Real-world Treatment Patterns Among Patients with Generalized Myasthenia Gravis in the United States.

IF 4.7 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2026-10-06 DOI:10.1007/s40120-026-01040-9

Kavita Grover, Martin Cloutier, Geoffroy Coteur, Porpong Boonmak, Antoine C El Khoury, Maryia Zhdanava, Anabelle Tardif-Samson, Yuxi Wang, Kavita Gandhi

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引用次数: 0

Abstract

Introduction: Conventional treatments for generalized myasthenia gravis (gMG) include acetylcholinesterase inhibitors (AChEi), systemic corticosteroids, and non-steroidal immunosuppressants; standard of care for exacerbation or myasthenic crisis includes immunoglobulin and plasmapheresis; newer advanced treatments include B-cell depleting agents, complement-5 inhibitors (C5i), and neonatal Fc receptor (FcRn) blockers. This study described real-world treatment patterns with currently available therapies and prevalence of myasthenia gravis (MG)-related clinical events (exacerbations or crises) among patients with gMG in the US.

Methods: Adults with gMG were identified from Komodo Research Data (January 1, 2017-February 28, 2025). Index date was the first MG diagnosis by a neurologist. Patients were followed for ≥ 12 months post-index. Treatment sequencing was characterized by individual classes and broader treatment types (conventional treatments, advanced immunotherapies, immunoglobulin). A treatment episode was defined based on treatment changes (discontinuation, switch, augmentation/add-on, or drop), with any remaining or newly initiated/added treatment marking a new episode. Prevalence of MG-related clinical events was also described.

Results: A total of 8759 patients were included (mean age: 61.5 years; 48.7% female; mean follow-up: 36.5 months). From the first (n = 7986) to fifth (n = 3340) treatment episode, AChEi use declined (80.3→57.6%), systemic corticosteroid use was consistent (49.0→63.0%), and use increased for non-steroidal immunosuppressants (12.7→40.2%), immunoglobulin (6.1→14.5%), rituximab (0.3→2.1%), C5i (0.3→3.4%), and FcRn blockers (0.3→3.8%). Conventional treatments were most frequently used in all episodes; advanced treatment use increased progressively from the first to fifth episodes, with initiation averaging 13.6-19.0 months post-diagnosis. Overall, 48.6% of patients experienced MG-related clinical events during follow-up, usually within the first year following gMG diagnosis (39.9%).

Conclusion: Conventional treatments were the predominant initial strategy for gMG, while immunoglobulin and advanced treatments became more common over successive treatment episodes. The high prevalence of MG-related clinical events within the first year post-gMG diagnosis, despite treatment with conventional therapies, suggests the need for more effective and earlier interventions.

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美国广泛性重症肌无力患者的现实世界治疗模式

简介：全身性重症肌无力（gMG）的常规治疗包括乙酰胆碱酯酶抑制剂（AChEi）、全身皮质类固醇和非甾体免疫抑制剂；加重或肌无力危象的标准治疗包括免疫球蛋白和血浆置换；较新的高级治疗包括b细胞消耗剂、补体-5抑制剂（C5i）和新生儿Fc受体（FcRn）阻滞剂。本研究描述了美国重症肌无力（MG）相关临床事件（恶化或危象）的流行情况，以及当前可用的治疗方法的现实世界治疗模式。方法：从Komodo研究数据（2017年1月1日- 2025年2月28日）中鉴定成人gMG。索引日期是神经科医生第一次诊断为MG。患者随访≥12个月。治疗排序的特点是个体分类和更广泛的治疗类型（常规治疗，高级免疫治疗，免疫球蛋白）。治疗发作的定义基于治疗变化（停药、切换、强化/附加治疗或下降），任何剩余或新开始/添加的治疗都标志着新的发作。mg相关临床事件的发生率也被描述。结果：共纳入8759例患者，平均年龄61.5岁，女性48.7%，平均随访36.5个月。从第一次治疗（n = 7986）到第5次治疗（n = 3340）， AChEi的使用下降（80.3→57.6%），全身皮质类固醇的使用保持一致（49.0→63.0%），非甾体免疫抑制剂（12.7→40.2%）、免疫球蛋白（6.1→14.5%）、利妥昔单抗（0.3→2.1%）、C5i（0.3→3.4%）和FcRn阻滞剂（0.3→3.8%）的使用增加。常规治疗在所有发作中最常用；晚期治疗的使用从第一次到第五次发作逐渐增加，平均在诊断后13.6-19.0个月开始。总体而言，48.6%的患者在随访期间经历了mg相关的临床事件，通常发生在gMG诊断后的第一年（39.9%）。结论：常规治疗是gMG的主要初始治疗策略，而免疫球蛋白和高级治疗在连续治疗中更为常见。尽管接受了常规治疗，但在gmg诊断后的第一年，mg相关临床事件的发生率很高，这表明需要更有效和更早的干预措施。

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来源期刊

Neurology and Therapy CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

8.10%

发文量

103

审稿时长

6 weeks

期刊介绍： Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Neurology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research. Rapid Publication The journal’s rapid publication timelines aim for a peer review decision within 2 weeks of submission. If an article is accepted, it will be published online 3-4 weeks from acceptance. These rapid timelines are achieved through the combination of a dedicated in-house editorial team, who closely manage article workflow, and an extensive Editorial and Advisory Board who assist with rapid peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model, this allows for the rapid and efficient communication of the latest research and reviews to support scientific discovery and clinical practice. Open Access All articles published by Neurology and Therapy are open access. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning that authors will always have a personal point of contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. We also encourage pre-submission enquiries and are always happy to provide a confidential assessment of manuscripts. 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