文献检索文档翻译深度研究
Suppr Zotero 插件Zotero 插件
邀请有礼套餐&价格历史记录

新学期,新优惠

限时优惠:9月1日-9月22日

30天高级会员仅需29元

1天体验卡首发特惠仅需5.99元

了解详情
不再提醒
插件&应用
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
高级版
套餐订阅购买积分包
AI 工具
文献检索文档翻译深度研究
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2025

重症肌无力全球协作研究观察性注册库(MGBase)

MGBase: A Global, Observational Registry for Collaborative Research in Myasthenia Gravis.

作者信息

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

机构信息

Eastern Health Clinical School, Monash University, Box Hill, Victoria, Australia.

Royal Melbourne Hospital, Department of Neurosciences, Parkville, Victoria, Australia.

出版信息

Muscle Nerve. 2025 Sep;72(3):424-432. doi: 10.1002/mus.28450. Epub 2025 Jun 4.


DOI:10.1002/mus.28450
PMID:40468746
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12338016/
Abstract

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.

摘要

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。

相似文献

[1]
MGBase: A Global, Observational Registry for Collaborative Research in Myasthenia Gravis.

Muscle Nerve. 2025-9

[2]
Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.

Lancet Neurol. 2023-5

[3]
Rituximab for myasthenia gravis.

Cochrane Database Syst Rev. 2025-7-3

[4]
National guidelines for diagnosis, treatment, and management of myasthenia gravis in Israel.

Ther Adv Neurol Disord. 2025-8-3

[5]
Comparative effectiveness of azathioprine and mycophenolate mofetil for myasthenia gravis (PROMISE-MG): a prospective cohort study.

Lancet Neurol. 2024-3

[6]
The Black Book of Psychotropic Dosing and Monitoring.

Psychopharmacol Bull. 2024-7-8

[7]
Switching to subcutaneous zilucoplan from intravenous complement component 5 inhibitors in generalised myasthenia gravis: a phase IIIb, open-label study.

Ther Adv Neurol Disord. 2025-7-5

[8]
Comparison of outcomes and postoperative immunotherapy between patients with non-thymomatous and thymomatous myasthenia gravis following thymectomy.

Ther Adv Neurol Disord. 2025-6-20

[9]
A Phase 3 Trial of Inebilizumab in Generalized Myasthenia Gravis.

N Engl J Med. 2025-6-19

[10]
The quantity, quality and findings of network meta-analyses evaluating the effectiveness of GLP-1 RAs for weight loss: a scoping review.

Health Technol Assess. 2025-6-25

本文引用的文献

[1]
Efficacy and Safety of Rituximab for New-Onset Generalized Myasthenia Gravis: The RINOMAX Randomized Clinical Trial.

JAMA Neurol. 2022-11-1

[2]
Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG Study.

Neurology. 2022-1-25

[3]
Accuracy of patient-reported data for an online patient registry of autoimmune myasthenia gravis and Lambert-Eaton myasthenic syndrome.

Neuromuscul Disord. 2021-7

[4]
The Duke myasthenia gravis clinic registry: I. Description and demographics.

Muscle Nerve. 2021-2

[5]
Exploring outcomes and characteristics of myasthenia gravis: Rationale, aims and design of registry - The EXPLORE-MG registry.

J Neurol Sci. 2020-7-15

[6]
Hospital admission and prevalence trends of adult myasthenia gravis in Finland in 2004-2014: A retrospective national registry study.

J Neurol Sci. 2019-10-17

[7]
Cross-sectional analysis of the Myasthenia Gravis Patient Registry: Disability and treatment.

Muscle Nerve. 2019-10-9

[8]
The MSBase registry: Informing clinical practice.

Mult Scler. 2019-5-23

[9]
Recommendations for Improving the Quality of Rare Disease Registries.

Int J Environ Res Public Health. 2018-8-3

[10]
Development and validation of the Myasthenia Gravis Impairment Index.

Neurology. 2016-8-30

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

推荐工具

医学文档翻译智能文献检索