依库珠单抗与利妥昔单抗治疗难治性抗乙酰胆碱受体抗体阳性的全身性肌无力:单中心经验

IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Acta Neurologica Scandinavica Pub Date : 2024-11-14 DOI:10.1155/2024/9924598
Hacer Durmus, Arman Çakar, Yesim Gülşen Parman
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引用次数: 0

摘要

背景:利妥昔单抗(RTX)和依库珠单抗(ECU)是难治性重症肌无力(MG)的治疗选择,但从现实世界中获得的比较临床数据却很有限。在此,我们描述了在本诊所接受ECU和/或RTX治疗的抗乙酰胆碱受体抗体阳性(AChR+)全身性重症肌无力(gMG)患者的基线特征、治疗和安全性结果:伊斯坦布尔医学院神经病学系接受 ECU 或/和 RTX 治疗 1 年以上的难治性 AChR+ gMG 患者被纳入本观察性研究。在征得患者书面同意后,研究人员从病历中回顾性地收集了数据:分析对象包括12名接受ECU治疗的患者和25名接受RTX治疗的患者。两组患者在人口统计学和临床特征(包括MG发病年龄、病程和胸腺瘤病史)方面具有可比性。与RTX相比,ECU的疗效明显更好,具体表现为治疗1个月(p = 0.024)、3个月(p < 0.001)、6个月(p < 0.001)和12个月(p < 0.001);治疗 1 年后的类固醇节省效果(平均 [标准差] 每日泼尼松龙剂量减少 -21.8 毫克 [13.5] 对 RTX 减少 -6.6 毫克 [9.4];p <;0.001);以及治疗期间的抢救治疗需求和肌无力危象发作次数(p <;0.001)。两种治疗方法均未观察到新的安全信号:我们的数据提供了真实世界的证据,证明ECU比RTX更适合治疗难治性AChR+ gMG患者。
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Eculizumab Versus Rituximab for Refractory Antiacetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: A Single-Center Experience

Background: Rituximab (RTX) and eculizumab (ECU) are treatment options for refractory myasthenia gravis (MG), but comparative clinical data derived from real-world experience are limited. Here, we describe the baseline characteristics, treatment, and safety outcomes of patients with antiacetylcholine receptor antibody-positive (AChR+) generalized myasthenia gravis (gMG) treated with ECU and/or RTX in our clinic.

Methods: Patients with refractory AChR+ gMG who received ECU or/and RTX treatment for more than 1 year at the Department of Neurology, Istanbul Faculty of Medicine were included in this observational study. After obtaining written patient consent, data were collected retrospectively from medical records.

Results: Twelve patients treated with ECU and 25 patients treated with RTX were included in the analysis. Groups were comparable with regard to demographic and clinical characteristics, including age at onset of MG, disease duration, and history of thymoma. ECU was associated with significantly better outcomes compared with RTX, as measured by decreases in the mean MG activities of daily living score at 1 (p = 0.024), 3 (p < 0.001), 6 (p < 0.001), and 12 (p < 0.001) months of treatment; steroid-sparing effect after 1 year of treatment (decrease in mean [standard deviation] daily prednisolone dose of −21.8 mg [13.5] vs. −6.6 mg [9.4] with RTX; p < 0.001); and need for rescue treatment and number of myasthenic crisis episodes during treatment (p < 0.001). No new safety signals were observed with either treatment.

Conclusion: Our data provide real-world evidence supporting ECU over RTX to treat patients with refractory AChR+ gMG.

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来源期刊
Acta Neurologica Scandinavica
Acta Neurologica Scandinavica 医学-临床神经学
CiteScore
6.70
自引率
2.90%
发文量
161
审稿时长
4-8 weeks
期刊介绍: Acta Neurologica Scandinavica aims to publish manuscripts of a high scientific quality representing original clinical, diagnostic or experimental work in neuroscience. The journal''s scope is to act as an international forum for the dissemination of information advancing the science or practice of this subject area. Papers in English will be welcomed, especially those which bring new knowledge and observations from the application of therapies or techniques in the combating of a broad spectrum of neurological disease and neurodegenerative disorders. Relevant articles on the basic neurosciences will be published where they extend present understanding of such disorders. Priority will be given to review of topical subjects. Papers requiring rapid publication because of their significance and timeliness will be included as ''Clinical commentaries'' not exceeding two printed pages, as will ''Clinical commentaries'' of sufficient general interest. Debate within the speciality is encouraged in the form of ''Letters to the editor''. All submitted manuscripts falling within the overall scope of the journal will be assessed by suitably qualified referees.
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