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. 2024 Nov;271(11):7239-7249.
doi: 10.1007/s00415-024-12714-5. Epub 2024 Oct 10.

Treatment of myasthenia gravis in france: A retrospective claims database study (STAMINA)

C Tard  1 , P Laforet  2   3 , G de Pouvourville  4 , A Crochard  5 , G Chollet  6 , C Nevoret  7 , S Bouée  7 , E Salort-Campana  8
Affiliations

Treatment of myasthenia gravis in france: A retrospective claims database study (STAMINA)

C Tard et al. J Neurol. 2024 Nov.

Abstract

Background: This study aimed to describe treatment patterns in patients with myasthenia gravis (MG) in France.

Methods: A retrospective cohort analysis was performed using the French National Health Data System (SNDS) database between 2008 and 2019. MG patients were identified using ICD-10 codes during hospitalization and/or long-term disease. We defined two adult subpopulations: a prevalent MG population of patients alive on 31/12/2019 and an incident population of newly identified patients with MG in 2012 and 2013.

Results: Among the 22,079 prevalent patients, 53.1% (n = 11,498) received at least one chronic MG treatment in 2019. Among these treated patients, 52.5% received Acetylcholinesterase inhibitors (AChEIs) only, 10.2% were treated with corticosteroids (CS) ± AChEIs, 7.3% with non-steroidal immunosuppressive treatments (NSIST) and CS, 24.2% with NSIST w/o CS, and 5.8% received immunoglobulin and/or plasma exchange. Among the 2,661 incident patients, 84.6% received at least one chronic MG treatment over the 6-year follow-up period, and among them, 79.0% had at least one treatment category change. During the first semester of follow-up, 28.1% of patients were treated with an immunomodulator (CS, NSIST). Among patients starting treatment with immunomodulator, the proportion of those treated with CS decreased from 35.3% at initiation to 10.9% at 6 years.

Conclusion: This study illustrates the complexity of MG management. Significant CS sparing was observed over time. The frequent treatment changes especially in patients with an immunomodulator treatment reflect the high variability of the disease severity. The need for personalised treatment approaches in the management of MG to reduce the burden of disease remains.

Keywords: Management; Myasthenia gravis; Real-world data; Treatment.

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Conflict of interest statement

Declarations Conflicts of interest SCE, LP, DePG, and TC received fees (advisory boards, consultation, education, presentations) from UCB. CA and CG are employees of UCB, which supported this study. NC, and BS are CEMKA employees, who received fees from UCB to conduct this analysis. Ethical approval The study protocol was approved by the HDH (Health Data Hub), the National Data Protection Authority CNIL (Commission Nationale de l’Informatique et des Libertes) (date of acceptation: 09/23/2021, reference MLD/MFI/AR2110968), and the National Medical Council (Conseil National de l’Ordre des Médecins—CNOM). Patient consent According to HDH and CNIL, no written informed consent was required for the study. The research complies with the guidelines for human studies and was conducted ethically in accordance with the World Medical Association Declaration of Helsinki.

Figures

Fig. 1
Fig. 1
Chronic treatments in 2019 for treated patients in the prevalent population (N = 11,498). The table on the right side gives the number of patients for all combinations of treatments. For example, of the 665 patients (5.8%) who were treated with’IVIg and/or PLEX’ (colored in grey), 173 patients were treated with IVIg/PLEX, AChEIs, and NSIST, and 151 patients were treated with IVIg/PLEX and AChEIs, etc. Definitions of chronic treatments: IVIg and/or PLEX: at least 4 sessions per year with or without AChEIs, NSIST or CS; NSIST w. CS: at least one NSIST delivery per year (mycophenolic acid, azathioprine, cyclosporine, tacrolimus, rituximab, and cyclophosphamide) and at least 5 CS deliveries over a 6-month period, and fewer than 4 sessions/year of IVIg/PLEX; NSIST w/o CS: at least one NSIST delivery per year with fewer than 5 CS deliveries over a 6-month period and fewer than 4 sessions/year of IVIg/PLEX; CS: at least 5 CS deliveries over a 6-month period with or without AChEIs and fewer than 4 sessions/year of IVIg/PLEX and no chronic NSIST delivery; AChEIs only: delivery of AChEIs and not meeting the criteria for any of the aforementioned categories
Fig. 2
Fig. 2
Sunburst diagram depicting 1-year treatment sequences over the first 6 years following MG identification (each ‘spoke’ follows a patient over time: successive treatments for the same patients are visualized through each circle, chronologically from the centre to the periphery). IVIg and/or PLEX: at least 4 sessions per year with or without AChEIs, NSIST, or CS; NSIST w. CS: at least one NSIST delivery per year (mycophenolic acid, azathioprine, cyclosporine, tacrolimus, rituximab, and cyclophosphamide) and at least 5 CS deliveries over a 6-month period, and fewer than 4 sessions/year of IVIg/PLEX; NSIST w/o CS: at least one NSIST delivery per year with fewer than 5 CS deliveries over a 6-month period and fewer than 4 sessions/year of IVIg/PLEX; CS: at least 5 CS deliveries over a 6-month period with or without AChEIs and fewer than 4 sessions/year of IVIg/PLEX and no chronic NSIST delivery; ACchEIs only: delivery of AChEIs and not meeting the criteria for any of the aforementioned categories
Fig. 3
Fig. 3
Modifications of treatment for the 2012–2013 incident patients treated with CS and/or NSIST in the first 6 months (N = 632 patients). IVIg and/or PLEX: at least 4 sessions per year with or without AChEIs, NSIST or CS; NSIST w. CS: at least one NSIST delivery per year (mycophenolic acid, azathioprine, cyclosporine, tacrolimus, rituximab, and cyclophosphamide) and at least 5 CS deliveries over a 6-month period, and fewer than 4 sessions/year of IVIg/PLEX; NSIST w/o CS: at least one NSIST delivery per year with fewer than 5 CS deliveries over a 6-month period and fewer than 4 sessions/year of IVIg/PLEX; CS: at least 5 CS deliveries over a 6-month period with or without AChEIs and fewer than 4 sessions/year of IVIg/PLEX and no chronic NSIST delivery; AChEIs only: delivery of AChEIs and not meeting the criteria for any of the aforementioned categories
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