Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Alexion Pharmaceuticals, Boston, MA.
Ann Clin Transl Neurol. 2020 Aug;7(8):1327-1339. doi: 10.1002/acn3.51121. Epub 2020 Jul 22.
To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups.
Patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open-label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open-label extension were analyzed.
Of the 125 patients who participated in REGAIN, 117 enrolled in the open-label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open-label extension.
Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.
评估末端补体抑制剂依库珠单抗是否改善了难治性抗乙酰胆碱受体抗体阳性全身型重症肌无力患者的患者和医生报告的结局（分别通过重症肌无力日常生活活动量表和定量重症肌无力量表评估），该研究评估了四个领域的结果，代表眼肌、球肌、呼吸肌和肢体/大运动肌肉群。
在 REGAIN 研究（NCT01997229）中，将难治性抗乙酰胆碱受体抗体阳性全身型重症肌无力患者以 1:1 的比例随机分配至接受安慰剂或依库珠单抗治疗组。完成 REGAIN 研究的患者有资格继续参加开放标签扩展试验（NCT02301624），最长可达 4 年。分析了整个 REGAIN 研究期间以及开放标签扩展的 130 周内记录的重症肌无力日常生活活动量表和定量重症肌无力量表的每个重症肌无力日常生活活动量表的四个领域评分和定量重症肌无力量表的四个领域评分。
在参加 REGAIN 的 125 名患者中，有 117 名患者参加了开放标签扩展试验；其中 61 名患者在 REGAIN 期间接受安慰剂治疗，56 名患者接受依库珠单抗治疗。依库珠单抗治疗使患者的总评分和重症肌无力日常生活活动量表和定量重症肌无力量表的所有四个领域评分均迅速改善。这些改善在开放标签扩展的 130 周内得以维持。
依库珠单抗治疗可迅速且持续改善难治性抗乙酰胆碱受体抗体阳性全身型重症肌无力患者的眼肌、球肌、呼吸肌和肢体/大运动肌肉群的肌肉力量以及相关的日常活动能力。
