Howard James F, Dodig Dubravka, Zeinali Lida, Macwan Samir P, Yegin Ashley, Pulley Michael T, Rodrigues Ema, Narayanaswami Pushpa
The University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC, USA.
The University of Toronto, Toronto, ON, Canada.
J Neurol Sci. 2025 Sep 15;476:123628. doi: 10.1016/j.jns.2025.123628. Epub 2025 Jul 16.
Complement component 5 inhibitors are indicated for the treatment of anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG). Clinical trials have demonstrated improved functional ability, muscle strength, and quality of life (QOL) in patients treated with eculizumab or ravulizumab. Evidence for their effectiveness and safety in clinical practice is reported here.
Data from the global MG SPOTLIGHT Registry were collected from patients with gMG currently or previously treated with eculizumab or ravulizumab in routine clinical practice. Effectiveness was evaluated among patients with ≥3 outcome measurements using MG Activities of Daily Living (MG-ADL) and MG-QOL 15-revised (MG-QOL15r) patient-reported outcomes and/or Myasthenia Gravis Foundation of America Clinical Classification (MGFA class) physician assessments. Safety data were also assessed.
189 patients were treated with eculizumab. Mean (95% CI) MG-ADL scores (n=110) decreased by 4.2 (-5.0, -3.4) points and MG-QOL15r scores (n=40) decreased by 7.5 (-9.9, -5.0) points from before eculizumab initiation to first assessment during/after treatment (median eculizumab treatment duration: 18.0 months). 45/103 (43.7%) patients were MGFA class 0-II before eculizumab versus 89/103 (86.4%) at first assessment. After transitioning to ravulizumab, improvements in MG-ADL scores (n=37), MG-QOL15r scores (n=14), and MGFA class (n=34) were maintained (median ravulizumab treatment duration: 8.7-11.4 months). Few treatment-related serious adverse events (eculizumab: Aspergillus infection, nervous system disorder [n=1 each]; ravulizumab: anaphylactic reaction [n=1]) and no meningococcal infections were reported with eculizumab or ravulizumab treatment.
In routine clinical practice, eculizumab was well tolerated and effective for patients with gMG. Treatment benefits were maintained after transition to ravulizumab.
补体成分5抑制剂被用于治疗抗乙酰胆碱受体抗体阳性的全身型重症肌无力（gMG）。临床试验已证明，接受依库珠单抗或瑞武利单抗治疗的患者功能能力、肌肉力量和生活质量（QOL）有所改善。本文报告了它们在临床实践中的有效性和安全性证据。
从全球重症肌无力聚焦注册研究中收集数据，这些数据来自在常规临床实践中目前或以前接受过依库珠单抗或瑞武利单抗治疗的gMG患者。使用重症肌无力日常生活活动（MG-ADL）和重症肌无力生活质量15项修订版（MG-QOL15r）患者报告结局和/或美国重症肌无力基金会临床分类（MGFA分级）医生评估，对有≥3项结局测量的患者进行有效性评估。同时也评估了安全性数据。
189例患者接受了依库珠单抗治疗。从依库珠单抗开始治疗到治疗期间/治疗后的首次评估，平均（95%CI）MG-ADL评分（n=110）下降了4.2（-5.0，-3.4）分，MG-QOL15r评分（n=40）下降了7.5（-9.9，-5.0）分（依库珠单抗治疗的中位持续时间：18.0个月）。在依库珠单抗治疗前，103例患者中有45例（43.7%）为MGFA 0-II级，而在首次评估时为89例（86.4%）。转为瑞武利单抗治疗后，MG-ADL评分（n=37）、MG-QOL15r评分（n=14）和MGFA分级（n=34）的改善得以维持（瑞武利单抗治疗的中位持续时间：8.7-11.4个月）。依库珠单抗或瑞武利单抗治疗报告的与治疗相关的严重不良事件很少（依库珠单抗：曲霉菌感染、神经系统疾病[各1例]；瑞武利单抗：过敏反应[1例]），且未报告脑膜炎球菌感染。
在常规临床实践中，依库珠单抗对gMG患者耐受性良好且有效。转为瑞武利单抗治疗后，治疗益处得以维持。
