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60. Saltwater intrusion impacts microbial diversity and function in groundwater ecosystems

Author

Houghton, K. M., Fournier, M., and Tschritter, C.

Subjects
Toxicity, Salt, Groundwater, Chloride, Ecosystems, Microbial Diversity
Abstract

Journal manuscript to be submitted Microbial communities in groundwater carry out biochemical processes which are essential ecosystem functions, such as nutrient cycling and removal of contaminants. The effects of increasing salinity from rising sea level, or over-abstraction, on these microbes are unknown. This study uses a combination of culture dependent (growth curves, isolation of bacteria) and culture-independent (16S rRNA gene sequencing) methods to identify the potential effects of saltwater intrusion on groundwater microbes and their ecosystem functions.

Published
2022
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76. Hypertensive disorders in women with peripartum cardiomyopathy: insights from the ESC EORP PPCM Registry

Author

Jackson, Alice M., Petrie, Mark C., Frogoudaki, Alexandra, Laroche, Cécile, Gustafsson, Finn, Ibrahim, Bassem, Mebazaa, Alexandre, Johnson, Mark R., Seferovic, Petar M., Regitz‐Zagrosek, Vera, Mbakwem, Amam, Böhm, Michael, Prameswari, Hawani Sasmaya, Abdel Gawad, Doaa Ahmed Fouad, Goland, Sorel, Damasceno, Albertino, Karaye, Kamilu, Farhan, Hasan Ali, Hamdan, Righab, Maggioni, Aldo P., Sliwa, Karen, Bauersachs, Johann, Meer, Peter, Favaloro, R., Favaloro, L., Carballo, M., Peradejordi, M., Renedo, M.F., Absi, D., Bertolotti, A., Ratto, R., Talavera, M.L., Gomez, R., Lockwood, S., Barton, T., Austin, M‐A., Arstall, M., Aldridge, E., Chow, Y.Y., Dekker, G., Mahadavan, G., Rose, J., Wittwer, M., Hoppe, U., Sandhofer, A., Bahshaliyev, A., Gasimov, Z., Babayev, A., Niftiyev, P., Hasanova, I., AlBannay, R., AlHaiki, W., Husain, A., Mahdi, N., Kurlianskaya, A., Lukyanchyk, M., Shatova, O., Troyanova‐Shchutskaia, T., Anghel, L., De Pauw, M., Gevaert, S., De Backer, J., De Hosson, M., Vervaet, P., Timmermans, P.J., Janssen, A., Yameogo, N.V., Kagambega, L.J., Cumyn, A., Caron, N., Cote, A‐M., Sauve, N., Nkulu, D. Ngoy, Lez, D. Malamba, Yolola, E. Ngoy, Krejci, J., Poloczkova, H., Ersboll, A., Gustafsson, F., Elrakshy, Y., Hassanein, M., Hammad, B., Eldin, O. Nour, Fouad, D., Salman, S., Zareh, Z., Abdeall, D., Elenin, H. Abo, Ebaid, H., El Nagar, A., Farag, S., Saed, M., El Rahman, Y H Abd, Ibrahim, B.S., Abdelhamid, M., Hanna, R.N. W., Youssef, G., Awad, R., Botrous, O.L. I., Halawa, S. Ibrahim, Nasr, G., Saad, A., El Tahlawi, M., Abdelbaset, M., El‐saadawy, M., El‐shorbagy, A., Shalaby, G., Anttonen, O., Tolppanen, H., Hamekoski, S., Menez, T., Noel, A., Lamblin, N., Mouquet, F., Coulon, C., Groote, P., Langlois, S., Schurtz, G., Cohen‐Solal, A., Mebazaa, A., Fournier, M‐C., Louadah, B., Akrout, N., Logeart, D., Leurent, G., Jovanova, S., Arnaudova‐Dezulovicj, F., Livrinova, V., Bauersachs, J., Hilfiker‐Kleiner, D., Berliner, D., Jungesblut, M., Koenig, T., Moulig, V.A., Pfeffer, T.J., Böhm, M., Kindermann, I., Schwarz, V., Schmitt, C., Swojanowsky, P., Pettit, S., Petrie, M., McAdam, M., Patton, D., Bakhai, A., Krishnamurthy, V., Lim, L., Clifford, P., Bowers, N., Clark, A. L., Witte, K., Cullington, D., Oliver, J., Simms, A., Mcginlay, M., McDonagh, T., Shah, A. M., Amin‐Youssef, G., De Courcey, J., Martin, K., Shaw, S., Vause, S., Wallace, S., Malin, G., Wick, C., Nikolaou, M., Rentoukas, I., Chinchilla, H., Andino, L., Iyengar, S., Chandra, S., Yadav, D.K., Babu, R. Ravi, Singh, A.K., Kumar, S., Karunamay, B.B., Chaubey, S.K., Dhiman, S.R., Jha, V.C., Singh, S.K., Kodati, D., Dasari, R., Sultana, S., Dewi, T.I., Prameswari, H. Sasmaya, Al‐Farhan, H.A., Al‐Hussein, A., Yaseen, I.F., Al‐Azzawi, Falah, Al‐Saedi, Ghazi, Mahmood, G.M., Mohammed, M.K., Ridha, A.F., Shotan, A., Vazan, A., Goland, S., Biener, M., Senni, M., Grosu, A., Martin, E., Esposti, D. Degli, Bacchelli, S., Borghi, C., Metra, M., Sciatti, E., Orabona, R., Sani, F., Brunetti, N.D., Sinagra, G., Bobbo, M., D'Agata Mottolese, B., Gesuete, V., Rakar, S., Ramani, F., Kamiya, C., Barasa, A., Ngunga, M., Bajraktari, G., Hyseni, V., Lleshi, D., Pllana, E., Pllana, T., Noruzbaeva, A., Ismailov, F., Mirrakhimov, E., Abilova, S., Lunegova, O., Kerimkulova, A., Osmankulova, G., Duishenalieva, M., Kurmanbekova, B., Turgunov, M., Mamasaidova, S., Bektasheva, E., Kavoliuniene, A., Muckiene, G., Vaitiekiene, A., Celutkiene, J., Balkevicine, L., Barysiene, J., Chee, K.H., Damasceno, A., Machava, M., Veldhuisen, D.J., Meer, P., Berg, M., Roos‐Hesselink, J., Hagen, I., Baris, L., Hurtado, P., Ezeonu, P., Isiguzo, G., Obeka, N., Onoh, R., Asogwa, F., Onyema, C., Otti, K., Ojji, D., Odili, A., Nwankwo, A., Karaye, K., Ishaq, N., Sanni, B., Abubakar, H., Mohammed, B., Sani, M., Kehinde, M., Mbakwem, A., Afolabi, B., Amadi, C., Kilasho, M., Qamar, N., Furnaz, S., Gurmani, S., Kayani, M.G.A. Mahmood, Munir, R., Hussain, S., Malik, S., Mumtaz, S., Saligan, J.R., Rubis, P., Biernacka‐Fijalkowska, B., Lesniak‐Sobelga, A., Wisniowska‐Smialek, S., Kasprzak, J.D., Lelonek, M., Zycinski, P., Jankowski, L., Grajek, S., Oko‐Sarnowska, Z., Rutkowska, A. Bartczak, Kaluzna‐Oleksy, M., Plaskota, K., Demkow, M., Dzielinska, Z., Henzel, J., Kryczka, K., Moiseeva, O., Irtyuga, O., Karelkina, E., Zazerskaya, I., Seferovic, P.M., Milinkovic, I., Živkovic, I., Ristic, A.D., Milasinovic, D., Kong, W. KF, Tan, L.K., Tan, J.L., Thain, S., Poh, K.K., Yip, J., Sliwa, K., Azibani, F., Hovelmann, J., Viljoen, C., Briton, O., Zamora, E., Orcajo, N. Alonso, Carbonell, R., Pascual, C., Muncharaz, J. Farre, Alonso‐Pulpon, L., Cubero, J. Segovia, Urquia, M. Taibo, Garcia‐Pavia, P., Gomez‐Bueno, M., Cobo‐Marcos, M., Briceno, A., Galvan, E. De Teresa, Garcia‐Pinilla, J.M., Robles‐Mezcua, A., Morcillo‐Hildalgo, L., Elbushi, A., Suliman, A., Ahamed, N., Jazzar, K., Murtada, M., Schaufelberger, M., Goloskokova, V., Hullin, R., Yarol, N., Arrigo, M., Cavusoglu, Y., Eraslan, S., Fak, A.S., Enar, S. Catirli, Sarac, L., Cankurtaran, B., Gumrukcuoglu, H., Ozturk, F., Omagino, J., Mondo, C., Lwabi, P., Ingabire, P., Nabbaale, J., Nyakoojo, W., Okello, E., Sebatta, E., Ssinabulya, I., Atukunda, E., Kitooleko, S., Semu, T., Salih, B.T., Komaranchath, A.M., Almahmeed, W.A.R., Gerges, F., Farook, F.S. Mohamed, Albakshy, F., Mahmood, N., Wani, S., Freudenberger, R., Islam, N., Quinones, J., Sundlof, D., Beitler, C., Centolanza, L., Cornell, K., Huffaker, S., Matos, L., Marzo, K., Paruchuri, V., Patel, D., Abdullaev, T., Alyavi, B., Mirzarakhimova, S., Tsoy, I., Bekbulatova, R., and Uzokov, J.

Abstract

Aims: \ud Hypertensive disorders occur in women with peripartum cardiomyopathy (PPCM). How often hypertensive disorders co-exist, and to what extent they impact outcomes, is less clear. We describe differences in phenotype and outcomes in women with PPCM with and without hypertensive disorders during pregnancy.\ud \ud Methods: \ud The European Society of Cardiology PPCM Registry enrolled women with PPCM from 2012-2018. Three groups were examined: 1) women without hypertension (‘PPCM-noHTN’); 2) women with hypertension but without pre-eclampsia (‘PPCM-HTN’); 3) women with pre-eclampsia (‘PPCM-PE’). Maternal (6-month) and neonatal outcomes were compared.\ud \ud Results: \ud Of 735 women included, 452 (61.5%) had PPCM-noHTN, 99 (13.5%) had PPCM-HTN and 184 (25.0%) had PPCM-PE. Compared to women with PPCM-noHTN, women with PPCM-PE had more severe symptoms (NYHA IV in 44.4% and 29.9%, p

Published
2021

79. ‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab

Author

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.

Published
2020
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80. Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Author

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. 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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

Subjects
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

Published
2020

83. Infections and multiple sclerosis: Recommendations from the French Multiple Sclerosis Society

Author

Papeix, C., primary, Donze, C., additional, Lebrun-Frénay, C., additional, Papeix, C., additional, Donzé, C., additional, Laplaud, D., additional, Thouvenot, E., additional, Ayrignac, X., additional, Pourcher-Martinez, V., additional, Zéphir, H., additional, de Seze, J., additional, Michel, L., additional, Bensa, C., additional, Cara-Dalliere, C., additional, Guen-noc, A.M., additional, Casez, O., additional, Maarouf, A., additional, Bourre, B., additional, Kwiatkowski, A., additional, Cohen, M., additional, Maillart, E., additional, Collongues, N., additional, Louapre, C., additional, Androdias, G., additional, Guegen, A., additional, Audoin, B., additional, Mattey, G., additional, Bernady, P., additional, Faucheux, J.M., additional, Labauge, P., additional, Meckies, C., additional, Stankoff, B., additional, Tourniaire, P., additional, Dinh, A., additional, Guennoc, A.M., additional, Durnad-Dubief, F., additional, Wiertlewski, S., additional, Derache, N., additional, Le page, E., additional, Pittion, S., additional, Vukusic, S., additional, Clavelou, P., additional, Heinzlef, O., additional, Colamarino, R., additional, Planque, E., additional, Rico, A., additional, Sheiber nogueira, C., additional, de Seze, M., additional, Ciron, J., additional, Alchaar, H., additional, Bensmail, D., additional, Biotti, D., additional, Branger, P., additional, Brochet, B., additional, Castan, B., additional, Creange, A., additional, Creisson, E., additional, DeBroucker, T., additional, Depaz, R., additional, Douay, X., additional, Dulau, C., additional, Faucher, M., additional, Fournier, M., additional, Fromont, A., additional, Gallien, P., additional, Gout, O., additional, Grimaud, J., additional, Hervé, Y., additional, Kerbrat, A., additional, Kremer, L., additional, Lanotte, L., additional, Magy, L., additional, Mania, A., additional, Maurousset, A., additional, Moisset, X., additional, Montcuquet, A., additional, Moreau, T., additional, Morel, N., additional, Patry, I., additional, Peaureaux, D., additional, Pouget, M.C., additional, Ruet, A., additional, Saint-Val, C., additional, Stahl, J.P., additional, Taithe, F., additional, Tattevin, P., additional, Vaillant, M., additional, and Vuoto, F., additional

Published
2021
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93. 1613P What are the barriers to routine clinical use of teleconsultation in oncology? A retrospective study on patient’s and their physician’s satisfaction with 603 video teleconsultations

Author

Mottet, T., primary, Soubeyran, P., additional, Godbert, Y., additional, Cabart, M., additional, Roubaud, G., additional, Chakiba, C., additional, Bourcier, K., additional, Haik, L., additional, Lebreton, C., additional, Floquet, A., additional, Charitansky, H., additional, Fournier, M., additional, Toulmonde, M., additional, Pernot, S., additional, Annonay, M., additional, Enfedaque, S., additional, Cassauba, S., additional, Italiano, A., additional, Mathoulin-Pelissier, S., additional, and Tueux, N. Quenel, additional

Published
2021
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94. 134P Enrolment in clinical trials (CT) among patients (pts) with early breast cancer (BC)

Author

Presti, D., primary, Havas, J., additional, Soldato, D., additional, Lapidari, P., additional, Martin, E., additional, Pistilli, B., additional, Martin, A-L., additional, Everhard, S., additional, Jouannaud, C., additional, Levy, C., additional, Rigal, O., additional, Fournier, M., additional, Soulié, P., additional, Mouret-Reynier, M-A., additional, Tarpin, C., additional, Campone, M., additional, Guillermet, S., additional, André, F., additional, Vaz-Luis, I., additional, and Di Meglio, A., additional

Published
2021
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95. Discovery of a Novel Non-Narcotic Analgesic Derived from the CL-20 Explosive: Synthesis, Pharmacology and Target Identification of Thio-wurtzine, a Potent Inhibitor of the Opioid Receptors and the Voltage-Dependent Calcium Channels

Author

S. V. Sysolyatin, Fournier M, A. I. Kalashnikov, Raphaël Terreux, S. G. Krylova, Alexander Vorozhtsov, Stephanie Aguero, D. A. Kulagina, Simon Megy, and Vadim V Zhdanov

Subjects
Nociceptin receptor, Voltage-dependent calcium channel, Mechanism of action, Opioid, In vivo, Chemistry, Docking (molecular), Analgesic, medicine, medicine.symptom, Pharmacology, Receptor, medicine.drug
Abstract

The number of candidate molecules for new non-narcotic analgesics is extremely limited. Here we report the identification of thiowurtzine, a new potent analgesic molecule with promising application in chronic pain treatment. We describe the chemical synthesis of this unique compound derived from the hexaazaisowurtzitane (CL-20) explosive molecule. Then we use animal experiments to assess its analgesic activity in vivo upon chemical, thermal and mechanical exposures, compared to the effect of several reference drugs. Finally, we investigate the potential receptors of thiowurtzine in order to better understand its complex mechanism of action. We use docking, molecular modeling and molecular dynamics simulations to identify and characterize the potential targets of the drug and confirm the results of the animal experiments. Our findings finally indicate that thiowurtzine may have a complex mechanism of action, by targeting the mu, kappa, delta and ORL1 opioid receptors, and the voltage-gated calcium channels as well.

Published
2021
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96. Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab:subgroup analysis of REGAIN and its extension study

Author

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

Subjects
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.

Published
2021
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97. 21. Efficacy and Safety of Maribavir as a Rescue Treatment for Investigator Assigned Therapy in Transplant Recipients With Refractory or Resistant Cytomegalovirus Infections in the SOLSTICE Study: Phase 3 Trial Results

Author

Pereira, M, Cervera, C, Kotton, C, Sasadeusz, J, Wu, J, Fournier, M, Pereira, M, Cervera, C, Kotton, C, Sasadeusz, J, Wu, J, and Fournier, M

Abstract

Background Refractory or resistant (R/R) cytomegalovirus (CMV) infection after hematopoietic cell transplant (HCT) and solid organ transplant (SOT) cause serious, potentially fatal complications; therapeutic options are limited. In a Phase 3 study (NCT02931539), maribavir (MBV) was superior to investigator-assigned therapy (IAT; val/ganciclovir, foscarnet, cidofovir) for CMV clearance (Wk 8) and clearance plus symptom control (Wk 8 through Wk 16) in HCT/SOT recipients with R/R CMV infections. Here we present further study results on efficacy and safety of MBV in the rescue arm. Methods Patients (pts) were stratified and randomized 2:1 to MBV (400 mg/bid) or IAT for 8-wk treatment then 12-wk follow-up. After minimum 3 wks’ treatment, pts in the IAT group meeting criteria (worsening/lack of improvement of CMV infection or failure to achieve viremia clearance plus IAT intolerance) could enter a MBV rescue arm (8-wk treatment, 12-wk follow-up). In the rescue arm, efficacy was evaluated by confirmed CMV viremia clearance (plasma CMV DNA < 137 IU/mL in 2 consecutive tests ≥ 5 days apart) at end of Wk 8 and confirmed clearance with symptom control at Wk 8 through Wk 16. Safety was assessed. Results A total of 352 pts were randomized (MBV: 235, IAT: 117, randomized set). Confirmed CMV viremia clearance at Wk 8 was achieved in 131 (55.7%) and 28 (23.9%) pts, respectively, in the randomized set. Having met criteria, 22 (18.8%) pts entered the MBV rescue arm; at entry, 6 (27.3%) pts had developed neutropenia and 9 (40.9%) had increased serum creatinine (Table 1). At Wk 8 of rescue therapy, 11 (50.0%) pts achieved confirmed CMV viremia clearance; 6 (27.3%) pts had CMV clearance with symptom control at Wk 8 maintained through Wk 16 (Table 2). All 22 pts reported treatment-emergent adverse events (TEAEs; Table 3); most common TEAEs of special interest were nausea, vomiting, and diarrhea (54.5%), and taste disturbance (50.0%). Neutropenia and acute kidney injury TE

Published
2021

98. Topology predicts long-term functional outcome in early psychosis

Author

Fournier, M, Scolamiero, M, Gholam-Rezaee, MM, Cleusix, M, Jenni, R, Ferrari, C, Golay, P, Baumann, PS, Cuenod, M, Conus, P, Do, KQ, Hess, K, Fournier, M, Scolamiero, M, Gholam-Rezaee, MM, Cleusix, M, Jenni, R, Ferrari, C, Golay, P, Baumann, PS, Cuenod, M, Conus, P, Do, KQ, and Hess, K

Abstract

Early intervention in psychosis is crucial to improving patient response to treatment and the functional deficits that critically affect their long-term quality of life. Stratification tools are needed to personalize functional deficit prevention strategies at an early stage. In the present study, we applied topological tools to analyze symptoms of early psychosis patients, and detected a clear stratification of the cohort into three groups. One of the groups had a significantly better psychosocial outcome than the others after a 3-year clinical follow-up. This group was characterized by a metabolic profile indicative of an activated antioxidant response, while that of the groups with poorer outcome was indicative of oxidative stress. We replicated in a second cohort the finding that the three distinct clinical profiles at baseline were associated with distinct outcomes at follow-up, thus validating the predictive value of this new stratification. This approach could assist in personalizing treatment strategies.

Published
2021

99. Online unsupervised occupancy anticipation system applied to residential heat load management

Author

Rueda, L., Agbossou, K., Henao, N., Kelouwani, S., Oviedo-Cepeda, J. C., Le Lostec, B., Sansregret, S., Fournier, M., Rueda, L., Agbossou, K., Henao, N., Kelouwani, S., Oviedo-Cepeda, J. C., Le Lostec, B., Sansregret, S., and Fournier, M.

Abstract

Human preferences and lifestyles significantly impact buildings' energy consumption. Consequently, a better understanding of occupants' behavior is crucial to decrease energy consumption and maintain occupants' comfort. Occupant-centric control (OCC) strategies are effective approaches to fulfil such a purpose. As such, occupancy detection and prediction are of prime importance, particularly to manage Electric Space Heating (ESH) systems, due to the relatively slow dynamics of the temperature in dwellings. This paper proposes an Explicit Duration Hidden Markov Model (EDHMM) for unsupervised online presence detection and a hazard-based approach for occupancy prediction. Moreover, a control strategy using a cost function, weighted by occupancy predictions, and a load-shifting strategy based on time-varying electricity price are put forward. This work initially validates the consistency of the proposed approach by using synthetic data generated by a Monte Carlo simulation. Subsequently, the performance of our framework is compared with previous methods presented in the literature through experimental validation. Results demonstrate that the proposed EDHMM approach is efficient in detecting occupancy states. Besides, the results of the field implementation show the potential of the proposed control strategy to preserve occupants' thermal comfort while decreasing the heating energy consumption.

Published
2021

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