The terminal complement inhibitor eculizumab was shown to improve myasthenia gravis-related symptoms in the 26-week, phase 3, randomized, double-blind, placebo-controlled REGAIN study (NCT01997229). In this 52-week sub-analysis of the open-label extension of REGAIN (NCT02301624), eculizumab's efficacy and safety were assessed in 11 Japanese and 88 Caucasian patients with anti-acetylcholine receptor antibody-positive refractory generalized myasthenia gravis. For patients who had received placebo during REGAIN, treatment with open-label eculizumab resulted in generally similar outcomes in the Japanese and Caucasian populations. Rapid improvements were maintained for 52 weeks, assessed by change in score from open-label extension baseline to week 52 (mean [standard error]) using the following scales (in Japanese and Caucasian patients, respectively): Myasthenia Gravis Activities of Daily Living (-2.4 [1.34] and - 3.3 [0.651); Quantitative Myasthenia Gravis (-2.9 [1.98] and - 4.3 [0.79]); Myasthenia Gravis Composite (-4.5 [2.63] and - 4.9 [1.19]); and Myasthenia Gravis Quality of Life 15-item questionnaire (-8.6 [5.68) and - 6.5 [1.93]). Overall, the safety of eculizumab was consistent with its known safety profile. In this interim sub-analysis, the efficacy and safety of eculizumab in Japanese and Caucasian patients were generally similar, and consistent with the overall REGAIN population.
Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis. A subgroup analysis of the REGAIN open-label extension study / Murai, Hiroyuki; Uzawa, Akiyuki; Suzuki, Yasushi; Imai, Tomihiro; Shiraishi, Hirokazu; Suzuki, Hidekazu; Okumura, Meinoshin; O'Brien, Fanny; Wang, Jing-Jing; Fujita, Kenji P; Kimiaki, Utsugisawa; Claudio Gabriel Mazia 11, ; Miguel Wilken 11, ; Fabio Barroso 11, ; Juliet Saba 11, ; Marcelo Rugiero 12, ; Mariela Bettini 12, ; Marcelo Chaves 12, ; Gonzalo Vidal 12, ; Alejandra Dalila Garcia 12, ; Jan De Bleecker 13, ; Guy Van den Abeele 13, ; Kathy de Koning 13, ; Katrien De Mey 13, ; Rudy Mercelis 14, ; Délphine Mahieu 14, ; Linda Wagemaekers 14, ; Philip Van Damme 15, ; Annelies Depreitere 16, ; Caroline Schotte 16, ; Charlotte Smetcoren 16, ; Olivier Stevens 16, ; Sien Van Daele 16, ; Nicolas Vandenbussche 16, ; Annelies Vanhee 16, ; Sarah Verjans 16, ; Jan Vynckier 16, ; Ann D'Hondt 16, ; Petra Tilkin 16, ; Alzira Alves de Siqueira Carvalho 17, ; Igor Dias Brockhausen 17, ; David Feder 17, ; Daniel Ambrosio 17, ; Pamela César 17, ; Ana Paula Melo 17, ; Renata Martins Ribeiro 17, ; Rosana Rocha 17, ; Bruno Bezerra Rosa 17, ; Thabata Veiga 17, ; Luiz Augusto da Silva 17, ; Murilo Santos Engel 17, ; Jordana Gonçalves Geraldo 17, ; Maria da Penha Ananias Morita 18, ; Erica Nogueira Coelho 18, ; Gabriel Paiva 18, ; Marina Pozo 18, ; Natalia Prando 18, ; Debora Dada Martineli Torres 18, ; Cristiani Fernanda Butinhao 18, ; Gustavo Duran 18, ; Tamires Cristina Gomes da Silva 18, ; Luiz Otavio Maia Gonçalves 18, ; Lucas Eduardo Pazetto 18, ; Tomás Augusto Suriane Fialho 18, ; Luciana Renata Cubas Volpe 18, ; Luciana Souza Duca 18, ; Acary Souza Bulle Oliveira 19, ; Ana Carolina Amaral Andrade 19, ; Marcelo Annes 19, ; Liene Duarte Silva 19, ; Valeria Cavalcante Lino 19, ; Wladimir Pinto 19, ; Natália Assis 19, ; Fernanda Carrara 19, ; Carolina Miranda 19, ; Iandra Souza 19, ; Patricia Fernandes 19, ; Zaeem Siddiqi 20, ; Cecile Phan 20, ; Jeffrey Narayan 20, ; Derrick Blackmore 20, ; Ashley Mallon 20, ; Rikki Roderus 20, ; Elizabeth Watt 20, ; Jana Junkerova 21, ; Barbora Kurkova 21, ; Katarina Reguliova 21, ; Olga Zapletalova 21, ; Jiri Pitha 22, ; Iveta Novakova 22, ; Michaela Tyblova 22, ; Ivana Jurajdova 22, ; Marcela Wolfova 22, ; Henning Andersen 23, ; Thomas Harbo 23, ; Lotte Vinge 23, ; Susanne Krogh 23, ; Anita Mogensen 23, ; John Vissing 24, ; Joan Højgaard 24, ; Nanna Witting 24, ; Anne Mette Ostergaard Autzen 24, ; Jane Pedersen 24, ; Juha-Pekka Eralinna 25, ; Mikko Laaksonen 25, ; Olli Oksaranta 25, ; Tuula Harrison 25, ; Jaana Eriksson 25, ; Csilla Rozsa 26, ; Melinda Horvath 26, ; Gabor Lovas 26, ; Judit Matolcsi 26, ; Gyorgyi Szabo 26, ; Gedeonne Jakab 26, ; Brigitta Szabadosne 26, ; Antonini, Giovanni; DI PASQUALE, Antonella; Garibaldi, Matteo; Morino, Stefania; Troili, Fernanda; Fionda, Laura; Allessandro Filla 28, ; Teresa Costabile 28, ; Enrico Marano 28, ; Francesco Saccà 28, ; Angiola Fasanaro 28, ; Angela Marsili 28, ; Giorgia Puorro 28, ; Renato Mantegazza 29, ; Carlo Antozzi 29, ; 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Mihoko Takada 35, ; Masako Tsurumaru 35, ; Yumi Yamashita 35, ; Tetsuya Akiyama 36, ; Koichi Narikawa 36, ; Ohito Tano 36, ; Kenichi Tsukita 36, ; Rikako Kurihara 36, ; Fumie Meguro 36, ; Yusuke Fukuda 36, ; Miwako Sato 36, ; Soichiro Funaka 37, ; Tomohiro Kawamura 37, ; Masayuki Makamori 37, ; Masanori Takahashi 37, ; Namie Taichi 37, ; Tomoya Hasuike 37, ; Eriko Higuchi 37, ; Hisako Kobayashi 37, ; Kaori Osakada 37, ; Emiko Tsuda 38, ; Shun Shimohama 38, ; Takashi Hayashi 38, ; Shin Hisahara 38, ; Jun Kawamata 38, ; Takashi Murahara 38, ; Masaki Saitoh 38, ; Shuichiro Suzuki 38, ; Daisuke Yamamoto 38, ; Yoko Ishiyama 38, ; Naoko Ishiyama 38, ; Mayuko Noshiro 38, ; Rumi Takeyama 38, ; Kaori Uwasa 38, ; Ikuko Yasuda 38, ; Anneke van der Kooi 39, ; Marianne de Visser 39, ; Tamar Gibson 39, ; Carlos Casasnovas 40, ; Maria Antonia Alberti Aguilo 40, ; Christian Homedes-Pedret 40, ; Natalia Julia Palacios 40, ; Laura Diez Porras 40, ; Valentina Velez Santamaria 40, ; Ana Lazaro 40, ; Exuperio Diez Tejedor 41, ; 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John Kincaid 57, ; Riley Snook 57, ; Sandra Guingrich 57, ; Angela Micheels 57, ; Vinay Chaudhry 58, ; Andrea Corse 58, ; Betsy Mosmiller 58, ; Andrea Kelley 58, ; Doreen Ho 59, ; Jayashri Srinivasan 59, ; Michal Vytopil 59, ; Jordan Jara 59, ; Nicholas Ventura 59, ; Stephanie Scala 59, ; Cynthia Carter 59, ; Craig Donahue 59, ; Carol Herbert 59, ; Elaine Weiner 59, ; Sharmeen Alam 59, ; Jonathan McKinnon 60, ; Laura Haar 60, ; Naya McKinnon 60, ; Karan Alcon 60, ; Kaitlyn McKenna 60, ; Nadia Sattar 60, ; Kevin Daniels 60, ; Dennis Jeffery 60, ; Miriam Freimer 61, ; Joseph Chad Hoyle 61, ; John Kissel 61, ; Julie Agriesti 61, ; Sharon Chelnick 61, ; Louisa Mezache 61, ; Colleen Pineda 61, ; Filiz Muharrem 61, ; Chafic Karam 62, ; Julie Khoury 62, ; Tessa Marburger 62, ; Harpreet Kaur 62, ; Diana Dimitrova 62, ; James Gilchrist 63, ; Brajesh Agrawal 63, ; Mona Elsayed 63, ; Stephanie Kohlrus 63, ; Angela Andoin 63, ; Taylor Darnell 63, ; Laura Golden 63, ; Barbara Lokaitis 63, ; Jenna Seelback 63, ; 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Richard Barohn 71, ; Mazen Dimachkie 71, ; Melanie Glenn 71, ; April McVey 71, ; Mamatha Pasnoor 71, ; Jeffery Statland 71, ; Yunxia Wang 71, ; Tina Liu 71, ; Kelley Emmons 71, ; Nicole Jenci 71, ; Jerry Locheke 71, ; Alex Fondaw 71, ; Kathryn Johns 71, ; Gabrielle Rico 71, ; Maureen Walsh 71, ; Laura Herbelin 71, ; Charlene Hafer-Macko 72, ; Justin Kwan 72, ; Lindsay Zilliox 72, ; Karen Callison 72, ; Valerie Young 72, ; Beth DiSanzo 72, ; Kerry Naunton 72, ; Michael Benatar 73, ; Martin Bilsker 73, ; Khema Sharma 73, ; Anne Cooley 73, ; Eliana Reyes 73, ; Sara-Claude Michon 73, ; Danielle Sheldon 73, ; Julie Steele 73, ; James Howard Jr 74, ; Chafic Karam 74, ; Manisha Chopra 74, ; Rebecca Traub 74, ; Tuan Vu 75, ; Lara Katzin 75, ; Terry McClain 75, ; Brittany Harvey 75, ; Adam Hart 75, ; Kristin Huynh 75, ; Said Beydoun 76, ; Amaiak Chilingaryan 76, ; Victor Doan 76, ; Brian Droker 76, ; Hui Gong 76, ; Sanaz Karimi 76, ; Frank Lin 76, ; Terry McClain 76, ; Krishna Pokala 76, ; Akshay Shah 76, ; Anh Tran 76, ; Salma Akhter 76, ; Ali Malekniazi 76, ; Rup Tandan 77, ; Michael Hehir 77, ; Waqar Waheed 77, ; Shannon Lucy 77, ; Michael Weiss 78, ; Jane Distad 78, ; Susan Strom 78, ; Sharon Downing 78, ; Bryan Kim 78, ; Richard Nowak 79, ; Daniel Dicapua 79, ; Benison Keung 79, ; Aditya Kumar 79, ; Huned Patwa 79, ; Kimberly Robeson 79, ; Irene Yang 79, ; Joan Nye 79, ; Hong, Vu. - In: JOURNAL OF THE NEUROLOGICAL SCIENCES. - ISSN 0022-510X. - 407:(2019), pp. 1-8. [10.1016/j.jns.2019.08.004]
Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis. A subgroup analysis of the REGAIN open-label extension study
Wang, Jing-Jing;Giovanni AntoniniMembro del Collaboration Group
;Antonella Di PasqualeMembro del Collaboration Group
;Matteo GaribaldiMembro del Collaboration Group
;Stefania MorinoMembro del Collaboration Group
;Fernanda TroiliMembro del Collaboration Group
;Laura FiondaMembro del Collaboration Group
;
2019
Abstract
The terminal complement inhibitor eculizumab was shown to improve myasthenia gravis-related symptoms in the 26-week, phase 3, randomized, double-blind, placebo-controlled REGAIN study (NCT01997229). In this 52-week sub-analysis of the open-label extension of REGAIN (NCT02301624), eculizumab's efficacy and safety were assessed in 11 Japanese and 88 Caucasian patients with anti-acetylcholine receptor antibody-positive refractory generalized myasthenia gravis. For patients who had received placebo during REGAIN, treatment with open-label eculizumab resulted in generally similar outcomes in the Japanese and Caucasian populations. Rapid improvements were maintained for 52 weeks, assessed by change in score from open-label extension baseline to week 52 (mean [standard error]) using the following scales (in Japanese and Caucasian patients, respectively): Myasthenia Gravis Activities of Daily Living (-2.4 [1.34] and - 3.3 [0.651); Quantitative Myasthenia Gravis (-2.9 [1.98] and - 4.3 [0.79]); Myasthenia Gravis Composite (-4.5 [2.63] and - 4.9 [1.19]); and Myasthenia Gravis Quality of Life 15-item questionnaire (-8.6 [5.68) and - 6.5 [1.93]). Overall, the safety of eculizumab was consistent with its known safety profile. In this interim sub-analysis, the efficacy and safety of eculizumab in Japanese and Caucasian patients were generally similar, and consistent with the overall REGAIN population.| File | Dimensione | Formato | |
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