biomedicine ,medicine.drug ,RC321-571 ,Research Article ,Adult ,medicine.medical_specialty ,Cyclophosphamide ,Gross motor skill ,Clinical Neurology ,Neurosciences. Biological psychiatry. Neuropsychiatry ,Placebo ,Antibodies, Monoclonal, Humanized ,ACETYLCHOLINE-RECEPTOR ,03 medical and health sciences ,Refractory ,Double-Blind Method ,Internal medicine ,Myasthenia Gravis ,medicine ,Humans ,Muscle Strength ,Patient Reported Outcome Measures ,RC346-429 ,Muscle, Skeletal ,Science & Technology ,business.industry ,Neurosciences ,medicine.disease ,Myasthenia gravis ,030104 developmental biology ,Complement Inactivating Agents ,ANTIBODY ,Monoclonal antibodies ,Neurosciences & Neurology ,Neurology (clinical) ,Neurology. Diseases of the nervous system ,business ,Anticossos monoclonals ,030217 neurology & neurosurgery - Abstract
OBJECTIVE: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. METHODS: Patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open-label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open-label extension were analyzed. RESULTS: Of the 125 patients who participated in REGAIN, 117 enrolled in the open-label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open-label extension. INTERPRETATION: Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis. ispartof: ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY vol:7 issue:8 pages:1327-1339 ispartof: location:United States status: published
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- 2020
9. Low Density Supersonic Decelerator (LDSD) Supersonic Flight Dynamics Test (SFDT) Plume Induced Environment Modelling
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Mobley, B. L, Smith, S. D, Van Norman, J. W, Muppidi, S, and Clark, I
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Aerodynamics ,Spacecraft Design, Testing And Performance - Abstract
Provide plume induced heating (radiation & convection) predictions in support of the LDSD thermal design (pre-flight SFDT-1) Predict plume induced aerodynamics in support of flight dynamics, to achieve targeted freestream conditions to test supersonic deceleration technologies (post-flight SFDT-1, pre-flight SFDT-2)
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- 2016
10. PND10 Conceptualization and Novel Budget Impact Analysis Framework for Treatments of Myasthenia Gravis
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Phillips, G.A., primary, Muppidi, S., additional, Sahagian, G., additional, Smith, G., additional, Huang, D., additional, and Campbell, D., additional
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- 2021
- Full Text
- View/download PDF
11. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
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Mantegazza, R, Wolfe, GI, Muppidi, S, Wiendl, H, Fujita, KP, O'Brien, FL, Booth, HDE, Howard, JF, Illa I., Cortés-Vicente E., Díaz-Manera J., Querol L.A., Rojas-García R., Vidal-Fernandez N., and REGAIN Study Group
- Abstract
Objective To evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension. Methods Patients who completed the REGAIN randomized controlled trial and continued into the open-label extension were included in this tertiary endpoint analysis. Patients were assessed for the MGFA post-intervention status of improved, unchanged, worse, MM, and pharmacologic remission at defined time points during REGAIN and through week 130 of the open-label study. Results A total of 117 patients completed REGAIN and continued into the open-label study (eculizumab/eculizumab: 56; placebo/eculizumab: 61). At week 26 of REGAIN, more eculizumab-treated patients than placebo-treated patients achieved a status of improved (60.7% vs 41.7%) or MM (25.0% vs 13.3%; common OR: 2.3; 95% CI: 1.1-4.5). After 130 weeks of eculizumab treatment, 88.0% of patients achieved improved status and 57.3% of patients achieved MM status. The safety profile of eculizumab was consistent with its known profile and no new safety signals were detected. Conclusion Eculizumab led to rapid and sustained achievement of MM in patients with AChR+ refractory gMG. These findings support the use of eculizumab in this previously difficult-to-treat patient population.
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- 2021
12. Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab:subgroup analysis of REGAIN and its extension study
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Siddiqi, Z. A., Nowak, R. J., Mozaffar, T., O'Brien, F., Yountz, M., Patti, F., Mazia, C. G., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Garcia, A. D., De Bleecker, J., Van den Abeele, G., de Koning, K., De Mey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., Van Damme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., Van Daele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hont, A., Tilkin, P., de Siqueira Carvalho, A. A., Brockhausen, I. D., Feder, D., Ambrosio, D., Cesar, P., Melo, A. P., Ribeiro, R. M., Rocha, R., Rosa, B. B., Veiga, T., da Silva, L. A., Engel, M. S., Geraldo, J. G., da Penha Ananias Morita, M., Coelho, E. N., Paiva, G., Pozo, M., Prando, N., Torres, D. D. M., Butinhao, C. F., Duran, G., Fialho, T. A. S., da Silva, T. C. G., Goncalves, L. O. M., Pazetto, L. E., Volpe, L. R. C., Duca, L. S., Friedrich, M. A. G., Guerreiro, A., Mohr, H., Martins, M. P., da Cruz Pacheco, D., Ferreira, L., Macagnan, A. P., Pinto, G., de Cassia Santos, A., Oliveira, A. S. B., de Andrade, A. C. A., Annes, M., Silva, L. D., Lino, V. C., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova, J., Kurkova, B., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Andersen, H., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Vissing, J., Hojgaard, J., Witting, N., Autzen, A. M. O., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., Di Pasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Sacca, F., Filla, A., Costabile, T., Marano, E., Fasanaro, A., Marsili, A., Puorro, G., Mantegazza, R., Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Evoli, A., Alboini, P. E., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Murai, H., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Utsugisawa, K., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Samukawa, M., Suzuki, H., Morikawa, M., Kamakura, S., Miyawaki, E., Shiraishi, H., Miyazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Okumura, M., Funaka, S., Kawamura, T., Nakamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., Kim, B. -J., Lee, C. N., Koo, Y. S., Seok, H. Y., Kang, H. N., H. J., Ra, Kim, B. J., Cho, E. B., Choi, M. S., Lee, H. L., Min, J. -H., Seok, J., Lee, J. E., Koh, D. Y., Kwon, J. Y., Park, S. A., Choi, E. H., Hong, Y. -H., Ahn, S. -H., Koo, D. L., Lim, J. -S., Shin, C. W., Hwang, J. Y., Kim, M., Kim, S. M., Jeong, H. -N., Jung, J. W., Kim, Y. -H., Lee, H. S., Shin, H. Y., Hwang, E. B., Shin, M., van der Kooi, A., de Visser, M., Gibson, T., Casasnovas, C., Aguilo, M. A. A., Homedes-Pedret, C., Palacios, N. J., Porras, L. D., Santamaria, V. V., Lazaro, A., Tejedor, E. D., Salcedo, P. G., Fernandez-Fournier, M., Ruiz, P. L., de Rivera, F. J. R., Sastre, M., Carbonell, J. G., Sune, P., Figueras, M. S., Gili, G., Mazuela, G., Illa, I., Vicente, E. C., Diaz-Manera, J., Gutierrez, L. A. Q., Garcia, R. R., Vidal, N., Arribas-Ibar, E., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C. E., Acar, N. P., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Onar, M. K., Sen, S., Cavdar, T. K., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Jacob, S., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Sanjak, M., Plevka, S., Burdette, M., Cunningham, S., Kramer, M., Nemeth, J., Schommer, C., Scott, T., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guingrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Carter, C., Donahue, C., Herbert, C., Scala, S., Weiner, E., Alam, S., Mckinnon, J., Haar, L., Mckinnon, N., Alcon, K., Mckenna, K., Sattar, N., Daniels, K., Jeffery, D., Freimer, M., Hoyle, J. C., Kissel, J., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Ardoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelbach, J., Muppidi, S., Goyal, N., Sakamuri, S., Y. T., So, Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., Gonzalez, A. T., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., Jones, H. J., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Berger, A. R., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Barohn, R., Dimachkie, M., Glenn, M., Mcvey, A., Pasnoor, M., Statland, J., Wang, Y., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., Disanzo, B., Naunton, K., Benatar, M., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Howard, J., Traub, R., Chopra, M., Vu, T., Katzin, L., Mcclain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Polaka, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Henderson, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., Vu, H., Neurology, ANS - Neuroinfection & -inflammation, and EURO-NMD
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medicine.medical_specialty ,Physiology ,Population ,Subgroup analysis ,Antibodies, Monoclonal, Humanized ,Placebo ,Cellular and Molecular Neuroscience ,rituximab ,Refractory ,immune system diseases ,Physiology (medical) ,Internal medicine ,Activities of Daily Living ,medicine ,Humans ,education ,education.field_of_study ,myasthenia gravis ,acetylcholine receptor ,business.industry ,Eculizumab ,medicine.disease ,Confidence interval ,Myasthenia gravis ,refractory ,Rituximab ,eculizumab ,Neurology (clinical) ,business ,medicine.drug - Abstract
Introduction/Aims: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti-acetylcholine receptor antibody-positive (AChR+) gMG previously treated with rituximab. Methods: This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open-label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE. Results: Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous-rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no-previous-rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous-rituximab and no-previous-rituximab groups (least-squares mean −4.4, standard error of the mean [SEM] 1.0 [n = 9] and least-squares mean −4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval −1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post-intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile. Discussion: Eculizumab is an effective therapy for patients with refractory AChR+ gMG, irrespective of whether they had received rituximab treatment previously.
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- 2021
13. Eculizumab Improves Fatigue in Refractory Generalized Myasthenia Gravis
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Andersen H., Mantegazza R., Wang J. J., O'Brien F., Patra K., Howard J. F., Mazia C. G., Wilken M., Barroso F., Saba J., Rugiero M., Bettini M., Chaves M., Vidal G., Garcia A. D., DeBleecker J., Van denAbeele G., deKoning K., DeMey K., Mercelis R., Mahieu D., Wagemaekers L., VanDamme P., Depreitere A., Schotte C., Smetcoren C., Stevens O., VanDaele S., Vandenbussche N., Vanhee A., Verjans S., Vynckier J., D'Hondt A., Tilkin P., AlvesdeSiqueira Carvalho A., DiasBrockhausen I., Feder D., Ambrosio D., Cesar P., Melo A. P., MartinsRibeiro R., Rocha R., BezerraRosa B., Veiga T., daSilva L. A., SantosEngel M., GoncalvesGeraldo J., daPenha AnaniasMorita M., NogueiraCoelho E., Paiva G., Pozo M., Prando N., MartineliTorres D. D., Butinhao C. F., Duran G., Gomes daSilva T. C., Otavio MaiaGoncalves L., Pazetto L. E., Fialho T. A. S., Renata CubasVolpe L., SouzaDuca L., GhellerFriedrich M. A., Guerreiro A., Mohr H., PereiraMartins M., daCruz Pacheco D., Ferreira L., Macagnan A. P., Pinto G., deCassia Santos A., Souza BulleOliveira A., AmaralAndrade A. C., Annes M., Duarte Silva L., CavalcanteLino V., Pinto W., Assis N., Carrara F., Miranda C., Souza I., Fernandes P., Siddiqi Z., Phan C., Narayan J., Blackmore D., Mallon A., Roderus R., Watt E., Vohanka S., Bednarik J., Chmelikova M., Cierny M., Toncrova S., Junkerova J., Kurkova B., Reguliova K., Zapletalova O., Pitha J., Novakova I., Tyblova M., Jurajdova I., Wolfova M., Harbo T., Vinge L., Krogh S., Mogensen A., Vissing J., Hojgaard J., Witting N., OstergaardAutzen A., Pedersen J., Eralinna J. -P., Laaksonen M., Oksaranta O., Harrison T., Eriksson J., Rozsa C., Horvath M., Lovas G., Matolcsi J., Szabo G., Jakab G., Szabadosne B., Vecsei L., Dezsi L., Varga E., Konyane M., Antonini G., DiPasquale A., Garibaldi M., Morino S., Troili F., Fionda L., Filla A., Costabile T., Marano E., Sacca F., Fasanaro A., Marsili A., Puorro G., Antozzi C., Bonanno S., Camera G., Locatelli A., Maggi L., Pasanisi M., Campanella A., Evoli A., Alboini P. E., D'Amato V., Iorio R., Inghilleri M., Frasca V., Giacomelli E., Gori M., Lopergolo D., Onesti E., Gabriele M., Uzawa A., Kanai T., Kawaguchi N., Mori M., Kaneko Y., Kanzaki A., Kobayashi E., Murai H., Masaki K., Matsuse D., Matsushita T., Uehara T., Shimpo M., Jingu M., Kikutake K., Nakamura Y., Sano Y., Utsugisawa K., Nagane Y., Kamegamori I., Tsuda T., Fujii Y., Futono K., Ozawa Y., Mizugami A., Saito Y., Suzuki H., Morikawa M., Samukawa M., Kamakura S., Miyawaki E., Shiraishi H., Mitazaki T., Motomura M., Mukaino A., Yoshimura S., Asada S., Yoshida S., Amamoto S., Kobashikawa T., Koga M., Maeda Y., Takada K., Takada M., Tsurumaru M., Yamashita Y., Suzuki Y., Akiyama T., Narikawa K., Tano O., Tsukita K., Kurihara R., Meguro F., Fukuda Y., Sato M., Okumura M., Funaka S., Kawamura T., Makamori M., Takahashi M., Taichi N., Hasuike T., Higuchi E., Kobayashi H., Osakada K., Imai T., Tsuda E., Shimohama S., Hayashi T., Hisahara S., Kawamata J., Murahara T., Saitoh M., Suzuki S., Yamamoto D., Ishiyama Y., Ishiyama N., Noshiro M., Takeyama R., Uwasa K., Yasuda I., van derKooi A., deVisser M., Gibson T., Kim B. -J., Lee C. N., Koo Y. S., Seok H. Y., Kang H. N., Ra H. J., Kim B. J., Cho E. B., Choi M. S., Lee H. L., Min J. -H., Seok J., Lee J. E., Koh D. Y., Kwon J. Y., Park S. A., Choi E. H., Hong Y. -H., Ahn S. -H., Koo D. L., Lim J. -S., Shin C. W., Hwang J. Y., Kim M., Kim S. M., Jeong H. -N., Jung J. W., Kim Y. -H., Lee H. S., Shin H. Y., Hwang E. B., Shin M., Casasnovas C., AlbertiAguilo M. A., Homedes-Pedret C., JuliaPalacios N., DiezPorras L., VelezSantamaria V., Lazaro A., DiezTejedor E., Gomez Salcedo P., Fernandez-Fournier M., LopezRuiz P., Rodriguez deRivera F. J., Sastre M., Gamez J., Sune P., Salvado M., Gili G., Mazuela G., Illa I., CortesVicente E., Diaz-Manera J., QuerolGutierrez L. A., RojasGarcia R., Vidal N., Arribas-Ibar E., Piehl F., Hietala A., Bjarbo L., Sengun I., Meherremova A., Ozcelik P., Balkan B., Tuga C., Ugur M., Erdem-Ozdamar S., Bekircan-Kurt C. E., Acar N. P., Yilmaz E., Caliskan Y., Orsel G., Efendi H., Aydinlik S., Cavus H., Kutlu A., Becerikli G., Semiz C., Tun O., Terzi M., Dogan B., Onar M. K., Sen S., KirbasCavdar T., Veske A., Norwood F., Dimitriou A., Gollogly J., Mahdi-Rogers M., Seddigh A., Sokratous G., Maier G., Sohail F., Jacob S., Sadalage G., Torane P., Brown C., Shah A., Sathasivam S., Arndt H., Davies D., Watling D., Amato A., Cochrane T., Salajegheh M., Roe K., Amato K., Toska S., Wolfe G., Silvestri N., Patrick K., Zakalik K., Katz J., Miller R., Engel M., Forshew D., Bravver E., Brooks B., Plevka S., Burdette M., Cunningham S., Sanjak M., Kramer M., Nemeth J., Schommer C., Tierney S., Juel V., Guptill J., Hobson-Webb L., Massey J., Beck K., Carnes D., Loor J., Anderson A., Pascuzzi R., Bodkin C., Kincaid J., Snook R., Guingrich S., Micheels A., Chaudhry V., Corse A., Mosmiller B., Kelley A., Ho D., Srinivasan J., Vytopil M., Jara J., Ventura N., Scala S., Carter C., Donahue C., Herbert C., Weiner E., Alam S., McKinnon J., Haar L., McKinnon N., Alcon K., McKenna K., Sattar N., Daniels K., Jeffery D., Kissel J., Freimer M., Hoyle J. C., Agriesti J., Chelnick S., Mezache L., Pineda C., Muharrem F., Karam C., Khoury J., Marburger T., Kaur H., Dimitrova D., Gilchrist J., Agrawal B., Elsayed M., Kohlrus S., Andoin A., Darnell T., Golden L., Lokaitis B., Seelback J., Muppidi S., Goyal N., Sakamuri S., So Y. T., Paulose S., Pol S., Welsh L., Bhavaraju-Sanka R., TobonGonzales A., Dishman L., Jones F., Gonzalez A., Padilla P., Saklad A., Silva M., Nations S., Trivedi J., Hopkins S., Kazamel M., Alsharabati M., Lu L., Nozaki K., Mumfrey-Thomas S., Woodall A., Mozaffar T., Cash T., Roy G., Mathew V., Maqsood F., Minton B., Jones H. J., Rosenfeld J., Garcia R., Echevarria L., Garcia S., Pulley M., Aranke S., Berger A. R., Shah J., Shabbir Y., Smith L., Varghese M., Gutmann L., Jerath N., Nance C., Swenson A., Olalde H., Kressin N., Sieren J., Barohn R., Dimachkie M., Glenn M., McVey A., Pasnoor M., Statland J., Wang J., Liu T., Emmons K., Jenci N., Locheke J., Fondaw A., Johns K., Rico G., Walsh M., Herbelin L., Hafer-Macko C., Kwan J., Zilliox L., Callison K., Young V., DiSanzo B., Naunton K., Benatar M., Bilsker M., Sharma K., Cooley A., Reyes E., Michon S. -C., Sheldon D., Steele J., Chopra M., Traub R., Vu T., Katzin L., McClain T., Harvey B., Hart A., Huynh K., Beydoun S., Chilingaryan A., Doan V., Droker B., Gong H., Karimi S., Lin F., Pokala K., Tran A., Akhter S., Malekniazi A., Tandan R., Hehir M., Waheed W., Lucy S., Weiss M., Distad J., Strom S., Downing S., Kim B., Bertorini T., Arnold T., Hendersen K., Pillai R., Liu Y., Wheeler L., Hewlett J., Vanderhook M., Nowak R., Dicapua D., Keung B., Kumar A., Patwa H., Robeson K., Yang I., Nye J., Vu H., Andersen, H., Mantegazza, R., Wang, J. J., O'Brien, F., Patra, K., Howard, J. F., Mazia, C. G., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Garcia, A. D., Debleecker, J., Van denAbeele, G., Dekoning, K., Demey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., Vandamme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., Vandaele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hondt, A., Tilkin, P., AlvesdeSiqueira Carvalho, A., Diasbrockhausen, I., Feder, D., Ambrosio, D., Cesar, P., Melo, A. P., Martinsribeiro, R., Rocha, R., Bezerrarosa, B., Veiga, T., Dasilva, L. A., Santosengel, M., Goncalvesgeraldo, J., daPenha AnaniasMorita, M., Nogueiracoelho, E., Paiva, G., Pozo, M., Prando, N., Martinelitorres, D. D., Butinhao, C. F., Duran, G., Gomes daSilva, T. C., Otavio MaiaGoncalves, L., Pazetto, L. E., Fialho, T. A. S., Renata CubasVolpe, L., Souzaduca, L., Ghellerfriedrich, M. A., Guerreiro, A., Mohr, H., Pereiramartins, M., daCruz Pacheco, D., Ferreira, L., Macagnan, A. P., Pinto, G., deCassia Santos, A., Souza BulleOliveira, A., Amaralandrade, A. C., Annes, M., Duarte Silva, L., Cavalcantelino, V., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Siddiqi, Z., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova, J., Kurkova, B., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Vissing, J., Hojgaard, J., Witting, N., Ostergaardautzen, A., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., Dipasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Filla, A., Costabile, T., Marano, E., Sacca, F., Fasanaro, A., Marsili, A., Puorro, G., Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Evoli, A., Alboini, P. E., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Murai, H., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Utsugisawa, K., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Suzuki, H., Morikawa, M., Samukawa, M., Kamakura, S., Miyawaki, E., Shiraishi, H., Mitazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Okumura, M., Funaka, S., Kawamura, T., Makamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., van derKooi, A., Devisser, M., Gibson, T., Kim, B. -J., Lee, C. N., Koo, Y. S., Seok, H. Y., Kang, H. N., Ra, H. J., Kim, B. J., Cho, E. B., Choi, M. S., Lee, H. L., Min, J. -H., Seok, J., Lee, J. E., Koh, D. Y., Kwon, J. Y., Park, S. A., Choi, E. H., Hong, Y. -H., Ahn, S. -H., Koo, D. L., Lim, J. -S., Shin, C. W., Hwang, J. Y., Kim, M., Kim, S. M., Jeong, H. -N., Jung, J. W., Kim, Y. -H., Lee, H. S., Shin, H. Y., Hwang, E. B., Shin, M., Casasnovas, C., Albertiaguilo, M. A., Homedes-Pedret, C., Juliapalacios, N., Diezporras, L., Velezsantamaria, V., Lazaro, A., Dieztejedor, E., Gomez Salcedo, P., Fernandez-Fournier, M., Lopezruiz, P., Rodriguez deRivera, F. J., Sastre, M., Gamez, J., Sune, P., Salvado, M., Gili, G., Mazuela, G., Illa, I., Cortesvicente, E., Diaz-Manera, J., Querolgutierrez, L. A., Rojasgarcia, R., Vidal, N., Arribas-Ibar, E., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C. E., Acar, N. P., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Onar, M. K., Sen, S., Kirbascavdar, T., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Jacob, S., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Plevka, S., Burdette, M., Cunningham, S., Sanjak, M., Kramer, M., Nemeth, J., Schommer, C., Tierney, S., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guingrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Scala, S., Carter, C., Donahue, C., Herbert, C., Weiner, E., Alam, S., Mckinnon, J., Haar, L., Mckinnon, N., Alcon, K., Mckenna, K., Sattar, N., Daniels, K., Jeffery, D., Kissel, J., Freimer, M., Hoyle, J. C., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Andoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelback, J., Muppidi, S., Goyal, N., Sakamuri, S., So, Y. T., Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., Tobongonzales, A., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Mozaffar, T., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., Jones, H. J., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Berger, A. R., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Barohn, R., Dimachkie, M., Glenn, M., Mcvey, A., Pasnoor, M., Statland, J., Wang, J., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., Disanzo, B., Naunton, K., Benatar, M., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Chopra, M., Traub, R., Vu, T., Katzin, L., Mcclain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Pokala, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Hendersen, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Nowak, R., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., and Vu, H.
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Male ,Activities of daily living ,Outcome Assessment ,Myasthenia Gravis/drug therapy ,Myasthenia gravi ,Placebos ,0302 clinical medicine ,Quality of life ,QUALITY-OF-LIFE ,Outcome Assessment, Health Care ,Monoclonal ,Receptors ,Activities of Daily Living ,Medicine and Health Sciences ,Clinical endpoint ,Receptors, Cholinergic ,Fatigue/drug therapy ,Myasthenia gravis ,Humanized ,Fatigue ,Cholinergic ,Public, Environmental & Occupational Health ,030503 health policy & services ,Terminal complement inhibition ,Eculizumab ,Middle Aged ,humanities ,Antibodies, Monoclonal, Humanized/therapeutic use ,030220 oncology & carcinogenesis ,Health Policy & Services ,Female ,0305 other medical science ,Life Sciences & Biomedicine ,medicine.drug ,medicine.medical_specialty ,Complement ,Brief Communication ,Antibodies, Monoclonal, Humanized ,Placebo ,Antibodies ,03 medical and health sciences ,Neuro-QOL Fatigue ,Refractory ,Internal medicine ,Myasthenia Gravis ,medicine ,Humans ,Generalized myasthenia ,Placebos/therapeutic use ,Quality of Life ,Receptors, Cholinergic/immunology ,Science & Technology ,business.industry ,Public Health, Environmental and Occupational Health ,Correction ,medicine.disease ,Health Care ,Health Care Sciences & Services ,business - Abstract
Purpose To evaluate the effect of eculizumab on perceived fatigue in patients with anti-acetylcholine receptor antibody-positive, refractory, generalized myasthenia gravis (MG) using the Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue subscale, and to evaluate correlations between improvements in Neuro-QOL Fatigue and other clinical endpoints. Methods Neuro-QOL Fatigue, MG Activities of Daily Living (MG-ADL), Quantitative MG (QMG), and the 15-item MG Quality of Life (MG-QOL15) scales were administered during the phase 3, randomized, placebo-controlled REGAIN study (eculizumab, n = 62; placebo, n = 63) and subsequent open-label extension (OLE). Data were analyzed using repeated-measures models. Correlations between changes in Neuro-QOL Fatigue and in MG-ADL, QMG, and MG-QOL15 scores were determined at REGAIN week 26. Results At REGAIN week 26, eculizumab-treated patients showed significantly greater improvements in Neuro-QOL Fatigue scores than placebo-treated patients (consistent with improvements in MG-ADL, QMG, and MG-QOL15 scores previously reported in REGAIN). Improvements with eculizumab were sustained through OLE week 52. Correlations between Neuro-QOL Fatigue and MG-QOL15, MG-ADL, and QMG scores were strong for eculizumab-treated patients at REGAIN week 26, and strong, moderate, and weak, respectively, for placebo-treated patients. Conclusions Compared with placebo, eculizumab was associated with improvements in perceived fatigue that strongly correlated with improvements in MG-specific outcome measures. Trial ID Registration: NCT01997229, NCT02301624.
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- 2019
14. Guidance for the management of myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) during the COVID-19 pandemic
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Jacob, S, Muppidi, S, Guidon, A, Guptill, J, Hehir, M, Howard, JF, Illag, I, Mantegazza, R, Murai, H, Utsugisawa, K, Vissing, J, Wiendl, H, Nowak, RJ, and Int MG COVID-19 Working Grp
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- 2020
15. ‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab
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Vissing, J., Jacob, S., Fujita, K. P., O'Brien, F., Howard, J. F., Mazia, C. G., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Garcia, A. D., DeBleecker, J., Vanden Abeele, G., deKoning, K., DeMey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., VanDamme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., VanDaele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hont, A., Tilkin, P., Alves deSiqueira Carvalho, A., DiasBrockhausen, I., Feder, D., Ambrosio, D., Cesar, P., Melo, A. P., MartinsRibeiro, R., Rocha, R., Rosa, B. B., Veiga, T., daSilva, L. A., SantosEngel, M., GoncalvesGeraldo, J., daPenha Ananias Morita, M., NogueiraCoelho, E., Paiva, G., Pozo, M., Prando, N., MartineliTorres, D. D., Butinhao, C. F., Duran, G., SurianeFialho, T. A., Gomes daSilva, T. C., MaiaGoncalves, L. O., Pazetto, L. E., CubasVolpe, L. R., SouzaDuca, L., GhellerFriedrich, M. A., Guerreiro, A., Mohr, H., PereiraMartins, M., daCruz Pacheco, D., Ferreira, L., Macagnan, A. P., Pinto, G., deCassia Santos, A., Souza BulleOliveira, A., Amaral deAndrade, A. C., Annes, M., DuarteSilva, L., CavalcanteLino, V., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Siddiqi, Z., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova BarboraKurkova, J., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Andersen, H., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Hojgaard, J., Witting, N., Mette OstergaardAutzen, A., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., DiPasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Sacca, F., Previous, Filla, A., Sub-Investigators, Costabile, T., Marano, E., Fasanaro, A., Marsili, A., Puorro, G., Mantegazza, R., Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Evoli, A., Alboini, P. E., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Murai, H., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Utsugisawa, K., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Samukawa, M., Suzuki, H., Morikawa, M., Kamakura, S., Miyawaki, E., Shiraishi, H., Mitazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Okumura, M., Funaka, S., Kawamura, T., Makamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., Kim, B. -J., Lee, C. N., Koo, Y. S., Seok, H. Y., Kang, H. N., H. J., Ra, Kim, B. J., Cho, E. B., Choi, M. S., Lee, H. L., Min, J. -H., Seok, J., Lee, J. E., Koh, D. Y., Kwon, J. Y., Park, S. A., Choi, E. H., Hong, Y. -H., Ahn, S. -H., Koo, D. L., Lim, J. -S., Shin, C. W., Hwang, J. Y., Kim, M., Kim, S. M., Jeong, H. -N., Jung, J. W., Kim, Y. -H., Lee, H. S., Shin, H. Y., Hwang, E. B., Shin, M., van derKooi, A., deVisser, M., Gibson, T., Casasnovas, C., AlbertiAguilo, M. A., Homedes-Pedret, C., Palacios, N. J., DiezPorras, L., VelezSantamaria, V., Lazaro, A., DiezTejedor, E., GomezSalcedo, P., Fernandez-Fournier, M., LopezRuiz, P., Rodriguez deRivera, F. J., Sastre, M., GamezCarbonell, J., Sune, P., SalvadoFigueras, M., Gili, G., Mazuela, G., Illa, I., CortesVicente, E., Diaz-Manera, J., QuerolGutierrez, L. A., RojasGarcia, R., Vidal, N., Arribas-Ibar, E., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C. E., Acar, N. P., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Onar, M. K., Sen, S., KirbasCavdar, T., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Sanjak, M., Plevka, S., Burdette, M., Cunningham, S., Kramer, M., Nemeth, J., Schommer, C., Tinerney, S., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guinrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Carter, C., Donahue, C., Herbert, C., Scala, S., Weiner, E., Alam, S., McKinnon, J., Haar, L., McKinnon, N., Alcon, K., McKenna, K., Sattar, N., Daniels, K., Jeffery, D., Freimer, M., Hoyle, J. C., Kissel, J., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Andoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelbach, J., Muppidi, S., Goyal, N., Sakamuri, S., Y. T., So, Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., TobonGonzalez, A., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Mozaffar, T., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., Jones, H. J., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Berger, A. R., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Barohn, R., Dimachkie, M., Glenn, M., McVey, A., Pasnoor, M., Statland, J., Wang, Y., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., DiSanzo, B., Naunton, K., Benatar, M., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Traub, R., Chopra, M., Vu, T., Katzin, L., McClain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Polaka, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Henderson, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Nowak, R., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., and Vu, H.
- Subjects
Acetylcholine receptor ,Eculizumab ,Minimal symptom expression ,Myasthenia gravis ,Refractory ,Adult ,Aged ,Antibodies, Monoclonal, Humanized ,Autoantibodies ,Double-Blind Method ,Female ,Humans ,Immunologic Factors ,Male ,Middle Aged ,Myasthenia Gravis ,Receptors, Cholinergic ,Activities of Daily Living ,Patient Reported Outcome Measures ,Quality of Life - Published
- 2020
16. Epidemiological evidence for a hereditary contribution to myasthenia gravis: A retrospective cohort study of patients from North America
- Author
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Green, J. D., Barohn, R. J., Bartoccion, E., Benatar, M., Blackmore, D., Chaudhry, V., Chopra, M., Corse, A., Dimachkie, M. M., Evoli Stampanoni-B, Amelia, Florence, J., Freimer, M., Howard, J. F., Jiwa, T., Kaminski, H. J., Kissel, J. T., Koopman, W. J., Lipscomb, B., Maestri, M., Marino, Mariapaola, Massey, J. M., Mcvey, A., Mezei, M. M., Muppidi, S., Nicolle, M. W., Oger, J., Pascuzzi, R. M., Pasnoor, M., Pestronk, A., Provenzano, Carlo, Ricciardi, R., Richman, D. P., Rowin, J., Sanders, D. B., Siddiqi, Z., Soloway, A., Wolfe, G. I., Wulf, C., Drachman, D. B., Traynor, B. J., Evoli A. (ORCID:0000-0003-0282-8787), Marino M. (ORCID:0000-0001-9155-6378), Provenzano C. (ORCID:0000-0001-5476-5517), Green, J. D., Barohn, R. J., Bartoccion, E., Benatar, M., Blackmore, D., Chaudhry, V., Chopra, M., Corse, A., Dimachkie, M. M., Evoli Stampanoni-B, Amelia, Florence, J., Freimer, M., Howard, J. F., Jiwa, T., Kaminski, H. J., Kissel, J. T., Koopman, W. J., Lipscomb, B., Maestri, M., Marino, Mariapaola, Massey, J. M., Mcvey, A., Mezei, M. M., Muppidi, S., Nicolle, M. W., Oger, J., Pascuzzi, R. M., Pasnoor, M., Pestronk, A., Provenzano, Carlo, Ricciardi, R., Richman, D. P., Rowin, J., Sanders, D. B., Siddiqi, Z., Soloway, A., Wolfe, G. I., Wulf, C., Drachman, D. B., Traynor, B. J., Evoli A. (ORCID:0000-0003-0282-8787), Marino M. (ORCID:0000-0001-9155-6378), and Provenzano C. (ORCID:0000-0001-5476-5517)
- Abstract
Objectives To approximate the rate of familial myasthenia gravis and the coexistence of other autoimmune disorders in the patients and their families. Design Retrospective cohort study. Setting Clinics across North America. Participants The study included 1032 patients diagnosed with acetylcholine receptor antibody (AChR)-positive myasthenia gravis. Methods Phenotype information of 1032 patients diagnosed with AChR-positive myasthenia gravis was obtained from clinics at 14 centres across North America between January 2010 and January 2011. A critical review of the epidemiological literature on the familial rate of myasthenia gravis was also performed. Results Among 1032 patients, 58 (5.6%) reported a family history of myasthenia gravis. A history of autoimmune diseases was present in 26.6% of patients and in 28.4% of their family members. Discussion The familial rate of myasthenia gravis was higher than would be expected for a sporadic disease. Furthermore, a high proportion of patients had a personal or family history of autoimmune disease. Taken together, these findings suggest a genetic contribution to the pathogenesis of myasthenia gravis.
- Published
- 2020
17. Quantitative sudomotor abnormalities in clinically isolated rapid eye movement sleep behavior disorder
- Author
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Zitser, J., primary, Muppidi, S., additional, Sinn, D.I., additional, Jaradeh, S., additional, and Miglis, M.G., additional
- Published
- 2020
- Full Text
- View/download PDF
18. MG-ADL: IS IT STILL A RELEVANT OUTCOME MEASURE?
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Muppidi, S., Wolfe, G. I., Conaway, M. R., and Burns, T. M.
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- 2010
19. Sudomotor abnormalities in idiopathic rem sleep behavior disorder
- Author
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Zitser, J., primary, Muppidi, S., additional, During, E., additional, Sinn, D.-I., additional, Jaradeh, S., additional, and Miglis, M., additional
- Published
- 2019
- Full Text
- View/download PDF
20. IVIG TREATMENT RESPONSE OF SUSPECTED CIDP PATIENTS NOT MEETING STANDARD DIAGNOSTIC CRITERIA
- Author
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Saperstein, D S, Levine, T D, Gorson, K G, Wolfe, G I, Muppidi, S, Shaibani, A, Hehir, M K, Burns, T, Hunt, M, Pasnoor, M, Dimachkie, M M, McVey, A L, and Barohn, R J
- Published
- 2008
21. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis
- Author
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Muppidi, S, Utsugisawa, K, Benatar, M, Murai, H, Barohn, RJ, Illa, I, Jacob, S, Vissing, J, Burns, TM, Kissel, JT, Nowak, RJ, Andersen, H, Casasnovas, C, de Bleecker, JL, Vu, TH, Mantegazza, R, O'Brien, FL, Wang, JJ, Fujita, KP, Howard, JF, Kaya, A, Khursigara, G, Armstrong, R, Diab, D, Capocelli, K, Lane, C, Sanders, V, Gandolfo, R, and REGAIN Study Grp
- Subjects
myasthenia gravis ,MG-QOL15 ,MGC ,MG-ADL ,QMG ,eculizumab - Abstract
Introduction: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy. Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results: The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001). Discussion: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019
- Published
- 2019
22. Correction to: Eculizumab improves fatigue in refractory generalized myasthenia gravis (Quality of Life Research, (2019), 28, 8, (2247-2254), 10.1007/s11136-019-02148-2)
- Author
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Andersen, H., Mantegazza, R., Wang, J. J., O'Brien, F., Patra, K., Howard, J. F., Mazia, C. G., Wilken, M., Barroso, F., Saba, J., Rugiero, M., Bettini, M., Chaves, M., Vidal, G., Garcia, A. D., Debleecker, J., Van denAbeele, G., Dekoning, K., Demey, K., Mercelis, R., Mahieu, D., Wagemaekers, L., Vandamme, P., Depreitere, A., Schotte, C., Smetcoren, C., Stevens, O., Vandaele, S., Vandenbussche, N., Vanhee, A., Verjans, S., Vynckier, J., D'Hondt, A., Tilkin, P., AlvesdeSiqueira Carvalho, A., Diasbrockhausen, I., Feder, D., Ambrosio, D., Cesar, P., Melo, A. P., Martinsribeiro, R., Rocha, R., Bezerrarosa, B., Veiga, T., Dasilva, L. A., Santosengel, M., Goncalvesgeraldo, J., daPenha AnaniasMorita, M., Nogueiracoelho, E., Paiva, G., Pozo, M., Prando, N., Martinelitorres, D. D., Butinhao, C. F., Duran, G., Gomes daSilva, T. C., Otavio MaiaGoncalves, L., Pazetto, L. E., Fialho, T. A. S., Renata CubasVolpe, L., Souzaduca, L., Ghellerfriedrich, M. A., Guerreiro, A., Mohr, H., Pereiramartins, M., daCruz Pacheco, D., Ferreira, L., Macagnan, A. P., Pinto, G., deCassia Santos, A., Souza BulleOliveira, A., Amaralandrade, A. C., Annes, M., Duarte Silva, L., Cavalcantelino, V., Pinto, W., Assis, N., Carrara, F., Miranda, C., Souza, I., Fernandes, P., Siddiqi, Z., Phan, C., Narayan, J., Blackmore, D., Mallon, A., Roderus, R., Watt, E., Vohanka, S., Bednarik, J., Chmelikova, M., Cierny, M., Toncrova, S., Junkerova, J., Kurkova, B., Reguliova, K., Zapletalova, O., Pitha, J., Novakova, I., Tyblova, M., Jurajdova, I., Wolfova, M., Harbo, T., Vinge, L., Krogh, S., Mogensen, A., Vissing, J., Hojgaard, J., Witting, N., Ostergaardautzen, A., Pedersen, J., Eralinna, J. -P., Laaksonen, M., Oksaranta, O., Harrison, T., Eriksson, J., Rozsa, C., Horvath, M., Lovas, G., Matolcsi, J., Szabo, G., Jakab, G., Szabadosne, B., Vecsei, L., Dezsi, L., Varga, E., Konyane, M., Antonini, G., Dipasquale, A., Garibaldi, M., Morino, S., Troili, F., Fionda, L., Filla, A., Costabile, T., Marano, E., Sacca, F., Fasanaro, A., Marsili, A., Puorro, G., Antozzi, C., Bonanno, S., Camera, G., Locatelli, A., Maggi, L., Pasanisi, M., Campanella, A., Evoli, A., Alboini, P. E., D'Amato, V., Iorio, R., Inghilleri, M., Frasca, V., Giacomelli, E., Gori, M., Lopergolo, D., Onesti, E., Gabriele, M., Uzawa, A., Kanai, T., Kawaguchi, N., Mori, M., Kaneko, Y., Kanzaki, A., Kobayashi, E., Murai, H., Masaki, K., Matsuse, D., Matsushita, T., Uehara, T., Shimpo, M., Jingu, M., Kikutake, K., Nakamura, Y., Sano, Y., Utsugisawa, K., Nagane, Y., Kamegamori, I., Tsuda, T., Fujii, Y., Futono, K., Ozawa, Y., Mizugami, A., Saito, Y., Suzuki, H., Morikawa, M., Samukawa, M., Kamakura, S., Miyawaki, E., Shiraishi, H., Mitazaki, T., Motomura, M., Mukaino, A., Yoshimura, S., Asada, S., Yoshida, S., Amamoto, S., Kobashikawa, T., Koga, M., Maeda, Y., Takada, K., Takada, M., Tsurumaru, M., Yamashita, Y., Suzuki, Y., Akiyama, T., Narikawa, K., Tano, O., Tsukita, K., Kurihara, R., Meguro, F., Fukuda, Y., Sato, M., Okumura, M., Funaka, S., Kawamura, T., Makamori, M., Takahashi, M., Taichi, N., Hasuike, T., Higuchi, E., Kobayashi, H., Osakada, K., Imai, T., Tsuda, E., Shimohama, S., Hayashi, T., Hisahara, S., Kawamata, J., Murahara, T., Saitoh, M., Suzuki, S., Yamamoto, D., Ishiyama, Y., Ishiyama, N., Noshiro, M., Takeyama, R., Uwasa, K., Yasuda, I., van derKooi, A., Devisser, M., Gibson, T., Kim, B. -J., Lee, C. N., Koo, Y. S., Seok, H. Y., Kang, H. N., H. J., Ra, Kim, B. J., Cho, E. B., Choi, M. S., Lee, H. L., Min, J. -H., Seok, J., Lee, J. E., Koh, D. Y., Kwon, J. Y., Park, S. A., Choi, E. H., Hong, Y. -H., Ahn, S. -H., Koo, D. L., Lim, J. -S., Shin, C. W., Hwang, J. Y., Kim, M., Kim, S. M., Jeong, H. -N., Jung, J. W., Kim, Y. -H., Lee, H. S., Shin, H. Y., Hwang, E. B., Shin, M., Casasnovas, C., Albertiaguilo, M. A., Homedes-Pedret, C., Juliapalacios, N., Diezporras, L., Velezsantamaria, V., Lazaro, A., Dieztejedor, E., Gomez Salcedo, P., Fernandez-Fournier, M., Lopezruiz, P., Rodriguez deRivera, F. J., Sastre, M., Gamez, J., Sune, P., Salvado, M., Gili, G., Mazuela, G., Illa, I., Cortesvicente, E., Diaz-Manera, J., Querolgutierrez, L. A., Rojasgarcia, R., Vidal, N., Arribas-Ibar, E., Piehl, F., Hietala, A., Bjarbo, L., Sengun, I., Meherremova, A., Ozcelik, P., Balkan, B., Tuga, C., Ugur, M., Erdem-Ozdamar, S., Bekircan-Kurt, C. E., Acar, N. P., Yilmaz, E., Caliskan, Y., Orsel, G., Efendi, H., Aydinlik, S., Cavus, H., Kutlu, A., Becerikli, G., Semiz, C., Tun, O., Terzi, M., Dogan, B., Onar, M. K., Sen, S., Kirbascavdar, T., Veske, A., Norwood, F., Dimitriou, A., Gollogly, J., Mahdi-Rogers, M., Seddigh, A., Sokratous, G., Maier, G., Sohail, F., Jacob, S., Sadalage, G., Torane, P., Brown, C., Shah, A., Sathasivam, S., Arndt, H., Davies, D., Watling, D., Amato, A., Cochrane, T., Salajegheh, M., Roe, K., Amato, K., Toska, S., Wolfe, G., Silvestri, N., Patrick, K., Zakalik, K., Katz, J., Miller, R., Engel, M., Forshew, D., Bravver, E., Brooks, B., Plevka, S., Burdette, M., Cunningham, S., Sanjak, M., Kramer, M., Nemeth, J., Schommer, C., Tierney, S., Juel, V., Guptill, J., Hobson-Webb, L., Massey, J., Beck, K., Carnes, D., Loor, J., Anderson, A., Pascuzzi, R., Bodkin, C., Kincaid, J., Snook, R., Guingrich, S., Micheels, A., Chaudhry, V., Corse, A., Mosmiller, B., Kelley, A., Ho, D., Srinivasan, J., Vytopil, M., Jara, J., Ventura, N., Scala, S., Carter, C., Donahue, C., Herbert, C., Weiner, E., Alam, S., Mckinnon, J., Haar, L., Mckinnon, N., Alcon, K., Mckenna, K., Sattar, N., Daniels, K., Jeffery, D., Kissel, J., Freimer, M., Hoyle, J. C., Agriesti, J., Chelnick, S., Mezache, L., Pineda, C., Muharrem, F., Karam, C., Khoury, J., Marburger, T., Kaur, H., Dimitrova, D., Gilchrist, J., Agrawal, B., Elsayed, M., Kohlrus, S., Andoin, A., Darnell, T., Golden, L., Lokaitis, B., Seelback, J., Muppidi, S., Goyal, N., Sakamuri, S., Y. T., So, Paulose, S., Pol, S., Welsh, L., Bhavaraju-Sanka, R., Tobongonzales, A., Dishman, L., Jones, F., Gonzalez, A., Padilla, P., Saklad, A., Silva, M., Nations, S., Trivedi, J., Hopkins, S., Kazamel, M., Alsharabati, M., Lu, L., Nozaki, K., Mumfrey-Thomas, S., Woodall, A., Mozaffar, T., Cash, T., Roy, G., Mathew, V., Maqsood, F., Minton, B., Jones, H. J., Rosenfeld, J., Garcia, R., Echevarria, L., Garcia, S., Pulley, M., Aranke, S., Berger, A. R., Shah, J., Shabbir, Y., Smith, L., Varghese, M., Gutmann, L., Jerath, N., Nance, C., Swenson, A., Olalde, H., Kressin, N., Sieren, J., Barohn, R., Dimachkie, M., Glenn, M., Mcvey, A., Pasnoor, M., Statland, J., Wang, J., Liu, T., Emmons, K., Jenci, N., Locheke, J., Fondaw, A., Johns, K., Rico, G., Walsh, M., Herbelin, L., Hafer-Macko, C., Kwan, J., Zilliox, L., Callison, K., Young, V., Disanzo, B., Naunton, K., Benatar, M., Bilsker, M., Sharma, K., Cooley, A., Reyes, E., Michon, S. -C., Sheldon, D., Steele, J., Chopra, M., Traub, R., Vu, T., Katzin, L., Mcclain, T., Harvey, B., Hart, A., Huynh, K., Beydoun, S., Chilingaryan, A., Doan, V., Droker, B., Gong, H., Karimi, S., Lin, F., Pokala, K., Tran, A., Akhter, S., Malekniazi, A., Tandan, R., Hehir, M., Waheed, W., Lucy, S., Weiss, M., Distad, J., Strom, S., Downing, S., Kim, B., Bertorini, T., Arnold, T., Hendersen, K., Pillai, R., Liu, Y., Wheeler, L., Hewlett, J., Vanderhook, M., Nowak, R., Dicapua, D., Keung, B., Kumar, A., Patwa, H., Robeson, K., Yang, I., Nye, J., and Vu, H.
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myasthenia gravis ,eculizumab - Published
- 2019
23. Flow and On-Water Synthesis and Cancer Cell Cytotoxicity of Caffeic Acid Phenethyl Amide (CAPA) Derivatives
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Anthony Saucedo, Muppidi Subbarao, Mauricio Jemal, Nakya L. Mesa-Diaz, Jadyn L. Smith, Alexandra Vernaza, Liqin Du, and Sean M. Kerwin
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anticancer ,natural product ,neuroblastoma ,Wittig reaction ,flow synthesis ,on-water synthesis ,Biology (General) ,QH301-705.5 ,Chemistry ,QD1-999 - Abstract
Caffeic acid phenethyl ester (CAPE) is a phenolic natural product with a wide range of biological activities, including anticancer activity; however, the ester group of CAPE is metabolically labile. The corresponding amide, CAPA, has improved metabolic stability but limited anticancer activity relative to CAPE. We report the synthesis using flow and on-water Wittig reaction approaches of five previously reported and five novel CAPA analogues. All of these analogues lack the reactive catechol functionality of CAPA and CAPE. Cytotoxicity studies of CAPE, CAPA, and these CAPA analogues in HeLa and BE(2)-C cells were carried out. Surprisingly, we found that CAPA is cytotoxic against the neuroblastoma BE(2)-C cell line (IC50 = 12 µM), in contrast to the weak activity of CAPA against HeLa cells (IC50 = 112 µM), and the literature reports of the absence of activity for CAPA against a variety of other cancer cell lines. One novel CAPA analogue, 3f, was identified as having cytotoxic activity similar to CAPE in HeLa cells (IC50 = 63 µM for 3f vs. 32 µM for CAPE), albeit with lower activity against BE(2)-C cells (IC50 = 91 µM) than CAPA. A different CAPA analogue, 3g, was found to have similar effects against BE(2)-C cells (IC50 = 92 µM). These results show that CAPA is uniquely active against neuroblastoma cells and that specific CAPA analogues that are predicted to be more metabolically stable than CAPE can reproduce CAPA’s activity against neuroblastoma cells and CAPE’s activity against HeLa cells.
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- 2024
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24. P.057 Achievement of minimal symptom expression in acetylcholine receptor antibody-positive participants treated with efgartigimod in ADAPT/ADAPT+
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Bril, V, Muppidi, S, Howard, JF, Murai, H, Phillips, G, Qi, C, Gelinas, D, Brauer, E, Zhao, S, and Vissing, J
- Abstract
Background: A key efficacy indicator in generalized myasthenia gravis (gMG) treatment is improvement in MG-ADL score. Minimal symptom expression (MSE, MG-ADL total score of 0 or 1) is explored as a novel proposed treatment target in gMG in the phase 3 study of intravenous efgartigimod, ADAPT, and its open-label extension, ADAPT+. Methods: Post hoc analyses of acetylcholine receptor antibody positive participants in ADAPT (n=129) and ADAPT+ (n=111) were performed. Results: In ADAPT, 44.6% receiving efgartigimod achieved MSE vs 10.9% of participants given placebo. Despite less frequent assessment during ADAPT+, 40.5% of participants achieved MSE. Eighty-one percent of participants treated with efgartigimod who achieved MSE in ADAPT also achieved MSE during ADAPT+; 23% who had not achieved MSE in ADAPT did in ADAPT+. Achieving MSE was associated with substantial improvements in QMG, MGC, MG-QoL15r, and EQ-5D-5L mean scores of 11.4, 16.0, 12.4, and 0.3 points, respectively, from baseline to best score (across all visits). These drastic improvements resulted in quality of life (QoL) comparable to that of healthy populations. MSE achievement also resulted in sustained improvements in these disease-specific and QoL measures. Conclusions: Participants who achieved MSE showed substantial and consistent improvements across multiple disease measures and experienced QoL comparable to that of healthy populations.
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- 2024
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25. A.01 Relieving the burden of myasthenia gravis: eculizumab reduces exacerbation, hospitalization and rescue therapy rates
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Siddiqi, Z, primary, Howard, JF, additional, Jacob, S, additional, Guptill, JT, additional, Meisel, A, additional, Wiendl, H, additional, Murai, H, additional, Utsugisawa, K, additional, Nowak, RJ, additional, Fujita, KP, additional, O’Brien, F, additional, Patra, K, additional, and Muppidi, S, additional
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- 2019
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26. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study
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Howard, J, Utsugisawa, K, Benatar, M, Murai, H, Barohn, R, Illa, I, Jacob, S, Vissing, J, Burns, T, Kissel, J, Muppidi, S, Nowak, R, O'Brien, F, Wang, J, Mantegazza, R, Bonanno, S, Howard, JF, Barohn, RJ, Burns, TM, Kissel, JT, Nowak, RJ, Wang, JJ, Howard, J, Utsugisawa, K, Benatar, M, Murai, H, Barohn, R, Illa, I, Jacob, S, Vissing, J, Burns, T, Kissel, J, Muppidi, S, Nowak, R, O'Brien, F, Wang, J, Mantegazza, R, Bonanno, S, Howard, JF, Barohn, RJ, Burns, TM, Kissel, JT, Nowak, RJ, and Wang, JJ
- Abstract
Background Complement is likely to have a role in refractory generalised myasthenia gravis, but no approved therapies specifically target this system. Results from a phase 2 study suggested that eculizumab, a terminal complement inhibitor, produced clinically meaningful improvements in patients with anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis. We further assessed the efficacy and safety of eculizumab in this patient population in a phase 3 trial. Methods We did a phase 3, randomised, double-blind, placebo-controlled, multicentre study (REGAIN) in 76 hospitals and specialised clinics in 17 countries across North America, Latin America, Europe, and Asia. Eligible patients were aged at least 18 years, with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of 6 or more, Myasthenia Gravis Foundation of America (MGFA) class II–IV disease, vaccination against Neisseria meningitides, and previous treatment with at least two immunosuppressive therapies or one immunosuppressive therapy and chronic intravenous immunoglobulin or plasma exchange for 12 months without symptom control. Patients with a history of thymoma or thymic neoplasms, thymectomy within 12 months before screening, or use of intravenous immunoglobulin or plasma exchange within 4 weeks before randomisation, or rituximab within 6 months before screening, were excluded. We randomly assigned participants (1:1) to either intravenous eculizumab or intravenous matched placebo for 26 weeks. Dosing for eculizumab was 900 mg on day 1 and at weeks 1, 2, and 3; 1200 mg at week 4; and 1200 mg given every second week thereafter as maintenance dosing. Randomisation was done centrally with an interactive voice or web-response system with patients stratified to one of four groups based on MGFA disease classification. Where possible, patients were maintained on existing myasthenia gravis therapies and rescue medication was allowed at the study physician's discretion. Patien
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- 2017
27. Randomized Trial of Thymectomy in Myasthenia Gravis
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Wolfe, Gi, Kaminski, Hj, Aban, Ib, Minisman, G, Kuo, Hc, Marx, A, Ströbel, P, Mazia, C, Oger, J, Cea, Jg, Heckmann, Jm, Evoli, A, Nix, W, Ciafaloni, E, Antonini, G, Witoonpanich, R, King, Jo, Beydoun, Sr, Chalk, Ch, Barboi, Ac, Amato, Aa, Shaibani, Ai, Katirji, B, Lecky, Br, Buckley, C, Vincent, A, Dias Tosta, E, Yoshikawa, H, Waddington Cruz, M, Pulley, Mt, Rivner, Mh, Kostera Pruszczyk, A, Pascuzzi, Rm, Jackson, Ce, Garcia Ramos GS, Verschuuren, Jj, Massey, Jm, Kissel, Jt, Werneck, Lc, Benatar, M, Barohn, Rj, Tandan, R, Mozaffar, T, Conwit, R, Odenkirchen, J, Sonett, Jr, 3rd, Jaretzki A., Newsom Davis, J, Cutter, Gr, MGTX study group including Cutter GR, Feese, M, Saluto, V, Rosenberg, M, Alvarez, V, Rey, L, King, J, Butzkueven, H, Goldblatt, J, Carey, J, Pollard, J, Reddel, S, Handel, N, Mccaughan, B, Pallot, L, Novis, R, Boasquevisque, C, Morato Fernandez, R, Ximenes, M, Werneck, L, Scola, R, Soltoski, P, Chalk, C, Moore, F, Mulder, D, Wadup, L, Mezei, M, Evans, K, Jiwa, T, Schaffar, A, White, C, Toth, C, Gelfand, G, Wood, S, Pringle, E, Zwicker, J, Maziak, D, Shamji, F, Sundaresan, S, Seely, A, Cea, G, Verdugo, R, Aguayo, A, Jander, S, Zickler, P, Klein, M, Weis, Ca, Melms, A, Bischof, F, Aebert, H, Ziemer, G, Thümler, B, Wilhem Schwenkmezger, T, Mayer, E, Schalke, B, Pöschel, P, Hieber, G, Wiebe, K, Clemenzi, A, Ceschin, V, Rendina, E, Venuta, F, Morino, S, Bucci, E, Durelli, Luca, Tavella, A, Clerico, Marinella, Contessa, G, Borasio, P, Servidei, S, Granone, P, Mantegazza, R, Berta, E, Novellino, L, Spinelli, L, Motomura, M, Matsuo, H, Nagayasu, T, Takamori, M, Oda, M, Matsumoto, I, Furukawa, Y, Noto, D, Motozaki, Y, Iwasa, K, Yanase, D, Ramos, Gg, Cacho, B, de la Garza, L, Lipowska, M, Kwiecinski, H, Potulska Chromik, A, Orlowski, T, Silva, A, Feijo, M, Freitas, A, Heckmann, J, Frost, A, Pan, El, Tucker, L, Rossouw, J, Drummond, F, Illa, I, Diaz, J, Leon, C, Yeh, Jh, Chiu, Hc, Hsieh, Ys, Tunlayadechanont, S, Attanavanich, S, Verschuuren, J, Straathof, C, Titulaer, M, Versteegh, M, Pels, A, Krum, Y, Leite, M, Hilton Jones, D, Ratnatunga, C, Farrugia, Me, Petty, R, Overell, J, Kirk, A, Gibson, A, Mcdermott, C, Hopkinson, D, Lecky, B, Watling, D, Marshall, D, Saminaden, S, Davies, D, Dougan, C, Sathasivam, S, Page, R, Sussman, J, Ealing, J, Krysiak, P, Amato, A, Salajegheh, M, Jaklitsch, M, Roe, K, Ashizawa, T, Smith, Rg, Zwischenberg, J, Stanton, P, Barboi, A, Jaradeh, S, Tisol, W, Gasparri, M, Haasler, G, Yellick, M, Dennis, C, Barohn, R, Pasnoor, M, Dimachkie, M, Mcvey, A, Gronseth, G, Dick, A, Kramer, J, Currence, M, Herbelin, L, Belsh, J, Li, G, Langenfeld, J, Mertz, Ma, Harrison, T, Force, S, Usher, S, Beydoun, S, Lin, F, Demeester, S, Akhter, S, Malekniazi, A, Avenido, G, Crum, B, Milone, M, Cassivi, S, Fisher, J, Heatwole, C, Watson, T, Hilbert, J, Smirnow, A, Distad, B, Weiss, M, Wood, D, Haug, J, Ernstoff, R, Cao, J, Chmielewski, G, Welsh, R, Duris, R, Gutmann, L, Pawar, G, Graeber, Gm, Altemus, P, Nance, C, Jackson, C, Grogan, P, Calhoon, J, Kittrell, P, Myers, D, Kaminski, H, Hayat, G, Naunheim, K, Eller, S, Holzemer, E, Alshekhlee, A, Robke, J, Karlinchak, B, Katz, J, Miller, R, Roan, R, Forshew, D, Kissel, J, Elsheikh, B, Ross, P, Chelnick, S, Lewis, R, Acsadi, A, Baciewicz, F, Masse, S, Massey, J, Juel, V, Onaitis, M, Lowe, J, Lipscomb, B, Thai, G, Milliken, J, Martin, V, Karayan, R, Muley, S, Parry, G, Shumway, S, Oh, S, Claussen, G, Lu, L, Cerfolio, R, Young, A, Morgan, M, Pascuzzi, R, Kincaid, J, Kesler, K, Guingrich, S, Michaels, A, Phillips, L, Burns, T, Jones, D, Fischer, C, Pulley, M, Berger, A, D'Agostino, H, Smith, L, Rivner, M, Pruitt, J, Landolfo, K, Hillman, D, Shaibani, A, Sermas, A, Ruel, R, Ismail, F, Sivak, M, Goldstein, M, Camunas, J, Bratton, J, Panitch, H, Leavitt, B, Jones, M, Wolfe, G, Muppidi, S, Vernino, S, Nations, S, Meyer, D, and Gorham, N.
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Male ,medicine ,medicine.medical_treatment ,030204 cardiovascular system & hematology ,Medical and Health Sciences ,Severity of Illness Index ,law.invention ,0302 clinical medicine ,Randomized controlled trial ,law ,Prednisone ,Adolescent ,Adult ,Aged ,Combined Modality Therapy ,Female ,Glucocorticoids ,Hospitalization ,Humans ,Middle Aged ,Myasthenia Gravis ,Single-Blind Method ,Treatment Outcome ,Young Adult ,Thymectomy ,Medicine (all) ,Young adult ,MGTX Study Group ,General Medicine ,Settore MED/26 - NEUROLOGIA ,6.1 Pharmaceuticals ,medicine.drug ,medicine.medical_specialty ,Clinical Trials and Supportive Activities ,Autoimmune Disease ,03 medical and health sciences ,Rare Diseases ,Clinical Research ,General & Internal Medicine ,Internal medicine ,Severity of illness ,business.industry ,Neurosciences ,Evaluation of treatments and therapeutic interventions ,Retrospective cohort study ,medicine.disease ,Myasthenia gravis ,Surgery ,Clinical research ,adolescent ,adult ,aged ,combined modality therapy ,female ,glucocorticoids ,hospitalization ,humans ,male ,middle aged ,myasthenia gravis ,prednisone ,severity of Illness index ,single-blind method ,treatment outcome ,young adult ,thymectomy ,business ,030217 neurology & neurosurgery - Abstract
BackgroundThymectomy has been a mainstay in the treatment of myasthenia gravis, but there is no conclusive evidence of its benefit. We conducted a multicenter, randomized trial comparing thymectomy plus prednisone with prednisone alone.MethodsWe compared extended transsternal thymectomy plus alternate-day prednisone with alternate-day prednisone alone. Patients 18 to 65 years of age who had generalized nonthymomatous myasthenia gravis with a disease duration of less than 5 years were included if they had Myasthenia Gravis Foundation of America clinical class II to IV disease (on a scale from I to V, with higher classes indicating more severe disease) and elevated circulating concentrations of acetylcholine-receptor antibody. The primary outcomes were the time-weighted average Quantitative Myasthenia Gravis score (on a scale from 0 to 39, with higher scores indicating more severe disease) over a 3-year period, as assessed by means of blinded rating, and the time-weighted average required dose of prednisone over a 3-year period.ResultsA total of 126 patients underwent randomization between 2006 and 2012 at 36 sites. Patients who underwent thymectomy had a lower time-weighted average Quantitative Myasthenia Gravis score over a 3-year period than those who received prednisone alone (6.15 vs. 8.99, P
- Published
- 2016
28. A Genome-wide Association Study of Myasthenia Gravis
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Renton, Ae, Pliner, Ha, Provenzano, Carlo, Evoli, Amelia, Ricciardi, R, Nalls, Ma, Marangi, Giuseppe, Abramzon, Y, Arepalli, S, Chong, S, Hernandez, Dg, Johnson, Jo, Bartoccioni, Emanuela, Scuderi, Flavia, Maestri, M, Gibbs, Jr, Errichiello, E, Chiò, A, Restagno, G, Sabatelli, Mario, Macek, M, Scholz, Sw, Corse, A, Chaudhry, V, Benatar, M, Barohn, Rj, Mcvey, A, Pasnoor, M, Dimachkie, Mm, Rowin, J, Kissel, J, Freimer, M, Kaminski, Hj, Sanders, Db, Lipscomb, B, Massey, Jm, Chopra, M, Howard, Jf, Koopman, Wj, Nicolle, Mw, Pascuzzi, Rm, Pestronk, A, Wulf, C, Florence, J, Blackmore, D, Soloway, A, Siddiqi, Z, Muppidi, S, Wolfe, G, Richman, D, Mezei, Mm, Jiwa, T, Oger, J, Drachman, Db, Traynor, Bj, Provenzano, Carlo (ORCID:0000-0001-5476-5517), Evoli, Amelia (ORCID:0000-0003-0282-8787), Marangi, Giuseppe (ORCID:0000-0002-6898-8882), Bartoccioni, Emanuela (ORCID:0000-0002-4434-8661), Sabatelli, Mario (ORCID:0000-0001-6635-4985), Renton, Ae, Pliner, Ha, Provenzano, Carlo, Evoli, Amelia, Ricciardi, R, Nalls, Ma, Marangi, Giuseppe, Abramzon, Y, Arepalli, S, Chong, S, Hernandez, Dg, Johnson, Jo, Bartoccioni, Emanuela, Scuderi, Flavia, Maestri, M, Gibbs, Jr, Errichiello, E, Chiò, A, Restagno, G, Sabatelli, Mario, Macek, M, Scholz, Sw, Corse, A, Chaudhry, V, Benatar, M, Barohn, Rj, Mcvey, A, Pasnoor, M, Dimachkie, Mm, Rowin, J, Kissel, J, Freimer, M, Kaminski, Hj, Sanders, Db, Lipscomb, B, Massey, Jm, Chopra, M, Howard, Jf, Koopman, Wj, Nicolle, Mw, Pascuzzi, Rm, Pestronk, A, Wulf, C, Florence, J, Blackmore, D, Soloway, A, Siddiqi, Z, Muppidi, S, Wolfe, G, Richman, D, Mezei, Mm, Jiwa, T, Oger, J, Drachman, Db, Traynor, Bj, Provenzano, Carlo (ORCID:0000-0001-5476-5517), Evoli, Amelia (ORCID:0000-0003-0282-8787), Marangi, Giuseppe (ORCID:0000-0002-6898-8882), Bartoccioni, Emanuela (ORCID:0000-0002-4434-8661), and Sabatelli, Mario (ORCID:0000-0001-6635-4985)
- Abstract
Myasthenia gravis is a chronic, autoimmune, neuromuscular disease characterized by fluctuating weakness of voluntary muscle groups. Although genetic factors are known to play a role in this neuroimmunological condition, the genetic etiology underlying myasthenia gravis is not well understood.
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- 2015
29. P.034 Minimal symptom expression following treatment with efgartigimod in patients with Generalized Myasthenia Gravis
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Siddiqi, Z, Howard, JF, Bril, V, Vu, T, Karam, C, Pasnoor, M, Muppidi, S, Peric, S, Murai, H, Ulrichts, P, T’joen, C, Utsugisawa, K, Verschuuren, J, and Mantegazza, R
- Abstract
Background: Efgartigimod is a human IgG1 antibody Fc-fragment that reduces IgG levels through FcRn blockade. A key efficacy indicator in the treatment of IgG autoantibody-mediated generalized myasthenia gravis (gMG) is improvement in MG-ADL score. Methods: The ADAPT phase 3 trial evaluated safety and efficacy of efgartigimod in patients with gMG, including reaching and maintaining of minimal symptom expression (MSE; defined as an MG-ADL total score of 0 or 1). Results: 167 patients (AChR-Ab+, n=129; AChR-Ab-, n=38) were randomized to receive treatment cycles of 4 weekly infusions of efgartigimod or placebo. Significantly more AChR-Ab+ efgartigimod-treated patients achieved MSE during cycle 1 compared to placebo-treated patients (40.0% [n=26/65] vs 11.1% [n=7/63; P<0.0001]). In cycle 2, 31.4% (n=16/51) of AChR-Ab+ patients in the efgartigimod cohort achieved MSE compared to none in the placebo cohort. MG-ADL score improved by ≥6 points in 56.9% of AChR-Ab+ efgartigimod-treated patients compared to 20.6% of placebo-treated patients in cycle 1. Most patients achieved MSE by week 4 of a cycle, paralleling early reduction in IgG levels, and MSE duration ranged from 1 to ≥10 weeks. Adverse events were predominantly mild to moderate. Conclusions: Efgartigimod treatment resulted in more patients with AChR-Ab+ gMG achieving both MSE and clinically meaningful MG-ADL improvements.
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- 2023
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30. Consolidation and maintenance therapy with lenalidomide, bortezomib and dexamethasone (RVD) in high-risk myeloma patients
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Nooka, A K, primary, Kaufman, J L, additional, Muppidi, S, additional, Langston, A, additional, Heffner, L T, additional, Gleason, C, additional, Casbourne, D, additional, Saxe, D, additional, Boise, L H, additional, and Lonial, S, additional
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- 2013
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31. Favorable impact of pre-transplant ATG on outcomes of reduced-intensity hematopoietic cell transplants from partially mismatched unrelated donors
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Langston, A A, primary, Prichard, J M, additional, Muppidi, S, additional, Nooka, A, additional, Lechowicz, M J, additional, Lonial, S, additional, Sinha, R, additional, Graiser, M, additional, Kaufman, J L, additional, Khoury, H J, additional, Flowers, C R, additional, and Waller, E K, additional
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- 2013
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32. Inpatient EMG Studies. Do They Help with Patient Care? (P01.197)
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Samuel, G., primary, Muppidi, S., additional, and Wolfe, G., additional
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- 2012
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33. Clinical and Electrophysiologic Characterization of Chronic and Critical Ischemic Monomelic Neuropathy after Revascularization: A Case Series (P07.151)
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Absalom, N., primary, Wolfe, G., additional, Nations, S., additional, Trivedi, J., additional, and Muppidi, S., additional
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- 2012
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34. Long-Term Outcomes in Autoimmune Autonomic Ganglionopathy (P05.210)
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Boydston, E., primary, Muppidi, S., additional, and Vernino, S., additional
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- 2012
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35. Reversible right vagal neuropathy
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Muppidi, S., primary, Gupta, P. K., additional, and Vernino, S., additional
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- 2011
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36. Multiple intracerebral hemorrhages after cervical epidural injections
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Mendelson, J., primary, Muppidi, S., additional, and Silberstein, S., additional
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- 2008
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37. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study
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Kazumi Takada, Vladislav Abramov, Seiko Yoshida, Pinar Ozcelik, Carolina Miranda, Jennifer Kane, Kaitlyn McKenna, Natasha Campbell, Sharon P. Nations, Shitiz Kumar Sriwastava, Yuko Fujii, Mayumi Murata, Linda Wagemaekers, Angela Andoin, Mollie Vanderhook, Yoshinori Okubo, Martin Bilsker, Taira Uehara, Vera Bril, Julia Wanschitz, Stanislava Toncrova, Mariela Bettini, Kazumi Futono, Shachie Aranke, Yool-hee Kim, Hiroyuki Murai, Anne Nyrhinen, Vinay Chaudhry, Raffaele Iorio, Takashi Kanda, Brittany Harvey, Francisco Javier Rodriguez de Rivera, Henning Andersen, Marianne de Visser, Miwako Sato, Yasuhiro Maeda, Fabienne Deruelle, Marina Pozo, Adam Hart, Masaki Saitoh, Wladimir Bocca Vieira de Rezende Pinto, Said R. Beydoun, Lindsay Zilliox, Akihiro Mukaino, Cinzia Caserta, Mahi Jasinarachchi, Andrea M. Corse, Nikoletta Papadopoulou, JuYoung Kwon, Fernanda Carrara, Juliet Saba, Masayuki Makamori, Vittorio Frasca, Luciana Souza Duca, Hoo Nam Kang, C. Trebst, Celile Phan, Muzeyyen Ugur, Eduardo Ng, Jonathan McKinnon, Hila Bali Kuperman, David Feder, Judit Matolcsi, Jiri Pitha, Martin Stangel, Kate Beck, Gabriel Paiva, Diego Lopergolo, Katrien De Mey, Hidenori Matsuo, Lucas Eduardo Pazetto, Eugene Lai, Amanda Anderson, Ann D'Hondt, Tetsuya Akiyama, Beverly Fyfe, Bella Gross, Elisabet Arribas-Ibar, Kathy de Koning, Gulmohor Roy, Dmitry Pokhabov, Maria Johanna Keijzers, Nicholas Ventura, Tessa Marburger, John Loor, Ji Eun Lee, Alessandro Filla, Celal Tuga, Stephanie Scala, Rudy Mercelis, Marc H. De Baets, Hisako Kobayashi, Stanislav Vohanka, Ana Paula Macagnan, Ana Carolina Amaral de Andrade, Heike Arndt, Giovanni Antonini, Yumi Yamashita, Gwendal Le Masson, Sonia Garcia, Sarah Verjans, James F. Howard, Zaeem A. Siddiqi, Yuen T. So, Megumi Koga, Exuperio Diez Tejedor, Teresa Costabile, Mihoko Takada Takada, Steve Hopkins, Jonathan S. Katz, Charlene Hafer-Macko, Erica Nogueira Coelho, Hung Youl Seok, Carol Herbert, Yuriko Nagane, Didem Altiparmak, Sachiko Kamakura, Mohammad Sanjak, Caroline Moreau, Jordi Díaz-Manera, Sivakumar Sathasivam, Michael Vytopil, Amelia Evoli, Masakatsu Motomura, Ester Reggio, Guy Van den Abeele, Hélène Zéphir, Asya Yarmoschuk, Jasmine Hewlett, Amy Wilson, Sachie Fukui, Cavit Boz, Iandra Souza, Morgane Gaboreau, Ivana Jurajdova, Sonia Decressac, Yong Seo Koo, Valentina Pegoraro, Seung Min Kim, Benison Keung, Rosana Rocha, Nanna Witting, John Vissing, Elaine Weiner, Ali Malekniazi, Larisa Babenko, Amanda C. Guidon, Gal Maier, Charlotte Smetcoren, Robert M. Pascuzzi, Domenico Marco Bonifati, Yumiko Nakamura, Tamires Cristina Gomes da Silva, Takashi Murahara, Sarah Plevka, Tomoko Tsuda, John C. Kincaid, Arnaud Lacour, Ibrez Bandukwala, Alan R. Berger, Chang Nyoung Lee, Jae-Sung Lim, Vern C. Juel, Tulio E. Bertorini, Valeria Cavalcante Lino, Namie Taichi, Ju-Hong Min, Josep Gamez, Nelly Greenbereg, William S. David, Srikanth Muppidi, Husnu Efendi, Pedro Lopez Ruiz, Baki Dogan, Cansu Semiz, Natalia Julia Palacios, Sharon Downing, Paola Cudia, Daniel Jacobs, Can Ebru Bekircan-Kurt, Takayasu Fukudome, Kristen Roe, Lena Bjarbo, Nicole Kassebaum, Makoto Samukawa, Shizuka Asada, Christina Dheel, Fatima Maqsood, Eun Bi Hwang, Kevin Daniels, Sevim Erdem-Ozdamar, Olivier Stevens, Claudio Mazia, Karan Alcon, Sibel Gazioglu, Keiko Kikutake, Luis Lay, Petra Tilkin, Corrado Angelini, Derrick Blackmore, Kimiaki Utsugisawa, Despoina Charalambous, Tuula Harrison, Kristin Huynh, Huned S. Patwa, Laura Echevarria, Henrique Mohr, Christian Homedes-Pedret, Richard J. Barohn, Byung Jo Kim, Daniel DiCapua, Terry McClain, Debora Dada Martineli Torres, Maria Salvado Figueras, Ana Paula Melo, Riley Snook, Miki Ogawa, Marcelo Annes, Yuka Saito, Isabel Illa, Evanthia Bernitsas, Nicole Smalley, Molly Lindsay, Robert G. Miller, Olga Azrilin, Silvia Bonanno, Evgeniya Kosykh, Marcela Wolfova, Olivier Outteryck, Shirli Toska, Anna Kostera-Pruszczyk, HyeJin Ra, Rup Tandan, Sotirios Papagiannopoulos, Natasha Willlems, Anne Mette Ostergaard Autzen, Meinoshin Okumura, Patrick Vermersch, Sarada Sakamuri, Maria Antonia Alberti Aguilo, Shigemi Shimose, Cynthia Carter, Ira Blount, Lisa Thompson, Maurer Pereira Martins, Richard Nowak, Hyung Seok Lee, Anna Kaminska, Joan Bratton, Nazire Pinar Acar, Junichi Ogasawara, Mohamed Mahdi-Rogers, Teiichiro Mitazaki, Marek Čierny, Craig Donahue, Jaya Trivedi, Neelam Goyal, Gonzalo Vidal, Brandy Quarles, Akiko Kanzaki, Yasuko Ikeda, Tomomi Kobashikawa, Morris Brown, Daisuke Yamamoto, Michel Deneve, Denis Korobko, Beth DiSanzo, Benedikt Schoser, Heidi Boterhoven, Eri Kobayashi, Maoko Shirane, Cristiani Fernanda Butinhao, Eriko Higuchi, Takashi Hayashi, Masanori Takahashi, Anne-Cécile Wielanek-Bachelet, Benjamin Rix Brooks, Emanuela Onesti, Tahseen Mozaffar, Liang Lu, Sevasti Bostantzopoulou, Christophe Vial, Shawn J. Bird, Sandi Mumfrey-Thomas, Julie Khoury, Kara Patrick, Kenichi Tsukita, Yoshiko Sano, Hiroshi Nakazora, David P. Richman, Gavin Brown, Yoon-Ho Hong, Tomohiro Kawamura, Igor Dias Brockhausen, Ye Liu, Acary Souza Bulle Oliveira, Soichiro Funaka, Tomoya Hasuike, Frank Lin, Luis Antonio Querol Gutierrez, Namita Goyal, Elena Pinzan, Michelle Mellion, Silvia Messina, Christopher Lindberg, Csilla Rozsa, J. Chad Hoyle, Yoko Kaneko, Gustavo Duran, Francesco Patti, Arshira Seddigh, Ele Kim Perez, Jayashri Srinivasan, Michael Benatar, Philip Van Damme, Salma Akhter, Daniel Ambrosio, Maria Salvado, Floyd Jones, Mark Sivak, Anneke J. van der Kooi, Karen Callison, Catherine Nigro, Rebekah Garcia, Thomas Arnold, Hideki Arima, Brigid Crabtree, Mary Varghese, Aditya Kumar, Miri Kim, Fanny O'Brien, Naya McKinnon, Lauren Wheeler, Hong Vu, Shunsuke Yoshimura, Masatoshi Omoto, Jeffrey T. Guptill, Maria Gabriele, Francoise Bouhour, Veena Mathew, Ritsu Nakayama, Rosa Hasan, Francesco Saccà, Mohammed Salajegheh, Diana Dimitrova, Alzira Alves de Siqueira Carvalho, Maurizio Inghilleri, George Sachs, Rekha Pillai, Enrico Marano, Monika Konyane, Anh Tran, Seda Aydinlik, Kendrick Henderson, Fumie Meguro, Alexandre Guerreiro, Amaiak Chilingaryan, Tiyonnoh Cash, Jun Kawamata, Julie Steele, Helene Gervais-Bernard, Thomas Harbo, Alejandra Dalila Garcia, Musa Kazim Onar, Sabrina Sacconi, Carlos Casasnovas Pons, Nadezhda Malkova, Denis Sazonov, Mireya Fernandez-Fournier, Karin Fricke, Laurie Gutmann, Amy Saklad, Clara Schommer, Sandra Taber, Fiona Norwood, Tugce Kirbas Cavdar, Monique Miesen, Fernanda Troili, Masanori Watanabe, Ratna Bhavaraju-Sanka, Ted M. Burns, Sari Atula, Faisal Sohail, Barbora Kurkova, Brigitta Szabadosne, Luciana Renata Cubas Volpe, Jane Pedersen, Jing Jing Wang, Masashi Inoue, Antonella Di Pasquale, Megan Kramer, Magda Chmelikova, Mehran Soltani, Tuan Vu, Laura Fionda, Eliz Agopian, Susan Shin, Anthony A. Amato, Lotte Vinge, Hakan Cavus, Gil I. Wolfe, Joan Nye, Delphine Mahieu, Miguel Wilken, Markus Färkkilä, Catherine Faber, Erin Manning, Emiko Tsuda, Rami Massie, Paolo Emilio Alboini, Yasmeen Shabbir, Angela Campanella, Aikaterini Dimitriou, Marcelo Rugiero, Cynthia Bodkin, Gyorgyi Szabo, Sharon Halton, Akshay Shah, Yasuko Maeda, Hans D. Katzberg, Yagmur Caliskan, Jaimin Shah, Katsuhisa Masaki, Valentina Damato, Blanka Andersson, Aline de Cassia Santos, Masahiro Mori, Renato Mantegazza, Misa Shimpo, Joanne Nemeth, Livia Dezsi, Anna De Rosa, Doreen Ho, Julie Moutarde, Efstathia Mitropoulou, Amy Woodall, Angela Micheels, László Vécsei, Byoung Joon Kim, Lisa Smith, Tomihiro Imai, Harpreet Kaur, Lorenzo Maggi, Jane Distad, Anita Mogensen, Ericka Simpson, Anne Cooley, Eliana Reyes, Ha Young Shin, Da Yoon Koh, Stefan Gingele, Susan Strom, Ezgi Yilmaz, Manisha Chopra, Anna Melnikova, Edouard Millois, Ludwig Gutmann, Miriam Freimer, Hirokazu Shinozaki, Heena Olalde, Kerry Naunton, Shunya Nakane, Ihsan Sengun, Dimos-Dimitrios Mitsikostas, Edina Varga, Juha-Pekka Erälinna, Wolfgang Löscher, Jan De Bleecker, Elena Bravver, Ana Lazaro, Eun Bin Cho, Thomas Cochrane, Jonathan Goldstein, Lisa D. Hobson-Webb, Michaela Tyblova, Angela Marsil, J. Edward Hartmann, Miyuki Morikawa, Karen Zakalik, Claude Desnuelle, Iveta Novakova, Michiaki Koga, Melinda Horvath, Luiz Otavio Maia Gonçalves, Elena Cortes Vicente, Alejandro Tobon Gonzalez, Stanley H. Appel, Brian Minton, Daniele Orrico, Brian Droker, Jacob Kaufman, Erica Coelho, Chafic Karam, Mikko Laaksonen, Katherine Amato, Jinmyoung Seok, Natalia Prando, Pauline Lahaut, Kaori Osakada, Phillipa Lamont, Alexandros Tselis, Daiane da Cruz Pacheco, Joan Højgaard, Hirokazu Shiraishi, Josef Bednarik, Stefania Morino, Mark Levine-Weinberg, Sara-Claude Michon, Yusuke Fukuda, Michael Pulley, Koichi Narikawa, Ricardo Rojas Garcia, Betsy Mosmiller, James Gilchrist, Maria da Penha Morita Ananias, Maryanne Burdette, Shingo Konno, Janelle Butters, Stephan Wenninger, Debbie Davies, Thomas Skripuletz, Mohammad Alsharabati, Katarina Reguliova, Gabor Lovas, Yuichiro Gondo, Miju Shin, HyeLim Lee, Bruno Bezerra Rosa, Michael D. Weiss, Martha Zampaki, Andrea Caramma, Jeffrey V. Rosenfeld, Cigdem Ozen Aydin, Shara Holzberg, Hélène Merle, Olga Zapletalova, Kurt-Wolfram Suehs, Robert P. Lisak, Dale J. Lange, Albert Hietala, Sedat Sen, Elena Giacomelli, Akiyuki Uzawa, Tomás Augusto Suriane Fialho, Matteo Garibaldi, Nadia Sattar, Wai-Kuen Leong, Lindsay Kaplan, Tetsuya Kanai, Jaana Eriksson, Akiko Nagaishi, Khema Sharma, Tamar Gibson, Mohamed Kazamel, Yulia Nesterova, Sascha Alvermann, Murat Terzi, Taylor Darnell, Donna Carnes, Victor Balyazin, John T. Kissel, Waqar Waheed, Jana Junkerova, Kimberly Robeson, Nicholas Vlaikidis, Nicholas Silvestri, Fredrik Piehl, Maurício André Gheller Friedrich, Shun Shimohama, Nuria Vidal, Eleni Kasioti, H. James Jones, Michael K. Hehir, Luiz Augusto da Silva, Dave Watling, Leslie Roberts, Casey Faigle, Caroline Hourquin, Olli Oksaranta, Tomomi Imamura, Shin Hisahara, Dennis Jeffery, Marie-Hélène Soriani, M. Kawai, Chieko Yoshikawa, Roseann Keo, Angela Genge, Michelangelo Maestri Tassoni, Milvia Pleitez, Michael H. Rivner, Maki Jingu, Giorgia Puorro, Andrea Swenson, Saiju Jacob, Carolina Ortea, Shuichiro Suzuki, Marguerite Engel, Ikuko Kamegamori, SangAe Park, Guilhem Sole, Lesly Welsh, Nichole Gallatti, Jakit Gollogly, Daniel Jons, Yasuteru Sano, Takuya Matsushita, Omar Khan, Maria Cristina Gori, Thabata Veiga, Julie Agriesti, Jos Maessen, Sandra Guinrich, Francesca Bevilacqua, Laura Haar, Jordana Gonçalves Geraldo, Justin Y. Kwan, Hidekazu Suzuki, Dai Matsuse, Kelly Jia, Ozlem Tun, Lara Katzin, Yasushi Suzuki, Shannon Lucy, Carlo Antozzi, ANS - Neuroinfection & -inflammation, Neurology, Howard, James F, Utsugisawa, Kimiaki, Benatar, Michael, Murai, Hiroyuki, Barohn, Richard J, Illa, Isabel, Jacob, Saiju, Vissing, John, Burns, Ted M, Kissel, John T, Muppidi, Srikanth, Nowak, Richard J, O'Brien, Fanny, Wang, Jing-Jing, Mantegazza, Renato, Mazia, Claudio Gabriel, Wilken, Miguel, Ortea, Carolina, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Vidal, Gonzalo, Garcia, Alejandra Dalila, Lamont, Phillipa, Leong, Wai-Kuen, Boterhoven, Heidi, Fyfe, Beverly, Roberts, Leslie, Jasinarachchi, Mahi, Willlems, Natasha, Wanschitz, Julia, Löscher, Wolfgang, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Wagemaekers, Linda, Mahieu, Delphine, Van Damme, Philip, Smetcoren, Charlotte, Stevens, Olivier, Verjans, Sarah, D'Hondt, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Hasan, Rosa, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, Melo, Ana Paula, Rocha, Rosana, Rosa, Bruno, Veiga, Thabata, Augusto da Silva, Luiz, Gonçalves Geraldo, Jordana, da Penha Morita Ananias, Maria, Nogueira Coelho, Erica, Paiva, Gabriel, Pozo, Marina, Prando, Natalia, Dada Martineli Torres, Debora, Fernanda Butinhao, Cristiani, Coelho, Erica, Renata Cubas Volpe, Luciana, Duran, Gustavo, Gomes da Silva, Tamires Cristina, Otavio Maia Gonçalves, Luiz, Pazetto, Lucas Eduardo, Souza Duca, Luciana, Suriane Fialho, Tomás Augusto, Gheller Friedrich, Maurício André, Guerreiro, Alexandre, Mohr, Henrique, Pereira Martins, Maurer, da Cruz Pacheco, Daiane, Macagnan, Ana Paula, de Cassia Santos, Aline, Bulle Oliveira, Acary Souza, Amaral de Andrade, Ana Carolina, Annes, Marcelo, Cavalcante Lino, Valeria, Pinto, Wladimir, Miranda, Carolina, Carrara, Fernanda, Souza, Iandra, Genge, Angela, Massie, Rami, Campbell, Natasha, Bril, Vera, Katzberg, Han, Soltani, Mehran, Ng, Eduardo, Siddiqi, Zaeem, Phan, Celile, Blackmore, Derrick, Vohanka, Stanislav, Bednarik, Josef, Chmelikova, Magda, Cierny, Marek, Toncrova, Stanislava, Junkerova, Jana, Kurkova, Barbora, Reguliova, Katarina, Zapletalova, Olga, Pitha, Jiri, Novakova, Iveta, Tyblova, Michaela, Wolfova, Marcela, Jurajdova, Ivana, Andersen, Henning, Harbo, Thoma, Vinge, Lotte, Mogensen, Anita, Højgaard, Joan, Witting, Nanna, Autzen, Anne Mette, Pedersen, Jane, Färkkilä, Marku, Atula, Sari, Nyrhinen, Anne, Erälinna, Juha-Pekka, Laaksonen, Mikko, Oksaranta, Olli, Eriksson, Jaana, Harrison, Tuula, Desnuelle, Claude, Sacconi, Sabrina, Soriani, Marie-Hélène, Decressac, Sonia, Moutarde, Julie, Lahaut, Pauline, Solé, Guilhem, Le Masson, Gwendal, Wielanek-Bachelet, Anne-Cécile, Gaboreau, Morgane, Moreau, Caroline, Wilson, Amy, Vial, Christophe, Bouhour, Françoise, Gervais-Bernard, Helene, Merle, Hélène, Hourquin, Caroline, Lacour, Arnaud, Outteryck, Olivier, Vermersch, Patrick, Zephir, Hélène, Millois, Edouard, Deneve, Michel, Deruelle, Fabienne, Schoser, Benedikt, Wenninger, Stephan, Stangel, Martin, Alvermann, Sascha, Gingele, Stefan, Skripuletz, Thoma, Suehs, Kurt-Wolfram, Trebst, Corinna, Fricke, Karin, Papagiannopoulos, Sotirio, Bostantzopoulou, Sevasti, Vlaikidis, Nichola, Zampaki, Martha, Papadopoulou, Nikoletta, Mitsikostas, Dimos-Dimitrio, Kasioti, Eleni, Mitropoulou, Efstathia, Charalambous, Despoina, Rozsa, Csilla, Horvath, Melinda, Lovas, Gabor, Matolcsi, Judit, Szabo, Gyorgyi, Szabadosne, Brigitta, Vecsei, Laszlo, Dezsi, Livia, Varga, Edina, Konyane, Monika, Gross, Bella, Azrilin, Olga, Greenbereg, Nelly, Bali Kuperman, Hila, Antonini, Giovanni, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Di Pasquale, Antonella, Filla, Alessandro, Costabile, Teresa, Marano, Enrico, Sacca, Francesco, Marsili, Angela, Puorro, Giorgia, Maestri Tassoni, Michelangelo, De Rosa, Anna, Bonanno, Silvia, Antozzi, Carlo, Maggi, Lorenzo, Campanella, Angela, Angelini, Corrado, Cudia, Paola, Pegoraro, Valentina, Pinzan, Elena, Bevilacqua, Francesca, Orrico, Daniele, Bonifati, Domenico Marco, Evoli, Amelia, Alboini, Paolo Emilio, D'Amato, Valentina, Iorio, Raffaele, Inghilleri, Maurizio, Fionda, Laura, Frasca, Vittorio, Giacomelli, Elena, Gori, Maria, Lopergolo, Diego, Onesti, Emanuela, Gabriele, Maria, Patti, Francesco, Salvatore Caramma, Andrea, Messina, Silvia, Reggio, Ester, Caserta, Cinzia, Uzawa, Akiyuki, Kanai, Tetsuya, Mori, Masahiro, Kaneko, Yoko, Kanzaki, Akiko, Kobayashi, Eri, Masaki, Katsuhisa, Matsuse, Dai, Matsushita, Takuya, Uehara, Taira, Shimpo, Misa, Jingu, Maki, Kikutake, Keiko, Nakamura, Yumiko, Sano, Yoshiko, Nagane, Yuriko, Kamegamori, Ikuko, Fujii, Yuko, Futono, Kazumi, Tsuda, Tomoko, Saito, Yuka, Suzuki, Hidekazu, Morikawa, Miyuki, Samukawa, Makoto, Kamakura, Sachiko, Shiraishi, Hirokazu, Mitazaki, Teiichiro, Motomura, Masakatsu, Mukaino, Akihiro, Yoshimura, Shunsuke, Asada, Shizuka, Kobashikawa, Tomomi, Koga, Megumi, Maeda, Yasuko, Takada, Kazumi, Takada, Mihoko Takada, Yamashita, Yumi, Yoshida, Seiko, Suzuki, Yasushi, Akiyama, Tetsuya, Narikawa, Koichi, Tsukita, Kenichi, Meguro, Fumie, Fukuda, Yusuke, Sato, Miwako, Matsuo, Hidenori, Fukudome, Takayasu, Gondo, Yuichiro, Maeda, Yasuhiro, Nagaishi, Akiko, Nakane, Shunya, Okubo, Yoshinori, Okumura, Meinoshin, Funaka, Soichiro, Kawamura, Tomohiro, Makamori, Masayuki, Takahashi, Masanori, Hasuike, Tomoya, Higuchi, Eriko, Kobayashi, Hisako, Osakada, Kaori, Taichi, Namie, Tsuda, Emiko, Hayashi, Takashi, Hisahara, Shin, Imai, Tomihiro, Kawamata, Jun, Murahara, Takashi, Saitoh, Masaki, Shimohama, Shun, Suzuki, Shuichiro, Yamamoto, Daisuke, Konno, Shingo, Imamura, Tomomi, Inoue, Masashi, Murata, Mayumi, Nakazora, Hiroshi, Nakayama, Ritsu, Ikeda, Yasuko, Ogawa, Miki, Shirane, Maoko, Kanda, Takashi, Kawai, Motoharu, Koga, Michiaki, Ogasawara, Junichi, Omoto, Masatoshi, Sano, Yasuteru, Arima, Hideki, Fukui, Sachie, Shimose, Shigemi, Shinozaki, Hirokazu, Watanabe, Masanori, Yoshikawa, Chieko, van der Kooi, Anneke, de Visser, Marianne, Gibson, Tamar, Maessen, Jo, de Baets, Marc, Faber, Catherine, Keijzers, Maria Johanna, Miesen, Monique, Kostera-Pruszczyk, Anna, Kaminska, Anna, Kim, Byung-Jo, Lee, Chang Nyoung, Koo, Yong Seo, Seok, Hung Youl, Kang, Hoo Nam, Ra, Hyejin, Kim, Byoung Joon, Cho, Eun Bin, Lee, Hyelim, Min, Ju-Hong, Seok, Jinmyoung, Koh, Da Yoon, Kwon, Juyoung, Lee, Jieun, Park, Sangae, Hong, Yoon-Ho, Lim, Jae-Sung, Kim, Miri, Kim, Seung Min, Kim, Yool-hee, Lee, Hyung Seok, Shin, Ha Young, Hwang, Eun Bi, Shin, Miju, Sazonov, Deni, Yarmoschuk, Asya, Babenko, Larisa, Malkova, Nadezhda, Melnikova, Anna, Korobko, Deni, Kosykh, Evgeniya, Pokhabov, Dmitry, Nesterova, Yulia, Abramov, Vladislav, Balyazin, Victor, Casasnovas Pons, Carlo, Alberti Aguilo, Maria, Homedes-Pedret, Christian, Palacios, Natalia Julia, Lazaro, Ana, Diez Tejedor, Exuperio, Fernandez-Fournier, Mireya, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Salvado Figueras, Maria, Gamez, Josep, Salvado, Maria, Cortes Vicente, Elena, Diaz-Manera, Jordi, Querol Gutierrez, Lui, Rojas Garcia, Ricardo, Vidal, Nuria, Arribas-Ibar, Elisabet, Piehl, Fredrik, Hietala, Albert, Bjarbo, Lena, Lindberg, Christopher, Jons, Daniel, Andersson, Blanka, Sengun, Ihsan, Ozcelik, Pinar, Tuga, Celal, Ugur, Muzeyyen, Boz, Cavit, Altiparmak, Didem, Gazioglu, Sibel, Ozen Aydin, Cigdem, Erdem-Ozdamar, Sevim, Bekircan-Kurt, Can Ebru, Yilmaz, Ezgi, Acar, Nazire Pinar, Caliskan, Yagmur, Efendi, Husnu, Aydinlik, Seda, Cavus, Hakan, Semiz, Cansu, Tun, Ozlem, Terzi, Murat, Dogan, Baki, Onar, Musa Kazim, Sen, Sedat, Cavdar, Tugce Kirba, Norwood, Fiona, Dimitriou, Aikaterini, Gollogly, Jakit, Mahdi-Rogers, Mohamed, Seddigh, Arshira, Maier, Gal, Sohail, Faisal, Sathasivam, Sivakumar, Arndt, Heike, Davies, Debbie, Watling, Dave, Rivner, Michael, Hartmann, J. Edward, Quarles, Brandy, Smalley, Nicole, Amato, Anthony, Cochrane, Thoma, Salajegheh, Mohammed, Roe, Kristen, Amato, Katherine, Toska, Shirli, Wolfe, Gil, Silvestri, Nichola, Patrick, Kara, Zakalik, Karen, Katz, Jonathan, Miller, Robert, Engel, Marguerite, Bravver, Elena, Brooks, Benjamin, Plevka, Sarah, Burdette, Maryanne, Sanjak, Mohammad, Kramer, Megan, Nemeth, Joanne, Schommer, Clara, Juel, Vern, Guptill, Jeffrey, Hobson-Webb, Lisa, Beck, Kate, Carnes, Donna, Loor, John, Anderson, Amanda, Lange, Dale, Agopian, Eliz, Goldstein, Jonathan, Manning, Erin, Kaplan, Lindsay, Holzberg, Shara, Kassebaum, Nicole, Pascuzzi, Robert, Bodkin, Cynthia, Kincaid, John, Snook, Riley, Guinrich, Sandra, Micheels, Angela, Chaudhry, Vinay, Corse, Andrea, Mosmiller, Betsy, Ho, Doreen, Srinivasan, Jayashri, Vytopil, Michael, Ventura, Nichola, Scala, Stephanie, Carter, Cynthia, Donahue, Craig, Herbert, Carol, Weiner, Elaine, Mckinnon, Jonathan, Haar, Laura, Mckinnon, Naya, Alcon, Karan, Daniels, Kevin, Sattar, Nadia, Jeffery, Denni, Mckenna, Kaitlyn, Guidon, Amanda, David, William, Dheel, Christina, Levine-Weinberg, Mark, Nigro, Catherine, Simpson, Ericka, Appel, Stanley H, Lai, Eugene, Lay, Lui, Pleitez, Milvia, Halton, Sharon, Faigle, Casey, Thompson, Lisa, Sivak, Mark, Shin, Susan, Bratton, Joan, Jacobs, Daniel, Brown, Gavin, Bandukwala, Ibrez, Brown, Morri, Kane, Jennifer, Blount, Ira, Freimer, Miriam, Hoyle, J. Chad, Agriesti, Julie, Khoury, Julie, Marburger, Tessa, Kaur, Harpreet, Dimitrova, Diana, Mellion, Michelle, Sachs, George, Crabtree, Brigid, Keo, Roseann, Perez, Ele Kim, Taber, Sandra, Gilchrist, Jame, Andoin, Angela, Darnell, Taylor, Goyal, Neelam, Sakamuri, Sarada, So, Yuen T, Welsh, Lesly Welsh, Bhavaraju-Sanka, Ratna, Tobon Gonzalez, Alejandro, Jones, Floyd, Saklad, Amy, Nations, Sharon, Trivedi, Jaya, Hopkins, Steve, Kazamel, Mohamed, Alsharabati, Mohammad, Lu, Liang, Mumfrey-Thomas, Sandi, Woodall, Amy, Richman, David, Butters, Janelle, Lindsay, Molly, Mozaffar, Tahseen, Cash, Tiyonnoh, Goyal, Namita, Roy, Gulmohor, Mathew, Veena, Maqsood, Fatima, Minton, Brian, Jones, H. Jame, Rosenfeld, Jeffrey, Garcia, Rebekah, Garcia, Sonia, Echevarria, Laura, Pulley, Michael, Aranke, Shachie, Berger, Alan Ro, Shah, Jaimin, Shabbir, Yasmeen, Smith, Lisa, Varghese, Mary, Gutmann, Laurie, Gutmann, Ludwig, Swenson, Andrea, Olalde, Heena, Hafer-Macko, Charlene, Kwan, Justin, Zilliox, Lindsay, Callison, Karen, Disanzo, Beth, Naunton, Kerry, Bilsker, Martin, Sharma, Khema, Reyes, Eliana, Cooley, Anne, Michon, Sara-Claude, Steele, Julie, Karam, Chafic Karam, Chopra, Manisha, Bird, Shawn, Kaufman, Jacob, Gallatti, Nichole, Vu, Tuan, Katzin, Lara, Mcclain, Terry, Harvey, Brittany, Hart, Adam, Huynh, Kristin, Beydoun, Said, Chilingaryan, Amaiak, Droker, Brian, Lin, Frank, Shah, Akshay, Tran, Anh, Akhter, Salma, Malekniazi, Ali, Tandan, Rup, Hehir, Michael, Waheed, Waqar, Lucy, Shannon, Weiss, Michael, Distad, Jane, Downing, Sharon, Strom, Susan, Lisak, Robert, Bernitsas, Evanthia, Khan, Omar, Kumar Sriwastava, Shitiz, Tselis, Alexandro, Jia, Kelly, Bertorini, Tulio, Arnold, Thoma, Henderson, Kendrick, Pillai, Rekha, Liu, Ye, Wheeler, Lauren, Hewlett, Jasmine, Vanderhook, Mollie, Dicapua, Daniel, Keung, Benison, Kumar, Aditya, Patwa, Huned, Robeson, Kimberly, Nye, Joan, Vu, Hong, Howard, J, Utsugisawa, K, Benatar, M, Murai, H, Barohn, R, Illa, I, Jacob, S, Vissing, J, Burns, T, Kissel, J, Muppidi, S, Nowak, R, O'Brien, F, Wang, J, Mantegazza, R, and Bonanno, S
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Male ,0301 basic medicine ,medicine.medical_treatment ,Drug Resistance ,Adult ,Aged ,Antibodies, Monoclonal, Humanized ,Autoantibodies ,Double-Blind Method ,Female ,Humans ,Middle Aged ,Myasthenia Gravis ,Receptors, Cholinergic ,Outcome Assessment (Health Care) ,Severity of Illness Index ,Neurology (clinical) ,law.invention ,Complement inhibitor ,0302 clinical medicine ,Randomized controlled trial ,law ,Monoclonal ,Receptors ,Clinical endpoint ,Humanized ,Cholinergic ,education.field_of_study ,Eculizumab ,Autoantibodie ,Myasthenia Gravi ,Settore MED/26 - NEUROLOGIA ,Human ,medicine.drug ,Meningitides ,medicine.medical_specialty ,Population ,Placebo ,Antibodies ,03 medical and health sciences ,Internal medicine ,medicine ,education ,business.industry ,Surgery ,Thymectomy ,030104 developmental biology ,business ,030217 neurology & neurosurgery - Abstract
Background Complement is likely to have a role in refractory generalised myasthenia gravis, but no approved therapies specifically target this system. Results from a phase 2 study suggested that eculizumab, a terminal complement inhibitor, produced clinically meaningful improvements in patients with anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis. We further assessed the efficacy and safety of eculizumab in this patient population in a phase 3 trial. Methods We did a phase 3, randomised, double-blind, placebo-controlled, multicentre study (REGAIN) in 76 hospitals and specialised clinics in 17 countries across North America, Latin America, Europe, and Asia. Eligible patients were aged at least 18 years, with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of 6 or more, Myasthenia Gravis Foundation of America (MGFA) class II-IV disease, vaccination against Neisseria meningitides, and previous treatment with at least two immunosuppressive therapies or one immunosuppressive therapy and chronic intravenous immunoglobulin or plasma exchange for 12 months without symptom control. Patients with a history of thymoma or thymic neoplasms, thymectomy within 12 months before screening, or use of intravenous immunoglobulin or plasma exchange within 4 weeks before randomisation, or rituximab within 6 months before screening, were excluded. We randomly assigned participants (1:1) to either intravenous eculizumab or intravenous matched placebo for 26 weeks. Dosing for eculizumab was 900 mg on day 1 and at weeks 1, 2, and 3; 1200 mg at week 4; and 1200 mg given every second week thereafter as maintenance dosing. Randomisation was done centrally with an interactive voice or web-response system with patients stratified to one of four groups based on MGFA disease classification. Where possible, patients were maintained on existing myasthenia gravis therapies and rescue medication was allowed at the study physician's discretion. Patients, investigators, staff, and outcome assessors were masked to treatment assignment. The primary efficacy endpoint was the change from baseline to week 26 in MG-ADL total score measured by worst-rank ANCOVA. The efficacy population set was defined as all patients randomly assigned to treatment groups who received at least one dose of study drug, had a valid baseline MG-ADL assessment, and at least one post-baseline MG-ADL assessment. The safety analyses included all randomly assigned patients who received eculizumab or placebo. This trial is registered with ClinicalTrials.gov, number NCT01997229. Findings Between April 30, 2014, and Feb 19, 2016, we randomly assigned and treated 125 patients, 62 with eculizumab and 63 with placebo. The primary analysis showed no significant difference between eculizumab and placebo (least-squares mean rank 56·6 [SEM 4·5] vs 68·3 [4·5]; rank-based treatment difference -11·7, 95% CI -24·3 to 0·96; p=0·0698). No deaths or cases of meningococcal infection occurred during the study. The most common adverse events in both groups were headache and upper respiratory tract infection (ten [16%] for both events in the eculizumab group and 12 [19%] for both in the placebo group). Myasthenia gravis exacerbations were reported by six (10%) patients in the eculizumab group and 15 (24%) in the placebo group. Six (10%) patients in the eculizumab group and 12 (19%) in the placebo group required rescue therapy. Interpretation The change in the MG-ADL score was not statistically significant between eculizumab and placebo, as measured by the worst-rank analysis. Eculizumab was well tolerated. The use of a worst-rank analytical approach proved to be an important limitation of this study since the secondary and sensitivity analyses results were inconsistent with the primary endpoint result; further research into the role of complement is needed. Funding Alexion Pharmaceuticals.
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- 2017
38. US Clinical Practice Experience with Eculizumab in Myasthenia Gravis: Acute Clinical Events and Healthcare Resource Utilization.
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Nowak RJ, Habib AA, Klink AJ, Muppidi S, Parthan A, Sader SC, Balanean A, Gajra A, and Howard JF Jr
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Background and Objective: The terminal complement inhibitor eculizumab is approved in the USA for the treatment of patients with acetylcholine receptor antibody-positive generalized myasthenia gravis (MG). The ELEVATE study aimed to examine clinical-practice outcome data on eculizumab effectiveness in US adults with MG (generalized or ocular). This paper reports the findings on MG exacerbations and crises and associated healthcare resource utilization, and the use of rescue therapy., Methods: A retrospective chart review was conducted of US adults with MG who initiated eculizumab. Outcomes assessed for up to 2 years before and after eculizumab initiation included percentages and rates per patient per year (PPPY) of exacerbations and crises (the latter defined as intubation/impending intubation), healthcare resource utilization, and rescue therapy administration., Results: A total of 119 patients diagnosed with MG were enrolled in the study; 92 patients had ≥ 3 months of data both before and during eculizumab therapy and were included in the analyses. The mean rate of MG exacerbations decreased from 0.385 PPPY before eculizumab initiation to 0.152 PPPY during eculizumab treatment (p = 0.0034); the mean rate of MG crises decreased from 0.411 to 0.056 PPPY (p = 0.0018). Rates of healthcare resource utilization and rescue therapy use also decreased significantly during eculizumab treatment., Conclusions: This retrospective chart review analysis provides evidence for a beneficial impact of eculizumab treatment on the incidence of MG exacerbations and crises and associated healthcare resource utilization in clinical practice, and on rescue therapy use. These data further support the therapeutic benefits of eculizumab in patients with MG., (© 2024. The Author(s).)
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- 2024
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39. United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use.
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Habib AA, Klink AJ, Muppidi S, Parthan A, Sader SC, Balanean A, Gajra A, Nowak RJ, and Howard JF Jr
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- Humans, Female, Male, Middle Aged, Retrospective Studies, Adult, Aged, United States, Activities of Daily Living, Myasthenia Gravis drug therapy, Antibodies, Monoclonal, Humanized administration & dosage, Antibodies, Monoclonal, Humanized therapeutic use, Antibodies, Monoclonal, Humanized pharmacology, Immunosuppressive Agents therapeutic use, Immunosuppressive Agents administration & dosage, Complement Inactivating Agents administration & dosage, Complement Inactivating Agents therapeutic use
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Background/objectives: The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG). The aim of the ELEVATE study was to assess the effectiveness of eculizumab in clinical practice in adults with MG in the United States., Methods: A retrospective chart review was conducted in adults with MG who initiated eculizumab treatment between October 23, 2017 and December 31, 2019. Outcomes assessed before and during eculizumab treatment using a pre- versus post-treatment study design included Myasthenia Gravis-Activities of Daily Living (MG-ADL) total scores; minimal symptom expression (MSE); physician impression of clinical change; minimal manifestation status (MMS); and concomitant medication use., Results: In total, 119 patients were included in the study. A significant reduction was observed in mean MG-ADL total score, from 8.0 before eculizumab initiation to 5.4 at 3 months and to 4.7 at 24 months after eculizumab initiation (both p < 0.001). At 24 months after eculizumab initiation, MSE was achieved by 19% of patients. MMS or better was achieved by 30% of patients at 24 months. Additionally, 64% of patients receiving prednisone at eculizumab initiation had their prednisone dosage reduced during eculizumab treatment and 13% discontinued prednisone; 32% were able to discontinue nonsteroidal immunosuppressant therapy., Discussion: Eculizumab treatment was associated with sustained improvements in MG-ADL total scores through 24 months in adults with MG. Prednisone dosage was reduced in approximately two-thirds of patients, suggesting a steroid-sparing effect for eculizumab., (© 2024. The Author(s).)
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- 2024
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40. Psychometric properties of MG-ADL items and MG-ADL score: An assessment of distributional characteristics, validity and factor structure in two large datasets.
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Janssen MF, Dewilde S, Wolfe GI, Muppidi S, and Phillips G
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- Humans, Male, Female, Middle Aged, Cross-Sectional Studies, Aged, Prospective Studies, Quality of Life psychology, Reproducibility of Results, Longitudinal Studies, Adult, Outcome Assessment, Health Care, Activities of Daily Living, Psychometrics methods, Myasthenia Gravis psychology, Myasthenia Gravis diagnosis
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Background: The Myasthenia Gravis-Activities of Daily Living scale (MG-ADL) is an 8-item outcome measure to assess symptoms and functional limitations in myasthenia gravis (MG) patients. The MG-ADL score is an equally weighted level sum score that is used as primary outcome measures in clinical trials, in clinical practice, and as an end-point in health economic evaluation. This data analysis aims to obtain detailed knowledge of measurement properties of MG-ADL items and the MG-ADL score., Methods: Cross-sectional data from a real-world prospective study (MRW) were combined with longitudinal data from the ADAPT trial. Outcome measures included were MG-ADL, Quantitative Myasthenia Gravis score (QMG), MG 15-item Quality of Life (MG-QOL15r) and EQ-5D-5L. Patients were categorized by their Myasthenia Gravis Foundation of America (MGFA) clinical classification. The following measurement properties were assessed: distributional characteristics, inter-item correlation, convergent, known groups and construct validity and internal factor structure., Results: Correlations of items within MG-ADL dimensions were moderate, while MG-ADL correlations between comparable MG-QOL15r and QMG items were mixed. Known groups validity for the MG-ADL score was demonstrated for MGFA class. Mean MG-ADL item level scores by MGFA class demonstrated construct validity. PCA, including all four outcome measures, resulted in a nine factor solution., Discussion: Psychometric properties of individual MG-ADL items were moderate to good. This study showed that the MG-ADL adequately captures the multidimensional heterogeneous nature of MG. This is, however, accompanied by mixed psychometric performance of the MG-ADL score, which may complicate health economic modelling., Registration: MyRealWorld-MG was registered on November 25, 2019, with registration numberNCT04176211. The ADAPT randomized clinical trial is registered atClinicalTrials.gov(NCT03669588)., (Copyright © 2024 The Authors. Published by Elsevier B.V. All rights reserved.)
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- 2024
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41. Ravulizumab use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice.
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Katyal N, Govindarajan R, Goyal N, Muley S, and Muppidi S
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Purpose: To describe the early experience of ravulizumab use in acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR+ve gMG)., Methods: This multicenter retrospective study included AChR+ve gMG patients who were treated with ravulizumab and had both pre- and post-ravulizumab myasthenia gravis activities of daily living (MG-ADL) scores. Clinical information regarding MG history, concomitant treatment(s), MG-ADL, other MG-specific measures, and adverse events were recorded., Results: A total of 18 patients with mean age of 61.83 (±16.08, n = 18) years were included in this cohort. In 10 complement inhibitor naive patients, a clinically meaningful reduction in mean Mg-ADL (baseline: 6.6 (±3.58) vs. 4.4 (±2.28), post ravulizumab) was seen. 6 out of 10 patients (60%) had clinically meaningful reduction post ravulizumab and two achieved minimum symptom expression (MSE). In 8 patients switched from eculizumab to ravulizumab, further reduction was noted in post ravulizumab mean MG-ADL (Baseline: 3.25 (±3.34) vs. 1.5 (±2.34) post ravulizumab). None of the patients who switched from eculizumab to ravulizumab experienced worsening symptoms. Eleven out of 14 (78.5%) patients on prednisone therapy were able to reduce their prednisone dose post-ravulizumab. None of the patients experienced any major side effects., Conclusion: In our clinical practice, 60% of AChR+ve gMG complement inhibitor naive patients experienced a clinically meaningful improvement in MG-ADL scores with ravulizumab. Patients were safely switched from eculizumab to ravulizumab and had further improvement in their mean MG-ADL scores. Of those on prednisone therapy, the majority were able to reduce their prednisone dosage., Competing Interests: RG served on the advisory board for Argenx, UCB, Janssen, Roche, and speakers bureau for Argenx and Alexion. NG has served on advisory board meetings for Argenx, Alexion, and UCB Pharma. SrM has served on advisory board meetings for Argenx, Alexion/Astrazaneca, ra/UCB, and Horizont Pharma. SuM speaker Bureau: Takeda, CSL Behring, Grifols, Catalyst, Alexion, Argenx. Consultant: UCB, Alexion, Argenx. Advisory Boards: Horizon, Argenx, Alexion, Grifols. The remaining author declares that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2024 Katyal, Govindarajan, Goyal, Muley and Muppidi.)
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- 2024
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42. Utilization of APE2 and RITE2 scores in autoimmune encephalitis patients with seizures.
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Rafferty T, Koeppen-Babcock A, Muppidi S, Li Y, and Le S
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- Humans, Female, Male, Adult, Middle Aged, Retrospective Studies, Hashimoto Disease complications, Hashimoto Disease blood, Aged, Sensitivity and Specificity, Autoantibodies blood, Young Adult, Immunotherapy methods, Adolescent, Seizures blood, Seizures etiology, Seizures diagnosis, Encephalitis complications, Encephalitis blood, Encephalitis immunology, Encephalitis diagnosis
- Abstract
Purpose: Immune-mediated seizures are rare but are increasingly recognized as an etiology of seizures resistant to anti-seizure medications (ASMs). Antibody Prevalence in Epilepsy 2 (APE2) and Response to Immunotherapy in Epilepsy 2 (RITE2) scores were developed recently to identify patients who may be seropositive for serum central nervous system (CNS) specific antibodies (Ab) and may benefit from immunotherapy (Dubey et al. 2018). The goal of this study was to apply APE2 and RITE2 scores to an independent cohort of patients with seizures secondary to autoimmune encephalitis (AE) and to further verify the sensitivity and specificity of the scores., Principal Results: We conducted a retrospective study at Stanford University Hospital between 2008 and 2021 and included patients who had acute seizures and AE using diagnostic criteria from Graus (n = 34 definite AE, 10 probable AE, and 12 possible AE) (Graus et al. 2016). Patients were excluded if they did not have a serum Ab panel investigated or had alternate diagnoses (n = 55). APE2 and RITE2 scores were calculated based on clinical and diagnostic data (n = 56). Serum Ab were positive in 73 % of patients, in which 63 % cases carried CNS specific Ab. An APE2 score ≥ 4 had a sensitivity of 97 % and specificity of 14 % to predict a positive serum CNS specific Ab. A RITE2 score ≥ 7 had a sensitivity of 93 % and specificity of 60 % to predict seizure responsiveness to immunotherapy., Conclusion: APE2 and RITE2 scores had high sensitivities but low specificities to predict seropositivity and seizure responsiveness to immunotherapy in patients with autoimmune encephalitis with seizures., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2024 Elsevier Inc. All rights reserved.)
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- 2024
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43. Soil Moisture and Heat Level Prediction for Plant Health Monitoring Using Deep Learning with Gannet Namib Beetle Optimization in IoT.
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Bhamidipati K, Muppidi S, Reddy PVB, and Merugula S
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- Animals, Hot Temperature, Soil, Deep Learning, Internet of Things, Coleoptera
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Plant health monitoring is crucial in ensuring a constant food supply to satisfy the growing demand for food. Hence, it is essential to monitor plant health to maximize the yield and minimize the risk of various diseases. Soil moisture and temperature are of critical importance in plant growth, and predicting them enables farmers to take preventive actions, thereby mitigating the issues affecting plant health. This work presents a plant health monitoring approach by forecasting soil moisture and heat levels by collecting data in an Internet of Things (IoT) environment. Here, for transmitting the soil data acquired by the IoT nodes, a cluster head (CH) selection and routing technique using Gannet Namib beetle optimization (GNBO) is used. The data is routed to a prediction module, wherein soil moisture and heat levels are predicted by Convolutional long short term memory (Conv-LSTM). Furthermore, the hyperparameters of the Conv-LSTM are optimized by the GNBO algorithm. The efficiency of the GNBO-Conv-LSTM is examined based on link life time (LLT), energy, delay, distance, negative predictive value (NPV), positive predictive value (PPV), and true negative rate (TNR) and is observed to have achieved values of 0.675, 0.478 J, 0.092 ms, 50.200 m, 0.885, 0.882, and 0.875, correspondingly., (© 2023. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)
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- 2024
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44. Plasma Exchange in Patients With Myositis due to Immune Checkpoint Inhibitor Therapy.
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Katyal N, Katsumoto TR, Ramachandran KJ, Yunce M, and Muppidi S
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- Male, Humans, Middle Aged, Aged, Immune Checkpoint Inhibitors, Plasma Exchange, Nivolumab adverse effects, Creatine Kinase, Myositis therapy, Myositis drug therapy, Myasthenia Gravis therapy, Myasthenia Gravis drug therapy
- Abstract
Abstract: Immune checkpoint inhibitors used to treat malignancies may lead to various immune-related adverse events (irAEs) including conditions such as myositis and myasthenia gravis (MG). Here, we describe 2 cases of myositis treated effectively with therapeutic plasma exchange (PLEX). A 64-year-old man with thymic cancer developed leg weakness and dyspnea 1 month after the second dose of nivolumab with moderate weakness in proximal and distal muscles, with elevated creatine kinase levels. Another 77-year-old man with Stage IIIB squamous cell carcinoma of the lung developed progressive proximal muscle weakness and became nonambulatory after cycle 2 of durvalumab with persistently high creatine kinase levels despite prednisone treatment. Electrophysiology revealed irritative myopathy without evidence of neuromuscular junction dysfunction and MG antibody testing was nonrevealing. With PLEX, both patients noticed rapid improvement in strength. PLEX in conjunction with other immunosuppressive agents can result in rapid improvement in irAE-myositis even in patients without associated MG., (Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc.)
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- 2023
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45. Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice.
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Katyal N, Halldorsdottir K, Govindarajan R, Shieh P, Muley S, Reyes P, Leung KK, Mullen J, Milani-Nejad S, Korb M, Goyal NA, Mozaffar T, Goyal N, Habib AA, and Muppidi S
- Abstract
Introduction/aims: Multiple novel therapies have been approved for patients with myasthenia gravis. Our aim is to describe the early experience of efgartigimod use in acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR+ve gMG)., Methods: This multicenter retrospective study included AChR+ve gMG patients from five major neuromuscular centers who were treated with efgartigimod and had both pre- and post-efgartigimod myasthenia gravis activities of daily living (MG-ADL) scores. Information regarding MG history, concomitant treatment(s), MG-ADL and other MG-specific measures, laboratory data, and adverse events were recorded., Results: A total of 37 patients (M:23, F:14) with a mean age of 65.56 (±14.74) y were included in this cohort. A total of 36/37 patients completed at least one cycle and 28 patients completed at least two cycles of efgartigimod. A total of 72% (26/36) of patients had a clinically meaningful reduction (≥2 point change) in MG-ADL after the completion of the first cycle of efgartigimod (mean pre-efgartigimod 8.02) (±3.09) versus post-efgartigimod 4.33 (±3.62). Twenty-five percent (9/36) achieved minimal symptom expression status after one cycle and 25% (7/28) after the second cycle. Treatment benefit was sustained after cycle 2. Three out of four patients with thymoma in this cohort had clinically significant reductions in MG-ADL scores. Immunoglobulin G (IgG) levels decreased by about 60% (n = 10). One patient had a relapse of Clostridium difficile infection resulting in the discontinuation of therapy. Four patients had mild side effects., Discussion: Efgartigimod led to clinically meaningful improvement in MG-ADL in diverse AChR+ve gMG patients but treatment frequency to achieve optimal symptom control needs to be explored., (© 2023 Wiley Periodicals LLC.)
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- 2023
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46. Utility of Repetitive Nerve Stimulation in the Diagnosis of Myasthenia Gravis in the Inpatient Setting.
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Clifford KM, Wu CK, Post D, Shaik R, and Muppidi S
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Objectives: Sensitivity and specificity of Repetitive Nerve Stimulation (RNS) is typically reported from outpatient centers, and we hypothesized that these values might not apply to hospitalized patients with higher grades of weakness. RNS may be helpful in rapidly confirming diagnosis of myasthenia gravis (MG) in the inpatient setting, as results from confirmatory antibody testing are often delayed. We sought to characterize the sensitivity and specificity of RNS in the inpatient setting to assist in the early diagnosis of MG., Methods: We performed a retrospective analysis of all adult patients who had inpatient RNS at our center from 2016 to 2021. Inclusion criteria included RNS performed at least at one site and a neurological evaluation which prompted an electrodiagnostic study to evaluate for neuromuscular junction (NMJ) pathology. Descriptive statistics and Fisher exact analysis were performed., Results: Of the 32 identified hospitalized patients, 6 had greater than 10% decrement on slow RNS, confirming NMJ dysfunction. Five were diagnosed with MG, and 1 with Lambert-Eaton myasthenic syndrome. Of the 26 patients with normal RNS, 25 ultimately had alternative causes of weakness. One was later diagnosed as seronegative MG based on clinical improvement with acetylcholinesterase inhibitors. In our inpatient population, the overall sensitivity and specificity of RNS were 83.3% and 96.2% respectively. There was a statistically significant association between a positive RNS and diagnosis of MG ( P = .0002)., Conclusions: RNS is a highly sensitive and specific test for the diagnosis of MG in an inpatient setting, and these results are likely more rapidly available compared to antibody testing., Competing Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2023.)
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- 2023
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47. Updated consensus statement: Intravenous immunoglobulin in the treatment of neuromuscular disorders report of the AANEM ad hoc committee.
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Tavee J, Brannagan TH 3rd, Lenihan MW, Muppidi S, Kellermeyer L, and D Donofrio P
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- Humans, Child, Immunoglobulins, Intravenous therapeutic use, Dermatomyositis, Neuromuscular Diseases therapy, Myasthenia Gravis therapy, Guillain-Barre Syndrome, Myositis, Polyneuropathies, Myositis, Inclusion Body
- Abstract
Intravenous immune globulin (IVIG) is an immune-modulating biologic therapy that is increasingly being used in neuromuscular disorders despite the paucity of high-quality evidence for various specific diseases. To address this, the AANEM created the 2009 consensus statement to provide guidance on the use of IVIG in neuromuscular disorders. Since then, there have been several randomized controlled trials for IVIG, a new FDA-approved indication for dermatomyositis and a revised classification system for myositis, prompting the AANEM to convene an ad hoc panel to update the existing guidelines.New recommendations based on an updated systemic review of the literature were categorized as Class I-IV. Based on Class I evidence, IVIG is recommended in the treatment of chronic inflammatory demyelinating polyneuropathy, Guillain-Barré Syndrome (GBS) in adults, multifocal motor neuropathy, dermatomyositis, stiff-person syndrome and myasthenia gravis exacerbations but not stable disease. Based on Class II evidence, IVIG is also recommended for Lambert-Eaton myasthenic syndrome and pediatric GBS. In contrast, based on Class I evidence, IVIG is not recommended for inclusion body myositis, post-polio syndrome, IgM paraproteinemic neuropathy and small fiber neuropathy that is idiopathic or associated with tri-sulfated heparin disaccharide or fibroblast growth factor receptor-3 autoantibodies. Although only Class IV evidence exists for IVIG use in necrotizing autoimmune myopathy, it should be considered for anti-hydroxy-3-methyl-glutaryl-coenzyme A reductase myositis given the risk of long-term disability. Insufficient evidence exists for the use of IVIG in Miller-Fisher syndrome, IgG and IgA paraproteinemic neuropathy, autonomic neuropathy, chronic autoimmune neuropathy, polymyositis, idiopathic brachial plexopathy and diabetic lumbosacral radiculoplexopathy., (Copyright © 2023 by the American Association of Neuromuscular & Electrodiagnostic Medicine, Inc. All rights reserved.)
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- 2023
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48. Neonatal Fc receptor blockade as emerging therapy in diseases with plasma exchange indications.
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Yunce M, Katyal N, Monis GF, and Muppidi S
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Neonatal Fc receptor (FcRn) blockade may represent a mechanism similar to plasma exchange (PLEX) in reducing immunoglobulin levels and thus have a broad implication for apheresis practitioners. Although only efgartigimod received FDA approval for myasthenia gravis in December 2021, multiple trials are currently underway with different FcRn therapies in a varied group of IgG antibody-mediated neurological and hematological disorders which are outlined in this review. In this review we discuss FcRn's mechanism of action, and its potential use in various neurological and non-neurological diseases. In addition, we further compare the kinetics and adverse events of PLEX and FcRn blockade. We encourage apheresis practitioners to be familiar with this class of drugs in order to better understand how these two therapies can be used either standalone, or in combination with other therapies as both FcRn antagonism and PLEX improve clinical state by reducing IgG levels and pathogenic antibodies., (© 2023 Wiley Periodicals LLC.)
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- 2023
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49. NGLY1 deficiency: a prospective natural history study.
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Tong S, Ventola P, Frater CH, Klotz J, Phillips JM, Muppidi S, Dwight SS, Mueller WF, Beahm BJ, Wilsey M, and Lee KJ
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- Humans, Child, Prospective Studies, Biomarkers, Quality of Life, Congenital Disorders of Glycosylation
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N-glycanase 1 (NGLY1) deficiency is a debilitating, ultra-rare autosomal recessive disorder caused by loss of function of NGLY1, a cytosolic enzyme that deglycosylates other proteins. It is characterized by severe global developmental delay and/or intellectual disability, hyperkinetic movement disorder, transient elevation of transaminases, (hypo)alacrima and progressive, diffuse, length-dependent sensorimotor polyneuropathy. A prospective natural history study (NHS) was conducted to elucidate clinical features and disease course. Twenty-nine participants were enrolled (15 onsite, 14 remotely) and followed for up to 32 months, representing ~29% of the ~100 patients identified worldwide. Participants exhibited profound developmental delays, with almost all developmental quotients below 20 on the Mullen Scales of Early Learning, well below the normative score of 100. Increased difficulties with sitting and standing suggested decline in motor function over time. Most patients presented with (hypo)alacrima and reduced sweat response. Pediatric quality of life was poor except for emotional function. Language/communication and motor skill problems including hand use were reported by caregivers as the most bothersome symptoms. Levels of the substrate biomarker, GlcNAc-Asn (aspartylglucosamine; GNA), were consistently elevated in all participants over time, independent of age. Liver enzymes were elevated for some participants but improved especially in younger patients and did not reach levels indicating severe liver disease. Three participants died during the study period. Data from this NHS informs selection of endpoints and assessments for future clinical trials for NGLY1 deficiency interventions. Potential endpoints include GNA biomarker levels, neurocognitive assessments, autonomic and motor function (particularly hand use), (hypo)alacrima and quality of life., (© The Author(s) 2023. Published by Oxford University Press. All rights reserved. For Permissions, please email: journals.permissions@oup.com.)
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- 2023
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50. Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG).
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Berrih-Aknin S, Palace J, Meisel A, Claeys KG, Muppidi S, Saccà F, Amini F, Larkin M, Quinn C, Beauchamp J, Philips G, De Ruyck F, Ramirez J, and Paci S
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- Adult, Humans, Female, Middle Aged, Longitudinal Studies, Surveys and Questionnaires, Anxiety epidemiology, Prospective Studies, Myasthenia Gravis therapy, Myasthenia Gravis drug therapy
- Abstract
Objectives: This study aims to explore the impact of myasthenia gravis (MG) - in terms of treatments, side effects, comorbidities, psychological health and work or study- in the real world from a patient perspective., Design and Participants: This is a prospective, observational, digital, longitudinal study. Adults diagnosed with MG residing in the USA, Japan, Germany, the UK, Italy, Spain or Canada were eligible to participate in the study. There were no other exclusion criteria. Participants used a bespoke smartphone application to confirm eligibility, provide consent and enter data about their MG into a profile, a tracker to record MG-related events and a series of patient-reported outcome instruments. 1693 participants completed at least 1 survey and were included in this analysis., Results: Results are presented as a percentage of respondents to each survey question. The study population was largely female (69% of 1586 respondents), with an average age of 49.9 years (SD 14.8). In the previous 12 months, 83.7% of 1412 respondents confirmed that they had received one or more routine treatments for MG, and 67.1% of 255 respondents confirmed that they had experienced a side effect in the previous month. Commonly experienced comorbidities reported by 966 respondents were thyroid problems, hypertension and anxiety, experienced by 37.5%, 31.4% and 28.0% of respondents, respectively.According to 889 respondents to the Hospital Anxiety and Depression Scale survey, 52.7% and 43.2% had a score indicative of at least mild anxiety and mild depression, respectively. Of 257 respondents, 33.0% reported experiencing a work or study impact in the past month., Conclusions: This analysis of baseline characteristics of the MyRealWorld MG study population indicates that, despite current treatments, patients experience notable burden. Further scheduled analyses will develop a longitudinal picture of MG burden., Trial Registration Number: NCT04176211., Competing Interests: Competing interests: The principal investigator, ML is CEO and owner of Vitaccess, which has been commissioned by argenx BV to carry out the study. FA and CQ are employees of Vitaccess. JB, GP, FDR, JR and SP are employees of argenx BV, the sponsor of the study. SB-A is a consultant and receives honoraria from argenx BV for this study. AM has received speaker honoraria, consulting fees or financial research support from Alexion, argenx BV, Grifols, Hormosan Pharma, Janssen, Octapharma and UCB. He serves as Chairman of the medical advisory board of the German Myasthenia Gravis Society. KGC has received advisory board honoraria, speaker fees and funding for research from Alnylam Pharmaceuticals, Biogen, CSL Behring and Sanofi-Genzyme, and travel reimbursement from Sanofi-Genzyme. KGC holds the Emil von Behring Chair in Neuromuscular and Neurodegenerative Disorders, sponsored by CSL Behring. FS has received public-speaking honoraria from Almirall, Biogen, Mylan, Novartis, Roche, Sanofi and Teva Pharmaceuticals, and served on advisory boards for Almirall, argenx BV, AveXis, Biogen, Forward Pharma, Lexeo Therapeutics, Merck, Novartis, Novatek Pharmaceuticals, Pomona, Roche, Sanofi and Takeda., (© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)
- Published
- 2023
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