Background In the phase III eculizumab for refractory generalized myasthenia gravis REGAIN study [ClinicalTrials.gov identifier: NCT01997229] and its open-label extension (OLE) [ClinicalTrials.gov identifier: NCT02301624], patients with treatment-refractory antiacetylcholine receptor antibody-positive generalized myasthenia gravis had clinically meaningful improvements with eculizumab versus placebo. This subgroup analysis evaluated data from patients with a recent history of chronic intravenous immunoglobulin (IVIg) use before study entry. Methods The subgroup comprised patients who had received IVIg at least four times in 1 year, with at least one IVIg treatment cycle during the 6 months before the first REGAIN study dose. Data from REGAIN and the OLE were analyzed. Response to eculizumab versus placebo was assessed using four validated, disease-specific measures. Incidences of exacerbations and safety endpoints were recorded. Results The subgroup had similar patient and disease characteristics as the overall REGAIN population. Clinical assessments showed sustained eculizumab efficacy during REGAIN and the OLE over 18 months. Patients receiving placebo in REGAIN experienced rapid improvements in assessment scores when treated with eculizumab in the OLE. There was a lower rate of disease exacerbations with eculizumab than with placebo during REGAIN, and eculizumab was well tolerated. Conclusion Eculizumab treatment, compared with placebo, results in meaningful clinical improvements and fewer disease exacerbations for patients who previously received chronic IVIg. Trial registration REGAIN [ClinicalTrials.gov identifier: NCT01997229]; REGAIN open-label extension [ClinicalTrials.gov identifier: NCT02301624].

中文翻译：

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最近接受慢性 IVIg 治疗的重症肌无力患者对依库珠单抗的反应：REGAIN 的亚组分析及其开放标签扩展研究。

背景 在依库珠单抗治疗难治性全身重症肌无力 REGAIN 研究 [ClinicalTrials.gov 标识符：NCT01997229] 及其开放标签扩展 (OLE) [ClinicalTrials.gov 标识符：NCT02301624] 中，难治性抗乙酰胆碱受体抗体阳性全身与安慰剂相比，依库珠单抗对重症肌无力有临床意义的改善。该亚组分析评估了进入研究前近期使用慢性静脉注射免疫球蛋白 (IVIg) 病史的患者的数据。方法 该亚组包括在 1 年内至少接受过四次 IVIg 的患者，在第一次 REGAIN 研究剂量之前的 6 个月内至少有一个 IVIg 治疗周期。分析了来自 REGAIN 和 OLE 的数据。对依库珠单抗与安慰剂的反应使用四个经过验证的，针对疾病的措施。记录恶化的发生率和安全终点。结果 该亚组具有与总体 REGAIN 人群相似的患者和疾病特征。临床评估显示，在 REGAIN 和 OLE 期间，依库珠单抗疗效持续超过 18 个月。在 REGAIN 中接受安慰剂的患者在 OLE 中接受依库珠单抗治疗时评估分数迅速提高。在 REGAIN 期间，依库珠单抗的疾病恶化率低于安慰剂，并且依库珠单抗的耐受性良好。结论 与安慰剂相比，依库珠单抗治疗对先前接受慢性 IVIg 的患者产生了有意义的临床改善和更少的疾病恶化。试验注册 REGAIN [ClinicalTrials.gov 标识符：NCT01997229]；REGAIN 开放标签扩展 [临床试验。