학술논문
Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study.
Document Type
Academic Journal
Author
Murai H; Department of Neurology, International University of Health and Welfare, Tokyo, Japan. Electronic address: murai@iuhw.ac.jp.; Uzawa A; Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan. Electronic address: auzawa@chiba-u.jp.; Suzuki Y; Department of Neurology, National Hospital Organization Sendai Medical Center, Sendai, Japan.; Imai T; Department of Neurology, Sapporo Medical University Hospital, Sapporo, Japan. Electronic address: toimai@sapmed.ac.jp.; Shiraishi H; Department of Neurology, Nagasaki University Hospital, Nagasaki, Japan.; Suzuki H; Department of Neurology, Kindai University Hospital, Osaka, Japan.; Okumura M; Osaka Toneyama Medical Center, Toyonaka, Japan. Electronic address: meinosin@toneyama.go.jp.; O'Brien F; Alexion Pharmaceuticals, Boston, MA, United States. Electronic address: fanny.obrien@alexion.com.; Wang JJ; Formerly of Alexion Pharmaceuticals, Boston, MA, United States.; Fujita KP; Formerly of Alexion Pharmaceuticals, Boston, MA, United States.; Utsugisawa K; Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan. Electronic address: kutsugi@s4.dion.ne.jp.
Source
Publisher: Elsevier Country of Publication: Netherlands NLM ID: 0375403 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-5883 (Electronic) Linking ISSN: 0022510X NLM ISO Abbreviation: J Neurol Sci Subsets: MEDLINE
Subject
Language
English
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 [0.65]); 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.
(Copyright © 2019 The Authors. Published by Elsevier B.V. All rights reserved.)
(Copyright © 2019 The Authors. Published by Elsevier B.V. All rights reserved.)