TY - JOUR
T1 - MGBase
T2 - A Global, Observational Registry for Collaborative Research in Myasthenia Gravis
AU - Buzzard, Katherine A.
AU - Van der Walt, Anneke
AU - Butzkueven, Helmut
AU - Zhang, Wen Wen
AU - Barnett-Tapia, Carolina
AU - Cutter, Gary
AU - Heckmann, Jeannine M.
AU - Farr, Pamela
AU - Tran, Carolyn
AU - Sartori, Charlotte
AU - Stupar, Dusko
AU - More, Rein
AU - Sim, Linda
AU - Le, Alison
AU - Tang, Qingxiao
AU - Reddel, Stephen W.
AU - the MGBase Study Group
A2 - Blum, Stefan
A2 - Lovas, Gabvor
A2 - McCombe, Pamela
A2 - Chroni, Elisabeth
A2 - Cruse, Belinda
A2 - Alroughani, Raed
A2 - Tzartos, John
A2 - Gomex-Figueroa, Enrique
A2 - Boz, Cavit
A2 - Karelis, Guntis
A2 - Kermode, Allan
A2 - Crump, Nicholas
N1 - Publisher Copyright:
© 2025 The Author(s). Muscle & Nerve published by Wiley Periodicals LLC.
PY - 2025
Y1 - 2025
N2 - 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.
AB - 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.
KW - database
KW - epidemiology
KW - global
KW - myasthenia gravis
KW - registry
UR - http://www.scopus.com/inward/record.url?scp=105007832979&partnerID=8YFLogxK
UR - https://pubmed.ncbi.nlm.nih.gov/40468746/
U2 - 10.1002/mus.28450
DO - 10.1002/mus.28450
M3 - Article
AN - SCOPUS:105007832979
SN - 0148-639X
JO - Muscle and Nerve
JF - Muscle and Nerve
ER -