Characterization of Adult Patients With Myasthenia Gravis Using French Rare Disease Registry
Author(s)
Attarian S1, Camdessanche JP2, Echaniz-Laguna A3, Blein C4, Ciumas M5, Messiaen C6, Jannot AS6, Sole G7
1Assistance Publique - Hopitaux de Marseille, Marseille, France, 2Hôpital Nord, University Hospital of Saint-Étienne, Euro-NMD, SAINT-ÉTIENNE, ST ETIENNE, France, 3APHP, CHU de Bicêtre, INSERM U1195, Paris-Saclay University, LE KREMLIN-BICÊTRE, PARIS SACLAY, PARIS, France, 4argenx, Zwijnaarde, Belgium, 5argenx, 9052 Zwijnaarde, Oost-Vlaanderen, Belgium, 6Assistance Publique Hôpitaux de Paris-Centre (AP-HP), Georges Pompidou European Hospital, Paris, France, France, 7Pellegrin Hospital, Bordeaux University Hospitals, BORDEAUX, BORDEAUX, France
Presentation Documents
OBJECTIVES: This study aims to describe the characteristics of patients with Myasthenia Gravis (MG) using data from the French rare disease registry, Banque Nationale de Données Maladies Rares (BNDMR).
METHODS: A retrospective cohort study was conducted using the BNDMR. This is a national rare disease registry managed by referent centers. Patients were selected based on presence of: (1) ≥1 confirmed diagnosis of MG, (2) ≥1 visit in a referent center between Jan 2007 and Dec 2021, (3) adults patients (≥18 years), (4) non opposed for data reuse. Extracted variables were demographic characteristics, diagnosis and disease history. Baseline characteristics were assessed at index date defined as the date of confirmed diagnosis or the first visit for patients already diagnosed.
RESULTS: Out of 5165 initial patients identified with the MG code, 3963 patients met the inclusion criteria. The mean follow-up duration of the selected population was 6.1 ± 3.8 years. Mean (SD) age at diagnosis was 55 (19) years. Mean (SD) age at index date was 58 (18) years (18-40 years: 20%; 41-65 years: 38%; >65 years: 42%), with a predominance in females (sex rate ratio: 1.26). Mean age of women was younger (55 years) with a distribution showing 2 sub-cohorts (<40 years versus >40 years). The number of MG cases recruited in the reference centers accumulated progressively following the creation of the database in 2007 to reach 3,529 patients in 2021, corresponding to a prevalence rate of 52.2 [50.5 – 53.9] cases per million in 2021.
CONCLUSIONS: Characteristics of overall MG patients were consistent with existing literature. The observed prevalence of MG is lower than previously reported, likely due to the study's focus on patients with at least one visit to a referent center. The cohort is representative and includes diagnosis certainty documented by 19 referent centers.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
RWD81
Topic
Epidemiology & Public Health, Study Approaches
Topic Subcategory
Public Health, Registries
Disease
Neurological Disorders, Rare & Orphan Diseases