High-Dose Steroids, Treatment Escalation, and Healthcare Burden in Myasthenia Gravis: A US Claims-Based Cohort Study.

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-06-01 Epub Date: 2025-04-30 DOI:10.1007/s40120-025-00714-0

Mar Pujades-Rodríguez, Adam Jedrzejczyk, Jiachen Zhou, Olga Pilipczuk, Thaïs Tarancón

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引用次数: 0

Abstract

Introduction: Myasthenia gravis (MG) is a rare neuromuscular autoimmune disease, characterized by chronic, fluctuating muscle weakness and fatigability. Despite established therapies, many patients have inadequately controlled MG. We describe treatment escalation patterns and outcomes for patients with MG between 2010 and 2018.

Methods: We conducted a retrospective cohort analysis of medical and pharmacy claims data. Patients aged ≥ 18 years at MG diagnosis were included from two US Merative™ MarketScan^® databases. MG treatment escalation was defined as an increase of the administered prednisolone-equivalent corticosteroid dose, use of add-on treatments, or treatment switches.

Results: The cohort included 4925 adults with newly diagnosed MG (1102 aged 18-49 and 3823 aged ≥ 50 at first MG diagnosis) from January 1, 2010 to December 31, 2018. Median follow-up was 30.0 months (18-49 group) and 27.5 months (≥ 50 group). Overall, 71.5% (3521/4925) of patients had ≥ 1 MG treatment escalation; escalation with high-dose corticosteroids was the most common type (64.8% [18-49 group] and 58.5% [≥ 50 group]). In the first year post-diagnosis, median (range) maximal daily prednisolone-equivalent corticosteroid dose was 40.0 (20.0-75.0) mg/day (18-49 group) and 40.0 (20.0-70.0) mg/day (≥ 50 group). Treatment breaks and de-escalation led to rescue treatment or treatment re-escalation in most patients. Exacerbations occurred in 26.3% (n = 290; 18-49 group) and 21.6% (n = 825; ≥ 50 group). Healthcare resource utilization (HCRU) and costs were highest in Year 1, with mean MG-related costs per patient between $24,982 (≥ 50 group) and $33,023 (18-49 group).

Conclusion: The study findings highlight that MG is inadequately controlled in a substantial proportion of patients despite conventional treatment and high reliance on corticosteroids. Earlier treatment with targeted therapies and improved safety profiles may reduce patient burden, HCRU, and costs.

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大剂量类固醇、治疗升级和重症肌无力的医疗负担：一项基于美国索赔的队列研究

重症肌无力（MG）是一种罕见的神经肌肉自身免疫性疾病，以慢性、波动性肌肉无力和疲劳为特征。尽管有成熟的治疗方法，但许多患者对MG的控制不充分。我们描述了2010年至2018年间MG患者的治疗升级模式和结果。方法：我们对医疗和药学索赔数据进行回顾性队列分析。MG诊断时年龄≥18岁的患者纳入了两个美国Merative™MarketScan®数据库。MG治疗升级定义为给予泼尼松龙等效皮质类固醇剂量的增加，使用附加治疗或治疗切换。结果：该队列纳入了2010年1月1日至2018年12月31日期间4925名新诊断为MG的成年人（1102名年龄在18-49岁，3823名年龄≥50岁）。中位随访时间分别为30.0个月（18-49岁组）和27.5个月（≥50岁组）。总体而言，71.5%（3521/4925）的患者治疗升级≥1mg；高剂量皮质类固醇的升级是最常见的类型（64.8%[18-49组]和58.5%[≥50组]）。在诊断后的第一年，每日强的松龙等效皮质类固醇剂量的中位（范围）最大值为40.0 (20.0-75.0)mg/天（18-49组）和40.0 (20.0-70.0)mg/天（≥50组）。治疗中断和降级导致大多数患者的抢救治疗或治疗再升级。病情加重26.3% (n = 290；18 ~ 49岁组),21.6% (n = 825；≥50组)。医疗资源利用率（HCRU）和成本在第一年最高，每位患者的mg相关平均成本在24,982美元（≥50组）和33,023美元（18-49组）之间。结论：研究结果强调，尽管常规治疗和对皮质类固醇的高度依赖，很大一部分患者的MG仍未得到充分控制。早期使用靶向治疗和改进的安全性可以减轻患者负担、HCRU和成本。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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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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