Rossini Elena, Di Stefano Vincenzo, Iorio Raffaele, Habetswallner Francesco, Maestri Michelangelo, Vinciguerra Claudia, Pennisi Elena Maria, Di Martino Giuseppe, Rini Nicasio, Falso Silvia, Marini Sofia, Ricciardi Dario, Guida Melania, Morino Stefania, Garibaldi Matteo, Leonardi Luca, Marando Demetrio, Tufano Laura, Antonini Giovanni, Fionda Laura
Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), Faculty of Medicine and Psychology, SAPIENZA University of Rome, Sant'Andrea Hospital, Via Di Grottarossa 1035-1039, 00189, Rome, Italy.
Neuromuscular and Rare Disease Centre, Neurology Unit, Sant'Andrea Hospital, Rome, Italy.
J Neurol. 2025 May 14;272(6):396. doi: 10.1007/s00415-025-13127-8.
Ravulizumab, a monoclonal antibody targeting C5, was recently approved for the treatment of anti-AChR positive generalized myasthenia gravis (gMG) patients. The objective of this study is to present the Italian multicenter real-world experience evaluating the safety and efficacy of ravulizumab in gMG within the context of the Expanded Early Access Program (EAP).
We conducted a retrospective study in 7 gMG referral centres in Italy. Demographic and clinical characteristics were recorded at baseline and during follow-up through clinical scale changes including Myasthenia Gravis-Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Composite (MGC). Frequency of minimal symptom expression (MSE) and changes in concomitant medications were also evaluated.
Twenty-four gMG patients (10/24 females) aged between 24 and 82 years (Median 60.5, IQR 52.5-67.5), were included. Fifteen patients had undergone thymectomy, and 14 had a thymoma. Median follow-up duration was 26 weeks (range 10-74, IQR 26-42). MG-ADL and QMG scores showed a significant decrease with respect to baseline (p < 0.001). MSE was achieved by 37.5% patients at the last available follow-up. Tapering of prednisone daily dosage was possible in 76% of patients. Thymoma was significantly associated with QMG score reduction and the frequency of QMG responders at week 2 (p = 0.03). Three patients discontinued treatment. One patient experienced a myasthenic exacerbation and needed rescue therapy. Infectious adverse events were reported in 5/24 patients, and a Stevens-Johnson syndrome in one patient.
Real-world data confirm the effectiveness, safety, and prednisone-sparing effect of ravulizumab in patients with gMG, especially in those with thymoma.
ravulizumab是一种靶向C5的单克隆抗体，最近被批准用于治疗抗乙酰胆碱受体（AChR）阳性的全身型重症肌无力（gMG）患者。本研究的目的是介绍意大利多中心的真实世界经验，即在扩大早期准入计划（EAP）背景下评估ravulizumab治疗gMG的安全性和有效性。
我们在意大利的7个gMG转诊中心进行了一项回顾性研究。通过包括重症肌无力日常生活活动（MG-ADL）、重症肌无力定量评分（QMG）和重症肌无力综合评分（MGC）在内的临床量表变化，在基线和随访期间记录人口统计学和临床特征。还评估了最小症状表现（MSE）的频率和伴随用药的变化。
纳入了24例年龄在24至82岁之间（中位数60.5，四分位间距52.5 - 67.5）的gMG患者（10/24为女性）。15例患者接受了胸腺切除术，14例患有胸腺瘤。中位随访时间为26周（范围10 - 74，四分位间距26 - 42）。MG-ADL和QMG评分相对于基线有显著下降（p < 0.001）。在最后一次可用随访时，37.5%的患者实现了MSE。76%的患者能够减少泼尼松的每日剂量。胸腺瘤与第2周时QMG评分降低及QMG反应者频率显著相关（p = 0.03）。3例患者停止治疗。1例患者发生重症肌无力加重，需要抢救治疗。5/24例患者报告了感染性不良事件，1例患者发生了史蒂文斯-约翰逊综合征。
真实世界数据证实了ravulizumab在gMG患者中，尤其是在胸腺瘤患者中的有效性、安全性和泼尼松节省效应。
