Buzzard Katherine A, Van der Walt Anneke, Butzkueven Helmut, Zhang Wen Wen, Barnett-Tapia Carolina, Cutter Gary, Heckmann Jeannine M, Farr Pamela, Tran Carolyn, Sartori Charlotte, Stupar Dusko, More Rein, Sim Linda, Le Alison, Tang Qingxiao, Reddel Stephen W
Eastern Health Clinical School, Monash University, Box Hill, Victoria, Australia.
Royal Melbourne Hospital, Department of Neurosciences, Parkville, Victoria, Australia.
Muscle Nerve. 2025 Sep;72(3):424-432. doi: 10.1002/mus.28450. Epub 2025 Jun 4.
INTRODUCTION/AIMS: Patient registries are valuable tools for outcomes research in rare diseases such as myasthenia gravis (MG). Existing MG registries are limited by factors including a lack of geographical scope. MGBase has been designed as a global, observational registry aimed at studying clinical practice outcomes in MG. METHODS: MGBase was developed with the support of the independent MSBase Foundation. An international scientific leadership group (SLG) established a minimum dataset and outcome measures. Data are entered on a purpose-designed platform in real time and held in a web-based registry. Members can request access to the global dataset for investigator-driven substudies. RESULTS: MGBase data collection commenced in October 2021. From inception until April 2024, 565 patients from 16 clinics and 8 countries were enrolled. The cohort is 56% female, with a mean age of 57 (SD19) years at the last visit and a median disease duration of 5 (IQR 1.8, 10.8) years. Seventy-six percent of patients are acetylcholine receptor antibody positive (AChR ab+) and 7% have antibodies to muscle-specific kinase (MuSK ab+). At diagnosis, 33% of patients had ocular MG. Immunotherapy was used in 87% of patients. A minority of patients (7%) required three or more concurrent immunotherapies. Thymectomy was performed in 24% of patients. DISCUSSION: MGBase is a global registry for collaborative research in MG. Interim analysis of registry data shows disease characteristics similar to those previously published. As global enrollments increase, the registry will generate clinical practice evidence of treatment outcomes, safety, and disease prognostic markers.
引言/目的:患者登记系统是重症肌无力(MG)等罕见病疗效研究的重要工具。现有的MG登记系统受到包括地域范围不足等因素的限制。MGBase被设计为一个全球性的观察性登记系统,旨在研究MG的临床实践结果。 方法:MGBase是在独立的MSBase基金会的支持下开发的。一个国际科学领导小组(SLG)确定了最小数据集和结果指标。数据通过专门设计的平台实时录入,并保存在基于网络的登记系统中。成员可以申请访问全球数据集以进行研究者驱动的子研究。 结果:MGBase数据收集于2021年10月开始。从启动到2024年4月,来自8个国家16个诊所的565名患者入组。该队列中女性占56%,最后一次就诊时的平均年龄为57(标准差19)岁,疾病持续时间中位数为5(四分位间距1.8,10.8)年。76%的患者乙酰胆碱受体抗体阳性(AChR ab+),7%的患者有肌肉特异性激酶抗体(MuSK ab+)。诊断时,33%的患者为眼肌型MG。87%的患者使用了免疫疗法。少数患者(7%)需要同时进行三种或更多种免疫疗法。24%的患者接受了胸腺切除术。 讨论:MGBase是一个用于MG合作研究的全球登记系统。登记系统数据的中期分析显示疾病特征与先前发表的相似。随着全球入组人数的增加,该登记系统将产生关于治疗结果、安全性和疾病预后标志物的临床实践证据。
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