From the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.
Neurology. 2021 Jan 26;96(4):e610-e618. doi: 10.1212/WNL.0000000000011207. Epub 2020 Nov 23.
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.
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.
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.
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.
REGAIN, NCT01997229; REGAIN open-label extension, NCT02301624.
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.
评估依库珠单抗是否有助于乙酰胆碱受体阳性（AChR+）难治性全身性重症肌无力（gMG）患者达到美国重症肌无力基金会（MGFA）干预后最小表现（MM）状态，我们评估了患者在 REGAIN（依库珠单抗治疗 AChR+难治性全身性重症肌无力的安全性和疗效）及其开放标签扩展研究中的状态。
完成 REGAIN 随机对照试验并继续进入开放标签扩展研究的患者纳入本三级终点分析。在 REGAIN 期间的规定时间点和开放标签研究的第 130 周，评估患者的 MGFA 干预后状态，包括改善、不变、恶化、MM 和药物缓解。
共有 117 名患者完成了 REGAIN 并继续进入开放标签研究（依库珠单抗/依库珠单抗：56 例；安慰剂/依库珠单抗：61 例）。在 REGAIN 的第 26 周，依库珠单抗治疗组比安慰剂治疗组有更多的患者达到改善状态（60.7%比 41.7%）或 MM 状态（25.0%比 13.3%；常见比值比：2.3；95%置信区间：1.1-4.5）。在接受依库珠单抗治疗 130 周后，88.0%的患者达到改善状态，57.3%的患者达到 MM 状态。依库珠单抗的安全性与已知的安全性一致，未发现新的安全性信号。
依库珠单抗使 AChR+难治性 gMG 患者迅速且持续地达到 MM 状态。这些发现支持在这一以前难以治疗的患者群体中使用依库珠单抗。
临床试验.gov 标识符：REGAIN，NCT01997229；REGAIN 开放标签扩展，NCT02301624。
这项研究提供了 II 级证据，表明在依库珠单抗治疗 26 周后，25.0%的 AChR+难治性 gMG 成人患者达到 MM，而安慰剂组为 13.3%。
