TY - JOUR
T1 - Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis
T2 - A subgroup analysis of the REGAIN open-label extension study
AU - REGAIN Study Group
AU - Murai, Hiroyuki
AU - Uzawa, Akiyuki
AU - Suzuki, Yasushi
AU - Imai, Tomihiro
AU - Shiraishi, Hirokazu
AU - Suzuki, Hidekazu
AU - Okumura, Meinoshin
AU - O'Brien, Fanny
AU - Wang, Jing-Jing
AU - Fujita, Kenji P
AU - Utsugisawa, Kimiaki
AU - Jacob, Saiju
N1 - Copyright © 2019 The Authors. Published by Elsevier B.V. All rights reserved.
PY - 2019/12/15
Y1 - 2019/12/15
N2 - 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 [0.65]); 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.
AB - 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 [0.65]); 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.
KW - Adult
KW - Aged
KW - Antibodies, Monoclonal, Humanized/adverse effects
KW - Asian Continental Ancestry Group
KW - Complement Inactivating Agents/adverse effects
KW - Female
KW - Humans
KW - Japan
KW - Male
KW - Middle Aged
KW - Myasthenia Gravis/drug therapy
KW - Treatment Outcome
U2 - 10.1016/j.jns.2019.08.004
DO - 10.1016/j.jns.2019.08.004
M3 - Article
C2 - 31698177
SN - 0022-510X
VL - 407
SP - 116419
JO - Journal of the Neurological Sciences
JF - Journal of the Neurological Sciences
ER -