Pujades-Rodríguez Mar, Jedrzejczyk Adam, Zhou Jiachen, Pilipczuk Olga, Tarancón Thaïs
UCB, Brussels, Belgium.
UCB, Warsaw, Poland.
Neurol Ther. 2025 Jun;14(3):1061-1082. doi: 10.1007/s40120-025-00714-0. Epub 2025 Apr 30.
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.
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.
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).
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.
重症肌无力（MG）是一种罕见的神经肌肉自身免疫性疾病，其特征为慢性、波动性肌肉无力和易疲劳性。尽管已有既定的治疗方法，但许多患者的重症肌无力仍未得到充分控制。我们描述了2010年至2018年间重症肌无力患者的治疗升级模式及治疗结果。
我们对医疗和药房理赔数据进行了一项回顾性队列分析。纳入了来自美国两个麦睿博™市场扫描数据库中年龄≥18岁的重症肌无力确诊患者。重症肌无力治疗升级的定义为泼尼松等效皮质类固醇给药剂量增加、使用附加治疗或更换治疗方案。
该队列包括2010年1月1日至2018年12月31日期间新确诊的4925例成年重症肌无力患者（首次重症肌无力诊断时1102例年龄在18 - 49岁，3823例年龄≥50岁）。中位随访时间为30.0个月（18 - 49岁组）和27.5个月（≥50岁组）。总体而言，71.5%（3521/4925）的患者有≥1次重症肌无力治疗升级；大剂量皮质类固醇升级是最常见的类型（18 - 49岁组为64.8%，≥50岁组为58.5%）。在确诊后的第一年，泼尼松等效皮质类固醇的最大日剂量中位数（范围）为40.0（20.0 - 75.0）mg/天（18 - 49岁组）和40.0（20.0 - 70.0）mg/天（≥50岁组）。治疗中断和降级导致大多数患者需要抢救治疗或治疗再次升级。18 - 49岁组26.3%（n = 290）和≥50岁组21.6%（n = 825）出现病情加重。医疗资源利用（HCRU）和成本在第1年最高，每位患者的平均重症肌无力相关成本在24,982美元（≥50岁组）至33,023美元（18 - 49岁组）之间。
研究结果突出表明，尽管采用了传统治疗方法且高度依赖皮质类固醇，但仍有相当一部分患者的重症肌无力未得到充分控制。早期采用靶向治疗并改善安全性可能会减轻患者负担、降低医疗资源利用和成本。
