Accès ouvert déclaré
2020
article
Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
Renato Mantegazza, Gil I. Wolfe, Srikanth Muppidi, Heinz Wiendl, Kenji P. Fujita, Fanny O’Brien, Heather Booth, James F. Howard, Claudio Mazia, Miguel Wilken, Fábio 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 Souto 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, Érica Nogueira Coelho, Gabriel Pina Paiva, M Tovar Pozo, Natália 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 Hünning Pinto, Aline de Cassia Santos, Acary Souza Bullé Oliveira, Ana Carolina Amaral de Andrade, Marcelo Annes, Liene Duarte Silva, Valéria Cavalcante Lino, Wladimir Bocca Vieira de Rezende Pinto, Natália Assis, Fernanda Carrara, Carolina Miranda, Iandra Souza, Zaeem A. Siddiqi, Cecile Phan, Jeffrey Narayan, Derrick Blackmore, Ashley Mallon, Rikki Roderus, Elizabeth Watt, Stanislav Voháňka, Josef Bednařík, Magda Chmelikova, Marek Čierny, Stanislava Toncrova, Jana Junkerová, Barbora Kurková, Katarína Reguliová, Olga Zapletalová, Jiří Piťha, Iveta Nováková, Michaela Týblová, 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 Rózsa, Melinda Horvath, Gábor Lovas, Judit Matolcsi, Gedeonne Jakab, G Szabó, Brigitta Szabadosne, László Vécsei, Lívia Dézsi, Edina Varga, Monika Konyane, Giovanni Antonini, Antonella Di Pasquale, Matteo Garibaldi, Stefania Morino, Fernanda Troili, Laura Fionda, Amelia Evoli, Paolo Emilio Alboini, Valentina Damato, Raffaele Iorio, Maurizio Inghilleri, Vittorio Frasca, Elena Giacomelli, Maria Cristina Gori, Diego Lopergolo, Emanuela Onesti, Maria Gabriele, Francesco Saccà, Alessandro Filla, Teresa Costabile, Enrico Marano, Angiola Maria Fasanaro, Angela Marsili, Giorgia Puorro, Carlo Antozzi, Silvia Bonanno, Giorgia Camera, Alberta Locatelli, Lorenzo Maggi, Maria Barbara 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, Shu-Ichiro Suzuki, Daisuke Yamamoto, Yoko Ishiyama, Naoko Ishiyama, Mayuko Noshiro, Rumi Takeyama, Kaori Uwasa, Ikuko Yasuda, Anneke J. 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, Ji‐Eun Lee, Da Yoon Koh, Juyoung Kwon, SangAe Park, Eun Haw Choi, Yoon‐Ho Hong, Sohyun Ahn, Dae Lim Koo, Jae‐Sung Lim, Chaewon Shin, Ji Ye Hwang, Miri Kim, Seung Min Kim, Ha-Neul Jeong, Jin-Woo Jung, Yool-hee Kim, Hyung‐Seok Lee, Ha Young Shin, Eun Bi Hwang, Miju Shin, Carlos Casasnovas, Maria Antònia Albertí Aguiló, Christian Homedes-Pedret, Natalia Juliá Palacios, Laura Diez Porras, Valentina Vélez-Santamaría, A. M. Lázaro, Josep Gamez Carbonell, Pilar Suñé, Maria Salvadó Figueras, Gisela Gili, Gonzalo Mazuela, Isabel Illa, Elena Cortés Vicente, Jordi Díaz‐Manera, Luis Antonio Querol Gutiérrez, Ricardo Rojas Garcia, Núria Vidal, Elisabet Arribas-Ibar, Exuperio Díez‐Tejedor, Pilar Gomez Salcedo, Mireya Fernández-Fournier, Pedro Lopez Ruiz, Francisco Javier Rodríguez de Rivera Garrido, María Sastre, Fredrik Piehl, Albert Hietala, Lena Bjarbo, İhsan Şükrü Şengün, Arzu Meherremova, Pınar Özçelik, Bengu Balkan, Celal Tuga, Müzeyyen Ugur, Sevim Erdem‐Özdamar, Can Ebru Bekircan‐Kurt, Nazire Pınar Acar, Ezgi Yılmaz, Yagmur Caliskan, Gulsah Orsel, Hüsnü Efendi, S. Aydınlık, Hakan Çavuş, Ayşe Kutlu, Gulsah Becerikli, Cansu Semiz, Ozlem Tun, Murat Terzi, Baki Doğan, Musa Onar, Sedat Şen, 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 J. Silvestri, Kara Patrick, Karen Zakalik, Jonathan Katz, Robert G. Miller, Marguerite Engel, Dallas Forshew, Elena Bravver, Benjamin Brooks, Mohammed Sanjak, Sarah Plevka, Maryanne Burdette, Scott Cunningham, Megan Kramer, Joanne Nemeth, Clara Schommer, Scott Tierney, Vern C. Juel, Jeffrey T. Guptill, Lisa D. Hobson‐Webb, Janice M. Massey, Kate Beck, Donna Carnes, John Loor, Amanda Anderson, Robert M. Pascuzzi, Cynthia Bodkin, John Kincaid, Sandra Guingrich, Angela Micheels, Vinay Chaudhry, Andrea M. 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, J. Chad Hoyle, John T. Kissel, Julie Agriesti, Sharon Chelnick, Louisa Mezache, Colleen Pineda, Filiz Muharrem, Chafic Karam, Julie Khoury, Tessa Marburger, Harpreet Kaur, Diana Dimitrova, James M. Gilchrist, Brajesh Agrawal, Mona Elsayed, Stephanie Kohlrus, Angela Ardoin, Taylor Darnell, Laura Golden, Barbara Lokaitis, J. D. Seelbach, Neelam Goyal, Sarada Sakamuri, Yuen T. So, S. Paulose, Sabrina Pol, Lesly Welsh, Ratna Bhavaraju‐Sanka, Alejandro Tobon Gonzalez, Lorraine Dishman, Floyd Jones, Anna González, Patricia Padilla, Amy R. Saklad, Marcela Silva, Sharon Nations, Jaya Trivedi, Steve Hopkins, Mohamed Kazamel, Mohammad Alsharabati, Lu Liang, Kenkichi Nozaki, Sandi Mumfrey-Thomas, Amy Woodall, Tahseen Mozaffar, Tiyonnoh Cash, Namita Goyal, Gulmohor Roy, Veena Mathew, Fatima Maqsood, Brian Minton, Helen Jones, Jeffrey Rosenfeld, Rebekah Garcia, Laura Echevarria, Sonia García, Michael Pulley, Shachie Aranke, Alan Ross Berger, Jaimin Shah, Yasmeen Shabbir, Lisa Smith, Mary Varghese, Laurie Gutmann, Ludwig Gutmann, Nivedita U. Jerath, Christopher Nance, Andrea Swenson, Heena Olalde, Nicole Kressin, Jeri Sieren, Richard J. Barohn, Mazen M. Dimachkie, Melanie Glenn, April McVey, Mamatha Pasnoor, Yunxia Wang, Tina Liu, Kelley Emmons, Nicole Jenci, Jerry Locheke, Alex Fondaw, Kathryn Johns, Gabrielle Rico, Maureen Walsh, Laura Herbelin, Charlene E. Hafer‐Macko, Justin Kwan, Lindsay Zilliox, Karen Callison, Valerie Young, Beth DiSanzo, Kerry Naunton, Michael Benatar, Martin Bilsker, Khema R. 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 R. Beydoun, Amaiak Chilingaryan, Victor Doan, Brian Droker, Hui Gong, Sanaz Karimi, Frank Y. Lin, Krishna Polaka, Akshay Shah, Anh Thu Tran, Salma Akhter, Ali Malekniazi, Rup Tandan, Michael K. Hehir, Waqar Waheed, S. Deborah Lucy, Michael D. Weiss, Jane Distad, Susan Strom, Sharon Downing, Bryan Kim, Tulio E. Bertorini, Thomas D. Arnold, Kendrick Q. Henderson, Rekha Pillai, Ye Liu, Lauren Wheeler, Jasmine Hewlett, Mollie Vanderhook, Richard J. Nowak, Daniel DiCapua, Benison Keung, Aditya Kumar, Huned Patwa, Kimberly Robeson, Irene Yang, Joan Nye, Hong Vu
80Citations signalées, ce qui n’est pas une note de qualité
7Institutions déclarées
3Pays d’affiliation déclarés
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Le résumé fourni par la source
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. CLINICALTRIALSGOV IDENTIFIER: REGAIN, NCT01997229; REGAIN open-label extension, NCT02301624. CLASSIFICATION OF EVIDENCE: This 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.
Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.
Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
- Date Crossref
- 26/01/2021
- Éditeur
- Ovid Technologies (Wolters Kluwer Health)
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Les institutions déclarées
Une affiliation ne permet pas de déduire la nationalité d’un auteur.
Les sujets associés
Myasthenia Gravis and ThymomaCoagulation, Bradykinin, Polyphosphates, and AngioedemaAdrenal Hormones and Disorders