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5. Edoxaban for Thromboembolism Prevention in Pediatric Patients With Cardiac Disease

Author

Brothers, Matthew, Marino, Bradley, Canter, Charles, Law, Mark, Nguyen, Nguyenvu, Sang, Charlie, Shimano, Kristin, Gupta, Dipankar, Portman, Michael, Williams, Derek, Glass, Lauren, Sperrazza, Charles, Herold, Steven, Garg, Ruchira, Vranicar, Mark, Awad, Sawsan, Asante-Korang, Alfred, Druzgal, Colleen, Ozment, Caroline, Del Toro, Kamill, Roses, Ferran, Jux, Christian, Gravenhorst, Verena, Schweigmann, Ulrich, Bhatt, Mihir, Sabapathy, Christine, Dahdah, Nagib, Bartonicek, Dototea, Tulzer, Gerald, Basargina, Elena, Zvereva, Tatiana, Pertels, Tatiana, Plotnikova, Irina, Pierre-Emmanuel, S.E.G.U.E.L.A., Amedro, Pascal, Yves, Dulac, BONNET, Damien, Saraco, Paola, Rimini, Alessandro, Digtiar, Valerii, Gonchar, Margaryta, Kryuchko, Tetyana, Yablon, Olga, Bedi, Varinder Singh, Patel, Jashvant, Mitra, Monjori, Kusa, Jacek, Domagala, Kowalczyk, KÖRNYEI, László, BERECZKI, Csaba, ABLONCZY, László, Levitas, Vivianne Aviva, Mishali, David, Revel-Vilk, Shoshana, Harlev, Dan, Sasmaz, Hatice Ilgen, Ozbek, Namik Yasar, Unal, Sule, Patıroglu, Türkan, Malbora, Baris, Agin, Hasan, Karakas, Zeynep, Kavakli, Ramazan Kaan, Chalmers, Elizabeth, Bu'Lock, Frances, Daubeney, Piers, Hamza, Hala, Badr, Mohamed, Elalfy, Mohsen, Mansour, Ahmed, Hassab, Hoda, Sabry, Ayman, Daou, Linda, Bitar, Fadi, Portman, Michael A., Jacobs, Jeffrey P., Newburger, Jane W., Berger, Felix, Grosso, Michael A., Duggal, Anil, Tao, Ben, and Goldenberg, Neil A.

Published
2022
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9. EBV-driven lymphoid neoplasms associated with pediatric ALL maintenance therapy

Author

Elitzur, Sarah, Vora, Ajay, Burkhardt, Birgit, Inaba, Hiroto, Attarbaschi, Andishe, Baruchel, Andre, Escherich, Gabriele, Gibson, Brenda, Liu, Hsi-Che, Loh, Mignon, Moorman, Anthony V., Moricke, Anja, Pieters, Rob, Uyttebroeck, Anne, Baird, Susan, Bartram, Jack, Barzilai-Birenboim, Shlomit, Batra, Sandeep, Ben-Harosh, Miriam, Bertrand, Yves, Buitenkamp, Trudy, Caldwell, Kenneth, Drut, Ricardo, Geerlinks, Ashley V., Gilad, Gil, Grainger, John, Haouy, Stephanie, Heaney, Nicholas, Huang, Mary, Ingham, Danielle, Krenova, Zdenka, Kuhlen, Michaela, Lehrnbecher, Thomas, Manabe, Atsushi, Niggli, Felix, Paris, Claudia, Revel-Vilk, Shoshana, Rohrlich, Pierre, Sinno, Mohamad G., Szczepanski, Tomasz, Tamesberger, Melanie, Warrier, Rajasekharan, Wolfl, Matthias, Nirel, Ronit, Izraeli, Shai, Borkhardt, Arndt, and Schmiegelow, Kjeld

Published
2022
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10. Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry

Author

Deegan, Patrick, primary, Lau, Heather, additional, Elstein, Deborah, additional, Fernandez-Sasso, Diego, additional, Giraldo, Pilar, additional, Hughes, Derralynn, additional, Zimran, Ari, additional, Istaiti, Majdolen, additional, Gadir, Noga, additional, Botha, Jaco, additional, and Revel-Vilk, Shoshana, additional

Published
2024
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11. Genetic backgrounds and clinical characteristics of congenital neutropenias in Israel

Author

Yeshareem, Lital, primary, Yacobovich, Joanne, additional, Lebel, Asaf, additional, Noy‐Lotan, Sharon, additional, Dgany, Orly, additional, Krasnov, Tanya, additional, Berger Pinto, Galit, additional, Oniashvili, Nino, additional, Mardoukh, Jacques, additional, Bielorai, Bella, additional, Laor, Ruth, additional, Mandel‐Shorer, Noa, additional, Ben Barak, Ayelet, additional, Levin, Carina, additional, Asleh, Mahdi, additional, Miskin, Hagit, additional, Revel‐Vilk, Shoshana, additional, Levin, Dror, additional, Benish, Marganit, additional, Zuckerman, Tsila, additional, Wolach, Ofir, additional, Pazgal, Idit, additional, Brik Simon, Dafna, additional, Gilad, Oded, additional, Yanir, Asaf David, additional, Goldberg, Tracie Alison, additional, Izraeli, Shai, additional, Tamary, Hannah, additional, and Steinberg‐Shemer, Orna, additional

Published
2024
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12. A mutation in POLR3E impairs antiviral immune response and RNA polymerase III

Author

Ramanathan, Aravind, Weintraub, Michael, Orlovetskie, Natalie, Serruya, Raphael, Mani, Dhivakar, Marcu, Orly, Stepensky, Polina, Weisblum, Yiska, Djian, Esther, Shaag, Avraham, Revel-Vilk, Shoshana, Fried, Iris, Kotler, Moshe, Rouvinski, Alex, Wolf, Dana, Elpeleg, Orly, and Jarrous, Nayef

Published
2020

13. Dabigatran etexilate for the treatment of acute venous thromboembolism in children (DIVERSITY): a randomised, controlled, open-label, phase 2b/3, non-inferiority trial

Author

Nurmeev, Ildar, Safina, Asiya, Zapletal, Ondrej, Brandão, Leonardo R, Kuhn, Tomas, Votava, Tomas, Felgenhauer, Judy, Sharathkumar, Anjali, Svirin, Pavel, Amid, Ali, Halton, Jacqueline, Gorbatikov, Kirill, Saracco, Paola, Kiss, Csongor, Halimeh, Susan, Reschke, Madlen, Wulff, Beate, David, Michele, Novak, Zbynek, Trunina, Inna, Albisetti, Manuela, Frisk, Tony, Glosli, Heidi, Groll, Andreas, Lvova, Olga, Sasmaz, Ilgen, Sosothikul, Darintr, Zilinskaite, Virginija, Cockrell, Erin, Digtyar, Valeriy, Hadacova, Ivana, Palmu, Sauli, Pawar, Anjali, Annichino Bizzacchi, Joyce Maria, Caliskan, Umran, Celkan, Tiraje, Dmytriiev, Dmytro, Druzgal, Colleen Harkins, Onelda Elena, Graciela, Kattamis, Antonis, Kavakli, Ramazan Kaan, Male, Christoph, Ozdemir, Nihal, Van Damme, An, Zvereva, Tatiana, Aarli, Aanen, Paredes Aguilera, Rogelio Alejandro, Aytac, Selin, Carneiro, Jorge, Chistolini, Antonio, Mazzucconi, Maria Gabriela, Corrales-Medina, Fernando, Couturaud, Francis, Croteau, Stacey E, Trenor III, Cameron, Damgaard, Michael, Dixon, Natalia, Galustyan, Anna, Hak, Jiri, Hoffmann, Marianne, Kupesiz, Alphan, Labarque, Veerle, van Geet, Christel, Lin, Ming-Chih, Fu, Yun-Ching, Loggetto, Sandra, Mondelaers, Veerle, Odri-Komazec, Irena, Revel-Vilk, Shoshana, Sevilla, Julian, Fuzzato Silva, Luciano, Kerr Saraiva, José, Montes Tapia, Fernando Felix, Woods-Swafford, Wendy, Luciani, Matteo, Bomgaars, Lisa, Chalmers, Elizabeth, Mitchell, Lesley G, Tartakovsky, Igor, Simetzberger, Monika, Huang, Fenglei, Sun, Zhichao, Kreuzer, Jörg, Gropper, Savion, Reilly, Paul, and Brueckmann, Martina

Published
2021
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14. Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry.

Author

Elstein, Deborah, Belmatoug, Nadia, Bembi, Bruno, Deegan, Patrick, Fernandez-Sasso, Diego, Giraldo, Pilar, Göker-Alpan, Özlem, Hughes, Derralynn, Lau, Heather, Lukina, Elena, Revel-Vilk, Shoshana, Schwartz, Ida Vanessa D., Istaiti, Majdolen, Botha, Jaco, Gadir, Noga, Schenk, Jörn, and Zimran, Ari

Subjects
MEDICAL registries, ENZYME replacement therapy, GAUCHER'S disease, GLOBAL method of teaching, ESOPHAGEAL varices, PLATELET count
Abstract

Background: Long-term patient registries are important for evaluating treatment outcomes in patients with rare diseases, and can provide insights into natural disease history and progression in real-world clinical practice. Initiated in 2010, the Gaucher Outcome Survey (GOS) is an ongoing, international, multicenter, observational registry (ClinicalTrials.gov Identifier: NCT03291223) for patients with a diagnosis of Gaucher disease (GD), irrespective of treatment type or status, with a primary objective to monitor safety and long-term effectiveness of velaglucerase alfa. Methods: Here, we evaluated the GOS population 12 years after the registry initiation. Results: As of 25 February 2023, 2084 patients enrolled in the GOS and 1643 received GD-specific treatment. Patients exhibited broad heterogeneity at baseline: age of diagnosis (0 to 85.3 years), hemoglobin concentrations (<80.0 g/L to >150 g/L), platelet counts (<50 × 109/L to >450 × 109/L), and liver and spleen volumes. Most patients treated with enzyme replacement therapy or substrate reduction therapy reported improvements in clinical parameters within 1 year of treatment initiation, maintained over the course of treatment up to 12 years, whereas untreated patients had baseline values closer to standard reference thresholds and showed stability over time. Conclusion: The 12-year data from the GOS confirm the impact of long-term treatment with GD-specific agents and offer insights into disease progression and outcomes in a real-world setting. [ABSTRACT FROM AUTHOR]

Published
2024
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17. Rivaroxaban compared with standard anticoagulants for the treatment of acute venous thromboembolism in children: a randomised, controlled, phase 3 trial

Author

Molinari, Angelo C., Nowak Göttl, Ulrike, Chain, Juan, Robertson, Jeremy, Thom, Katharina, Streif, Werner, Schwarz, Rudolf, Schmitt, Klaus, Grangl, Gernot, Van Damme, An, Maes, Philip, Labarque, Veerle, Petrilli, Antonio, Loggeto, Sandra, Azeka, Estela, Brandao, Leonardo, Le, Doan, Sabapathy, Christine, Giordano, Paola, Wu, Runhui, Ding, Jie, Huang, Wenyan, Mao, Jianhua, Lähteenmäki, Päivi, Amedro, Pascal, Decramer, Stephane, Bernig, Toralf, Chada, Martin, Chan, Godfrey, Kally, Krisztian, Nolan, Beatrice, Revel-Vilk, Shoshana, Tamary, Hannah, Levin, Carina, Tormene, Daniela, Abbattista, Maria, Artoni, Andrea, Ikeyama, Takanari, Inuzuka, Ryo, Yasukochi, Satoshi, Morales Soto, Michelle, Solis Labastida, Karina A, Suijker, Monique H, Bartels, Marike, Tamminga, Rienk Y, Van Ommen, C Heleen, Te Loo, D. Maroeska, Anjos, Rui, Zubarovskaya, Lyudmila, Popova, Natalia, Samochatova, Elena, Belogurova, Margarita, Svirin, Pavel, Shutova, Tatiana, Lebedev, Vladimir, Barbarash, Olga, Koh, Pei L, Mei, Joyce C, Podracka, Ludmila, Berrueco, Ruben, Fernandez, Maria F, Frisk, Tony, Grunt, Sebastian, Rischewski, Johannes, Albisetti-Pedroni, Manuela, Antmen, Ali, Tokgoz, Huseyin, Karakas, Zeynep, Chalmers, Elizabeth, Motwani, Jayashree, Williams, Michael, Grainger, John, Payne, Jeanette, Richards, Mike, Baird, Susan, Bhatnagar, Neha, Aramburo, Angela, Crary, Shelley, Wynn, Tung, Carpenter, Shannon, Hege, Kerry, Ahuja, Sanjay, Goldenberg, Neil, Woods, Gary, Godder, Kamar, Scott-Emuakpor, Ajovi, Roach, Gavin, Raffini, Leslie, Shah, Nirmish, Shah, Sanjay, Thornburg, Courtney, Zia, Ayesha, Berkow, Roger, Male, Christoph, Lensing, Anthonie W A, Palumbo, Joseph S, Kumar, Riten, Nurmeev, Ildar, Bonnet, Damien, Connor, Philip, Hooimeijer, Hélène L, Torres, Marcela, Chan, Anthony K C, Kenet, Gili, Holzhauer, Susanne, Santamaría, Amparo, Simioni, Paolo, Bhat, Rukhmi V, Yee, Donald L, Lvova, Olga, Beyer-Westendorf, Jan, Biss, Tina T, Martinelli, Ida, Saracco, Paola, Peters, Marjolein, Kállay, Krisztián, Gauger, Cynthia A, Massicotte, M Patricia, Young, Guy, Pap, Akos F, Majumder, Madhurima, Smith, William T, Heubach, Jürgen F, Berkowitz, Scott D, Thelen, Kirstin, Kubitza, Dagmar, Crowther, Mark, Prins, Martin H, and Monagle, Paul

Published
2020
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22. Diagnostic high-throughput sequencing of 2396 patients with bleeding, thrombotic, and platelet disorders

Author

Downes, Kate, Megy, Karyn, Duarte, Daniel, Vries, Minka, Gebhart, Johanna, Hofer, Stefanie, Shamardina, Olga, Deevi, Sri V.V., Stephens, Jonathan, Mapeta, Rutendo, Tuna, Salih, Al Hasso, Namir, Besser, Martin W., Cooper, Nichola, Daugherty, Louise, Gleadall, Nick, Greene, Daniel, Haimel, Matthias, Martin, Howard, Papadia, Sofia, Revel-Vilk, Shoshana, Sivapalaratnam, Suthesh, Symington, Emily, Thomas, Will, Thys, Chantal, Tolios, Alexander, Penkett, Christopher J., Ouwehand, Willem H., Abbs, Stephen, Laffan, Michael A., Turro, Ernest, Simeoni, Ilenia, Mumford, Andrew D., Henskens, Yvonne M.C., Pabinger, Ingrid, Gomez, Keith, and Freson, Kathleen

Published
2019
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23. Bodyweight-adjusted rivaroxaban for children with venous thromboembolism (EINSTEIN-Jr): results from three multicentre, single-arm, phase 2 studies

Author

Monagle, Paul, Robertson, Jeremy, Grangl, Gernot, Male, Christoph, Streif, Werner, Carniero, Jorge, Logetto, Sandra, Brandao, Leonardo, Halton, Jacqueline, Amedro, Pascal, Bonnet, Damien, Beyer-Westendorf, Jan, Dietrich, Sven, Holzhauer, Susanne, Nowak-Göttl, Ulrike, Kallay, Krisztian, Kenet, Gili, Revel-Vilk, Shoshana, Tamary, Hannah, Giordano, Paola, Abbattista, Maria, Artoni, Andrea, Molinari, Angelo C, Saracco, Paola, Simioni, Paolo, Tormene, Daniela, Ono, Hiroshi, Te Loo, Maroeska WM, Van Ommen, Heleen, Veening, Margreet A, Suiker, Monique H, Peters, Marjolein, Samochatova, Elena, Shamardina, Anastasia, Novgorod, Nizhny, Zubarovskaya, Lyudmila, Dasi, Maria A, Elorza, Maria, Santamaria, Amparo, Sánchez-Luna, Manuel, Albisetti, Manuela, Grunt, Sebastian, Kavakli, Kaan, Biss, Tina, Connor, Philip, Williams, Mike, Acharya, Suchitra, Bhat, Rumi, Kearney, Susan, Kumar, Riten, Patel, Kavita, Rajpukar, Madvi, Schwartz, Jeffrey H, Vik, Terry, Wynn, Tung, Yee, Donald L, Young, Guy, Lensing, Anthonie W A, Thelen, Kirstin, Martinelli, Ida, Santamaría, Amparo, Connor, Phillip, Kapsa, Stefanie, Willmann, Stefan, Pap, Akos F, Becka, Michael, Twomey, Teresa, Prins, Martin H, and Kubitza, Dagmar

Published
2019
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24. Rapid home therapy infusion of velaglucerase alfa in naïve patients with Gaucher disease.

Author

Becker‐Cohen, Michal, Revel‐Vilk, Shoshana, Frydman, Dafna, Dinur, Tama, Tiomkin, Maayan, Istaiti, Majdolen, Arbel, Naama, Bauer, Peter, Cozma, Claudia, Rolfs, Arndt, Szer, Jeff, and Zimran, Ari

Subjects
*HOME care services, *QUESTIONNAIRES, *CLINICAL trials, *GAUCHER'S disease, *DESCRIPTIVE statistics, *DRUG dosage, *INFUSION therapy, *INTRAVENOUS therapy, *LONGITUDINAL method, *DRUG efficacy, *QUALITY of life, *HEALTH outcome assessment, *GLYCOSIDASES, *BIOMARKERS
Abstract

Background: Enzyme replacement therapy (ERT) has revolutionised the management of patients with Gaucher disease (GD). In 2018, we published the safety and efficacy of rapid 10‐min infusion of velaglucerase alfa in previously treated patients, mostly on low‐dose therapy. Aim: To improve quality of life (QoL) for patients needing lifelong bi‐weekly infusions by introducing a 10‐min infusion instead of 1 h per label in patients naive to ERT and on high‐dose therapy. Methods: Fifteen naive patients were enrolled; all received bi‐weekly infusions of 60 units/kgBW velaglucerase alfa; the infusion rate was gradually reduced in the hospital, followed by home infusions. Each infusion was followed for safety. Efficacy parameters were assessed every 3 months. Patient‐reported outcome questionnaires were collected at baseline and follow‐up. Results: Ten‐minute rapid infusions were well tolerated without related severe adverse events (SAEs). Two patients experienced a non‐related SAE and another a possibly related AE. In three patients, the infusion rate was increased to 30 or 60 min (two because of suboptimal response and one because of AE). Two patients dropped out because of an unwillingness to attend follow‐up visits during the COVID‐19 pandemic. All 13 remaining patients reached the 24‐month end‐point. The platelet counts increased by a median (range) of 68.38% (12.5–300%) and the lyso‐Gb1 levels decreased by 62.6% (32.9–89.9%). Conclusion: Home therapy with rapid infusion of high‐dose velaglucerase alfa was a safe, effective and preferable alternative for patients with GD naïve to treatment. We believe that shortening the infusion time improves the QoL of patients with GD who have a lifelong commitment to intravenous therapy. [ABSTRACT FROM AUTHOR]

Published
2024
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26. Erratum to ‘Illustrated State-of-the-Art Capsules of the ISTH 2024 Congress’ [Research and Practice in Thrombosis and Haemostasis Volume 8, Issue 4, May 2024, 102432]

Author

Ward, Chris, Curry, Nicola, El-Ekiaby, Magdy, Jurk, Kerstin, Versteeg, Henri H., Keragala, Charithani, Burstyn-Cohen, Tal, Antoniak, Silvio, Suzuki, Yuko, Baker, Ross I., Christophe, Olivier, Revel-Vilk, Shoshana, Hart, Alice, Deppermann, Carsten, Tran, Huyen, Pozzi, Nicola, Kahr, Walter H.A., Grover, Steven P., Wenzel, Philip, Brown, Ashley C., Oury, Cécile, Shea, Susan M., Fredenburgh, James, Passam, Freda H., Winearls, James, Moore, Hunter B., Tole, Soumitra, Merriman, Eileen, Barnes, Geoffrey D., Leonardo Liu, Z., Sholzberg, Michelle, Rivera, José, and Marín-Quilez, Ana

Published
2024
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28. Illustrated State-of-the-Art Capsules of the ISTH 2024 Congress

Author

Ward, Chris, Curry, Nicola, El-Ekiaby, Magdy, Jurk, Kerstin, Versteeg, Henri H., Keragala, Charithani, Burstyn-Cohen, Tal, Antoniak, Silvio, Suzuki, Yuko, Baker, Ross I., Christophe, Olivier, Revel-Vilk, Shoshana, Hart, Alice, Deppermann, Carsten, Tran, Huyen, Pozzi, Nicola, Kahr, Walter H.A., Grover, Steven P., Wenzel, Philip, Brown, Ashley C., Oury, Cécile, Shea, Susan M., Fredenburgh, James, Passam, Freda H., Winearls, James, Moore, Hunter B., Tole, Soumitra, Merriman, Eileen, Barnes, Geoffrey D., Liu, Z. Leonardo, and Sholzberg, Michelle

Published
2024
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30. Insights into the Value of Lyso-Gb1 as a Predictive Biomarker in Treatment-Naïve Patients with Gaucher Disease Type 1 in the LYSO-PROOF Study

Author

Curado, Filipa, primary, Rösner, Sabine, additional, Zielke, Susanne, additional, Westphal, Gina, additional, Grittner, Ulrike, additional, Skrahina, Volha, additional, Alasel, Mohammed, additional, Malik, Ahmad Mehmood, additional, Beetz, Christian, additional, Böttcher, Tobias, additional, Barel, Gal, additional, Sah, Ashish Prasad, additional, Dinur, Tama, additional, Anjum, Nadeem, additional, Ichraf, Quidad, additional, Kriouile, Yamna, additional, Hadipour, Zahra, additional, Hadipour, Fatemeh, additional, Revel-Vilk, Shoshana, additional, Cozma, Claudia, additional, Hartkamp, Jörg, additional, Cheema, Huma, additional, Zimran, Ari, additional, Bauer, Peter, additional, and Rolfs, Arndt, additional

Published
2023
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35. Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on “Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158”

Author

Hughes, Derralynn A., primary, Deegan, Patrick, additional, Giraldo, Pilar, additional, Göker-Alpan, Özlem, additional, Lau, Heather, additional, Lukina, Elena, additional, Revel-Vilk, Shoshana, additional, Scarpa, Maurizio, additional, Botha, Jaco, additional, Gadir, Noga, additional, and Zimran, Ari, additional

Published
2023
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43. EBV-driven lymphoid neoplasms associated with pediatric ALL maintenance therapy

Author

Elitzur, Sarah, Vora, Ajay, Burkhardt, Birgit, Inaba, Hiroto, Attarbaschi, Andishe, Baruchel, Andre, Escherich, Gabriele, Gibson, Brenda, Liu, Hsi Che, Loh, Mignon, Moorman, Anthony V., Möricke, Anja, Pieters, Rob, Uyttebroeck, Anne, Baird, Susan, Bartram, Jack, Barzilai-Birenboim, Shlomit, Batra, Sandeep, Ben-Harosh, Miriam, Bertrand, Yves, Buitenkamp, Trudy, Caldwell, Kenneth, Drut, Ricardo, Geerlinks, Ashley V., Gilad, Gil, Grainger, John, Haouy, Stephanie, Heaney, Nicholas, Huang, Mary, Ingham, Danielle, Krenova, Zdenka, Kuhlen, Michaela, Lehrnbecher, Thomas, Manabe, Atsushi, Niggli, Felix, Paris, Claudia, Revel-Vilk, Shoshana, Rohrlich, Pierre, Sinno, Mohamad G., Szczepanski, Tomasz, Tamesberger, Melanie, Warrier, Rajasekharan, Wolfl, Matthias, Nirel, Ronit, Izraeli, Shai, Borkhardt, Arndt, Schmiegelow, Kjeld, Elitzur, Sarah, Vora, Ajay, Burkhardt, Birgit, Inaba, Hiroto, Attarbaschi, Andishe, Baruchel, Andre, Escherich, Gabriele, Gibson, Brenda, Liu, Hsi Che, Loh, Mignon, Moorman, Anthony V., Möricke, Anja, Pieters, Rob, Uyttebroeck, Anne, Baird, Susan, Bartram, Jack, Barzilai-Birenboim, Shlomit, Batra, Sandeep, Ben-Harosh, Miriam, Bertrand, Yves, Buitenkamp, Trudy, Caldwell, Kenneth, Drut, Ricardo, Geerlinks, Ashley V., Gilad, Gil, Grainger, John, Haouy, Stephanie, Heaney, Nicholas, Huang, Mary, Ingham, Danielle, Krenova, Zdenka, Kuhlen, Michaela, Lehrnbecher, Thomas, Manabe, Atsushi, Niggli, Felix, Paris, Claudia, Revel-Vilk, Shoshana, Rohrlich, Pierre, Sinno, Mohamad G., Szczepanski, Tomasz, Tamesberger, Melanie, Warrier, Rajasekharan, Wolfl, Matthias, Nirel, Ronit, Izraeli, Shai, Borkhardt, Arndt, and Schmiegelow, Kjeld

Abstract

The development of a second malignancy after the diagnosis of childhood acute lymphoblastic leukemia (ALL) is a rare event. Certain second malignancies have been linked with specific elements of leukemia therapy, yet the etiology of most second neoplasms remains obscure and their optimal management strategies are unclear. This is a first comprehensive report of non-Hodgkin lymphomas (NHL) following pediatric ALL therapy, excluding stem-cell transplantation. We analyzed data of patients who developed NHL following ALL diagnosis and were enrolled in 12 collaborative pediatric ALL trials between 1980-2018. Eighty-five patients developed NHL, with mature B-cell lymphoproliferations as the dominant subtype (56 of 85 cases). Forty-six of these 56 cases (82%) occurred during or within 6 months of maintenance therapy. The majority exhibited histopathological characteristics associated with immunodeficiency (65%), predominantly evidence of Epstein-Barr virus–driven lymphoproliferation. We investigated 66 cases of post-ALL immunodeficiency-associated lymphoid neoplasms, 52 from our study and 14 additional cases from a literature search. With a median follow-up of 4.9 years, the 5-year overall survival for the 66 patients with immunodeficiency-associated lymphoid neoplasms was 67.4% (95% confidence interval [CI], 56-81). Five-year cumulative risks of lymphoid neoplasm– and leukemia-related mortality were 20% (95% CI, 10.2-30) and 12.4% (95% CI, 2.7-22), respectively. Concurrent hemophagocytic lymphohistiocytosis was associated with increased mortality (hazard ratio, 7.32; 95% CI, 1.62-32.98; P =.01). A large proportion of post-ALL lymphoid neoplasms are associated with an immunodeficient state, likely precipitated by ALL maintenance therapy. Awareness of this underrecognized entity and pertinent diagnostic tests are crucial for early diagnosis and optimal therapy.

Published
2023

44. A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders

Author

Simeoni, Ilenia, Stephens, Jonathan C., Hu, Fengyuan, Deevi, Sri V.V., Megy, Karyn, Bariana, Tadbir K., Lentaigne, Claire, Schulman, Sol, Sivapalaratnam, Suthesh, Vries, Minka J.A., Westbury, Sarah K., Greene, Daniel, Papadia, Sofia, Alessi, Marie-Christine, Attwood, Antony P., Ballmaier, Matthias, Baynam, Gareth, Bermejo, Emilse, Bertoli, Marta, Bray, Paul F., Bury, Loredana, Cattaneo, Marco, Collins, Peter, Daugherty, Louise C., Favier, Rémi, French, Deborah L., Furie, Bruce, Gattens, Michael, Germeshausen, Manuela, Ghevaert, Cedric, Goodeve, Anne C., Guerrero, Jose A., Hampshire, Daniel J., Hart, Daniel P., Heemskerk, Johan W.M., Henskens, Yvonne M.C., Hill, Marian, Hogg, Nancy, Jolley, Jennifer D., Kahr, Walter H., Kelly, Anne M., Kerr, Ron, Kostadima, Myrto, Kunishima, Shinji, Lambert, Michele P., Liesner, Ri, López, José A., Mapeta, Rutendo P., Mathias, Mary, Millar, Carolyn M., Nathwani, Amit, Neerman-Arbez, Marguerite, Nurden, Alan T., Nurden, Paquita, Othman, Maha, Peerlinck, Kathelijne, Perry, David J., Poudel, Pawan, Reitsma, Pieter, Rondina, Matthew T., Smethurst, Peter A., Stevenson, William, Szkotak, Artur, Tuna, Salih, van Geet, Christel, Whitehorn, Deborah, Wilcox, David A., Zhang, Bin, Revel-Vilk, Shoshana, Gresele, Paolo, Bellissimo, Daniel B., Penkett, Christopher J., Laffan, Michael A., Mumford, Andrew D., Rendon, Augusto, Gomez, Keith, Freson, Kathleen, Ouwehand, Willem H., and Turro, Ernest

Published
2016
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45. The extended phenotype of LPS-responsive beige-like anchor protein (LRBA) deficiency

Author

Gámez-Díaz, Laura, August, Dietrich, Stepensky, Polina, Revel-Vilk, Shoshana, Seidel, Markus G., Noriko, Mitsuiki, Morio, Tomohiro, Worth, Austen J.J., Blessing, Jacob, Van de Veerdonk, Frank, Feuchtinger, Tobias, Kanariou, Maria, Schmitt-Graeff, Annette, Jung, Sophie, Seneviratne, Suranjith, Burns, Siobhan, Belohradsky, Bernd H., Rezaei, Nima, Bakhtiar, Shahrzad, Speckmann, Carsten, Jordan, Michael, and Grimbacher, Bodo

Published
2016
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46. Real-World Experiences with Taliglucerase Alfa Home Infusions for Patients with Gaucher Disease: A Global Cohort Study.

Author

Revel-Vilk, Shoshana, Mansfield, Royston, Feder-Krengel, Neta, Machtiger-Azoulay, Noya, Kuter, David, Szer, Jeff, Rosenbaum, Hanna, Ferreira, David Cavalcanti, Ruhrman-Shahar, Noa, Wajnrajch, Michael, and Zimran, Ari

Subjects
*GAUCHER'S disease, *ENZYME replacement therapy, *COHORT analysis, *BLINKING (Physiology), *CHEST pain, *HOME safety
Abstract

Taliglucerase alfa is an enzyme replacement therapy approved for Gaucher disease. We assessed the duration/compliance/safety of such home infusions in commercial use in four countries where home infusion programs are available. The treatment duration/compliance study included 173 patients (Israel, 58; US, 61; Brazil, 48; Australia, 6) who received ≥1 taliglucerase alfa home infusion through 6/2021. The median age at home therapy initiation was 38 (range, 2–87) years; 58% were females. The median treatment duration (at home) was 2.7 (range, 0.04–9.0) years. The annual compliance rate was stable (≥95%) throughout the study period. A search of the Pfizer global safety database (through 6/2021), identified 19 adverse events (AEs) as related to "definite home use" and 14 to "possible home use" of taliglucerase alfa; 42.4% of these AEs were serious; none were fatal. Twelve serious AEs in five separate case reports were considered treatment related: one case of chest discomfort/pain and hypertension and one case of erythema associated with a toe blister, for which causality could not be excluded; pain in extremity; projectile vomiting and chills, alongside excessive eye blinking; and an infusion-related AE (pruritus). In conclusion, this real-life global study demonstrated that taliglucerase alfa home infusions are safe with high compliance rates. [ABSTRACT FROM AUTHOR]

Published
2023
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47. EBV-driven lymphoid neoplasms associated with pediatric ALL maintenance therapy

Author

Elitzur, Sarah, primary, Vora, Ajay, additional, Burkhardt, Birgit, additional, Inaba, Hiroto, additional, Attarbaschi, Andishe, additional, Baruchel, Andre, additional, Escherich, Gabriele, additional, Gibson, Brenda, additional, Liu, Hsi-Che, additional, Loh, Mignon, additional, Moorman, Anthony V., additional, Möricke, Anja, additional, Pieters, Rob, additional, Uyttebroeck, Anne, additional, Baird, Susan, additional, Bartram, Jack, additional, Barzilai-Birenboim, Shlomit, additional, Batra, Sandeep, additional, Ben-Harosh, Miriam, additional, Bertrand, Yves, additional, Buitenkamp, Trudy, additional, Caldwell, Kenneth, additional, Drut, Ricardo, additional, Geerlinks, Ashley V., additional, Gilad, Gil, additional, Grainger, John, additional, Haouy, Stephanie, additional, Heaney, Nicholas, additional, Huang, Mary, additional, Ingham, Danielle, additional, Krenova, Zdenka, additional, Kuhlen, Michaela, additional, Lehrnbecher, Thomas, additional, Manabe, Atsushi, additional, Niggli, Felix, additional, Paris, Claudia, additional, Revel-Vilk, Shoshana, additional, Rohrlich, Pierre, additional, Sinno, Mohamad G., additional, Szczepanski, Tomasz, additional, Tamesberger, Melanie, additional, Warrier, Rajasekharan, additional, Wolfl, Matthias, additional, Nirel, Ronit, additional, Izraeli, Shai, additional, Borkhardt, Arndt, additional, and Schmiegelow, Kjeld, additional

Published
2023
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