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. 2024 Jun 4;13(11):3312.
doi: 10.3390/jcm13113312.

Drug Use Patterns in Myasthenia Gravis: A Real-World Population-Based Cohort Study in Italy

Marco Finocchietti  1 , Giada Crescioli  2   3 , Olga Paoletti  4 , Paola Brunori  5 , Francesco Sciancalepore  6 , Marco Tuccori  3   7 , Antonio Addis  1 , Alfredo Vannacci  2   3 , Niccolò Lombardi  2   3 , Ursula Kirchmayer  1 , The Caesar Study Group
Affiliations

Drug Use Patterns in Myasthenia Gravis: A Real-World Population-Based Cohort Study in Italy

Marco Finocchietti et al. J Clin Med. .

Abstract

Background: In the context of a comparative study of efficacy and safety of drugs used in rare neuromuscular and neurodegenerative diseases (CAESAR-call AIFA_FV_2012-13-14), we assessed the use patterns of drugs indicated for myasthenia gravis (MG). Methods: A retrospective cohort study was conducted based on administrative healthcare data. For a cohort of MG patients, prevalent and incident use of pyridostigmine (Py) and other indicated drugs in the first year after case identification was evaluated. Prevalent combined use of major therapies (azathioprine (Az), prednisone (Pr), vitamin D (Vd)) stratified by Py use was assessed, and a comparison between therapies at the time of MG identification and during the first year of follow-up was performed. Results: We included 2369 MG patients between 2013 and 2019. Among them, prevalent and incident Py users were 38.4% and 22.0%, respectively. In the first year of follow-up, the use of Pr was observed in 74.5% of Py prevalent users and in 82.0% of Py incident users, respectively; the use of Az was observed in 24.9% and 23.0%, respectively; and the use of Vd was observed in 53.3% and 48.2%, respectively. Among 910 Py prevalent users, 13.1% also used Az, Pr, and Vd, while 15.3% used none of these. Among 938 non-Py users, 2.7% used Az, Pr, and Vd, while 53.8% used none of these. During the first year, an increase in combined therapies was evident in incident Py users. Conclusions: Our results suggest that, for some MG patients, there may be a need for treatments that combine a rapid onset of benefit with long-term and consistent disease control. These issues may be addressed by the new treatments currently being developed. To date, more studies are needed to address the heterogeneity, quality, and generalizability of the existing data and to evaluate patterns of use, efficacy, and safety of new or emerging therapies for MG.

Keywords: cohort study; corticosteroid; drug use; myasthenia gravis; pharmacoepidemiology; pyridostigmine.

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Conflict of interest statement

The authors declare no conflicts of interest. The funders had no role in the design of the study, in the collection, analyses, or interpretation of data, in the writing of the manuscript; or in the decision to publish the results.

Figures

Figure 1
Figure 1
Cohort selection of MG patients in three Italian regions in the period 2013–2019.
Figure 2
Figure 2
Frequency of drugs indicated for MG during follow-up.
Figure 3
Figure 3
Use patterns of the study drugs during follow-up stratified by pyridostigmine use.
Figure 4
Figure 4
Changes in drug use patterns between baseline and follow-up stratified by pyridostigmine use.
See this image and copyright information in PMC

References

    1. Heitmiller R.F. Myasthenia gravis: Clinical features, pathogenesis, evaluation, and medical management. Semin. Thorac. Cardiovasc. Surg. 1999;11:41–46. doi: 10.1016/s1043-0679(99)70018-3. - DOI - PubMed
    1. Mantegazza R., Cavalcante P. Diagnosis and treatment of myasthenia gravis. Curr. Opin. Rheumatol. 2019;31:623–633. doi: 10.1097/BOR.0000000000000647. - DOI - PubMed
    1. Gilhus N.E., Verschuuren J.J. Myasthenia gravis: Subgroup classification and therapeutic strategies. Lancet Neurol. 2015;14:1023–1036. doi: 10.1016/S1474-4422(15)00145-3. - DOI - PubMed
    1. Hehir M.K., Silvestri N.J. Generalized Myasthenia Gravis: Classification, Clinical Presentation, Natural History, and Epidemiology. Neurol. Clin. 2018;36:253–260. doi: 10.1016/j.ncl.2018.01.002. - DOI - PubMed
    1. Dresser L., Wlodarski R., Rezania K., Soliven B. Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. J. Clin. Med. 2021;10:2235. doi: 10.3390/jcm10112235. - DOI - PMC - PubMed