Ricciardi Dario, Erra Carmen, Tuccillo Francesco, De Martino Bernardo Maria, Fasolino Alessandra, Habetswallner Francesco
UOC Neurophysiopathology, AORN Cardarelli, Via Antonio Cardarelli 9, Naples, 80131, Italy.
I Division of Neurology and Neurophysiopathology, University of Campania "Luigi Vanvitelli", Naples, Italy.
Neurol Sci. 2025 Feb;46(2):951-959. doi: 10.1007/s10072-024-07861-6. Epub 2024 Nov 4.
Immunosuppressive treatment is effective in most Myasthenia gravis patients, but 10-15% of patients areconsidered refractory due to inadequate response or intolerance to therapy. Eculizumab, a humanized monoclonalantibody directed against C5 complement protein, was approved in Italy to treat Ab-AchR generalized refractoryMG (rMG) in October 2022.
We aim to describe a real-world Italian experience in a population of refractory myasthenia gravis patients with oneyear follow up.
A retrospective data analysis was conducted on patients with refractory generalized MG treated with eculizumabbetween November 2022 and May 2024. Clinical assessment through specific scales (MG ADL - QMG - MGFA -PIS), rescue, and background therapy was recorded after one, three, six, and twelve months.
21 rMG patients were treated with eculizumab with a medium follow up of 10.4 months and 14 patients had at leastone year follow up. A clinically meaningful reduction in total MG-ADL and QMG scores was achieved in the firstmonth. It was maintained throughout the first, third, sixth, and twelfth month along with concomitant reduction ofimmunosuppressive treatments. A drastic reduction of myasthenic exacerbations and crisis was observed duringfollow up and intravenous immunoglobulin treatment was discontinued in all patients except one. The total dailydose of prednisone was significantly reduced.
This single-center real-world study confirmes safety and effectiveness of eculizumab. Eculizumab improved rapidlyall clinical outcome measures, leading to discontinuation of intravenous immunoglobulin treatment and remarkable immunosuppressant-sparing benefits.
免疫抑制治疗对大多数重症肌无力患者有效，但10%-15%的患者因治疗反应不足或不耐受而被认为是难治性的。依库珠单抗是一种针对C5补体蛋白的人源化单克隆抗体，于2022年10月在意大利获批用于治疗抗乙酰胆碱受体（Ab-AchR）全身性难治性重症肌无力（rMG）。
我们旨在描述意大利在难治性重症肌无力患者群体中的真实世界经验，并进行一年的随访。
对2022年11月至2024年5月期间接受依库珠单抗治疗的难治性全身性重症肌无力患者进行回顾性数据分析。在1个月、3个月、6个月和12个月后，通过特定量表（MG日常生活活动量表 - QMG - MGFA - PIS）进行临床评估，并记录急救和背景治疗情况。
21例rMG患者接受了依库珠单抗治疗，中位随访时间为10.4个月，14例患者至少随访了1年。在第一个月，MG-ADL总分和QMG评分出现了具有临床意义的降低。这种降低在第1个月、第3个月、第6个月和第12个月一直保持，同时免疫抑制治疗也相应减少。在随访期间，肌无力加重和危象明显减少，除1例患者外，所有患者均停止了静脉注射免疫球蛋白治疗。泼尼松的每日总剂量显著降低。
这项单中心真实世界研究证实了依库珠单抗的安全性和有效性。依库珠单抗迅速改善了所有临床结局指标，导致静脉注射免疫球蛋白治疗中断，并带来了显著的免疫抑制剂节省效益。
