Journal of neurology · 2025
Ravulizumab for generalized Myasthenia Gravis: a multicenter real-life experience.
СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод пока не подготовлен
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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
Аннотация
INTRODUCTION: 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).
METHODS: 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.
RESULTS: 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.
CONCLUSIONS: Real-world data confirm the effectiveness, safety, and prednisone-sparing effect of ravulizumab in patients with gMG, especially in those with thymoma.
METHODS: 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.
RESULTS: 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.
CONCLUSIONS: Real-world data confirm the effectiveness, safety, and prednisone-sparing effect of ravulizumab in patients with gMG, especially in those with thymoma.