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IF 3.2 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2025-03-04 DOI:10.1177/22143602241308181

Ali A Habib, Artur Drużdż, Julian Grosskreutz, Renato Mantegazza, Sabrina Sacconi, Kimiaki Utsugisawa, Tuan Vu, John Vissing, Maryam Gayfieva, Irene Pulido-Valdeolivas, Thaïs Tarancón, Franz Woltering, Vera Bril

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引用次数: 0

摘要

背景：全身性肌无力（gMG）是一种罕见的慢性、波动性和异质性自身免疫性疾病，需要终身治疗。MycarinG三期研究表明，对成年重症肌无力患者每周使用6周周期的罗扎尼单抗具有疗效和安全性。开放标签扩展研究表明，在更多的治疗周期中，症状会得到持续改善：介绍罗扎尼珠单抗重复周期治疗的长期安全性的汇总分析结果：方法：汇总3期随机MycarinG研究（NCT03971422）和正在进行的开放标签扩展研究MG0007（NCT04650854）的数据，以评估周期性治疗期间的安全性结果，包括任何治疗突发不良事件（TEAEs）、严重TEAEs、严重TEAEs和导致停药的TEAEs的发生率。另外还对头痛、感染和超敏反应等 TEAE 进行了分析：截至数据截止日（2022年7月8日），MycarinG和MG0007共188名患者接受了≥1个周期的罗扎尼珠单抗治疗；研究总时间为174.71个患者年。总体而言，169/188（89.9%）名患者发生了任何 TEAE：89/188（47.3%）名患者发生了任何头痛（包括偏头痛、先兆偏头痛）；85/188（45.2%）名患者发生了感染；25/188（13.3%）名患者发生了超敏反应。一名患者发生了无菌性脑膜炎。大多数 AE 的强度为轻度至中度，且发生率不会随着反复周期性治疗而增加。共有50/188（26.6%）名患者出现了严重的TEAEs，其中最常见的是罗扎尼单抗7 mg/kg组和罗扎尼单抗10 mg/kg组分别有4/133（3.0%）和7/131（5.3%）名患者的MG恶化，0名和4/131（3.1%）名患者的MG危象，以及1/133（0.8%）和7/131（5.3%）名患者的头痛：这些研究的汇总结果（174.71 个患者年）表明，使用罗扎尼单抗治疗GMG 患者的耐受性良好，在该患者群体中，TEAEs 的发生率一致，且不会随着重复周期的增加而增加。

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Long-term safety of cyclical rozanolixizumab in patients with generalized myasthenia gravis: Results from the Phase 3 MycarinG study and an open-label extension.

Background: Generalized myasthenia gravis (gMG) is a rare, chronic, fluctuating and heterogeneous autoimmune disease requiring lifelong treatment. The Phase 3 MycarinG study demonstrated the efficacy and safety of one 6-week cycle of weekly rozanolixizumab in adult patients with gMG. Open-label extension studies demonstrated consistent symptom improvement over additional treatment cycles.

Objective: To present findings from pooled analyses on the long-term safety of repeated cycles of rozanolixizumab.

Methods: Data from the Phase 3 randomized MycarinG study (NCT03971422) and the ongoing open-label extension study MG0007 (NCT04650854) were pooled to assess safety outcomes during cyclical treatment, including incidence of any treatment-emergent adverse events (TEAEs), severe TEAEs, serious TEAEs and TEAEs leading to discontinuations. Additional analyses were performed for TEAEs, including headache, infections, and hypersensitivity reactions.

Results: At data cutoff (July 8, 2022), a total of 188 patients in MycarinG and MG0007 had received ≥1 treatment cycle with rozanolixizumab; total time in studies was 174.71 patient-years. Overall, 169/188 (89.9%) patients experienced any TEAE: 89/188 (47.3%) experienced any headache (including migraine, migraine with aura); 85/188 (45.2%) experienced an infection; 25/188 (13.3%) experienced a hypersensitivity reaction. One patient experienced an event of aseptic meningitis. The majority of AEs were mild-to-moderate in intensity, and incidence did not increase with repeated cyclic treatment. A total of 50/188 (26.6%) patients experienced severe TEAEs, the most common of which were MG worsening in 4/133 (3.0%) and 7/131 (5.3%) patients in the rozanolixizumab 7 mg/kg and rozanolixizumab 10 mg/kg groups, respectively, MG crisis in 0 and 4/131 (3.1%) patients, and headache in 1/133 (0.8%) and 7/131 (5.3%) patients.

Conclusions: These pooled results, representing 174.71 patient-years in the studies, demonstrate that treatment with rozanolixizumab in patients with gMG was well tolerated, and TEAEs were consistent and did not increase in incidence over repeated cycles in this patient population.

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来源期刊

Journal of neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.