{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T02:59:41Z","timestamp":1790909981657,"version":"4.1.0"},"reference-count":33,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2024,6,4]],"date-time":"2024-06-04T00:00:00Z","timestamp":1717459200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Italian Medicines Agency (AIFA)","award":["2012-13-14"],"award-info":[{"award-number":["2012-13-14"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background: In the context of a comparative study of efficacy and safety of drugs used in rare neuromuscular and neurodegenerative diseases (CAESAR\u2014call 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.<\/jats:p>","DOI":"10.3390\/jcm13113312","type":"journal-article","created":{"date-parts":[[2024,6,4]],"date-time":"2024-06-04T07:39:49Z","timestamp":1717486789000},"page":"3312","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Drug Use Patterns in Myasthenia Gravis: A Real-World Population-Based Cohort Study in Italy"],"prefix":"10.3390","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0009-0008-4265-448X","authenticated-orcid":false,"given":"Marco","family":"Finocchietti","sequence":"first","affiliation":[{"name":"Department of Epidemiology, Lazio Regional Health Service, 00147 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1071-6120","authenticated-orcid":false,"given":"Giada","family":"Crescioli","sequence":"additional","affiliation":[{"name":"Section of Pharmacology and Toxicology, Department of Neurosciences, Psychology, Drug Research and Child Health, University of Florence, 50122 Florence, Italy"},{"name":"Tuscan Regional Centre of Pharmacovigilance, 50139 Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-0434-8745","authenticated-orcid":false,"given":"Olga","family":"Paoletti","sequence":"additional","affiliation":[{"name":"Pharmacoepidemiology Unit, Regional Health Agency of Tuscany, 50141 Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paola","family":"Brunori","sequence":"additional","affiliation":[{"name":"Neurophysiopathology, Perugia Hospital, 06129 Perugia, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Sciancalepore","sequence":"additional","affiliation":[{"name":"National Center for Disease Prevention and Health Promotion, Italian National Institute of Health, 00161 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco","family":"Tuccori","sequence":"additional","affiliation":[{"name":"Tuscan Regional Centre of Pharmacovigilance, 50139 Florence, Italy"},{"name":"Unit of Pharmacology and Pharmacovigilance, Department of Clinical and Experimental Medicine, University of Pisa, 56126 Pisa, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0962-9959","authenticated-orcid":false,"given":"Antonio","family":"Addis","sequence":"additional","affiliation":[{"name":"Department of Epidemiology, Lazio Regional Health Service, 00147 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5259-2026","authenticated-orcid":false,"given":"Alfredo","family":"Vannacci","sequence":"additional","affiliation":[{"name":"Section of Pharmacology and Toxicology, Department of Neurosciences, Psychology, Drug Research and Child Health, University of Florence, 50122 Florence, Italy"},{"name":"Tuscan Regional Centre of Pharmacovigilance, 50139 Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9666-0438","authenticated-orcid":false,"given":"Niccol\u00f2","family":"Lombardi","sequence":"additional","affiliation":[{"name":"Section of Pharmacology and Toxicology, Department of Neurosciences, Psychology, Drug Research and Child Health, University of Florence, 50122 Florence, Italy"},{"name":"Tuscan Regional Centre of Pharmacovigilance, 50139 Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7732-0166","authenticated-orcid":false,"given":"Ursula","family":"Kirchmayer","sequence":"additional","affiliation":[{"name":"Department of Epidemiology, Lazio Regional Health Service, 00147 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"The CAESAR Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,6,4]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1016\/S1043-0679(99)70018-3","article-title":"Myasthenia gravis: Clinical features, pathogenesis, evaluation, and medical management","volume":"11","author":"Heitmiller","year":"1999","journal-title":"Semin. 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