{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T06:20:43Z","timestamp":1793686843871,"version":"4.5.1"},"reference-count":29,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2022,5,9]],"date-time":"2022-05-09T00:00:00Z","timestamp":1652054400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2022,5,9]],"date-time":"2022-05-09T00:00:00Z","timestamp":1652054400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["BMC Neurol"],"published-print":{"date-parts":[[2022,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Patients with generalized myasthenia gravis (MG) often experience debilitating exacerbations, with the possibility of life-threatening respiratory crises requiring hospitalization. Long-term longitudinal studies are needed to understand the burden of MG, including in patients whose disease is refractory to conventional treatment.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      A retrospective, longitudinal, cohort study was conducted of patients in England aged\u2009\u2265\u200918\u00a0years with treatment-refractory or non-refractory MG, using data recorded during 1997\u20132016 in the Clinical Practice Research Datalink and the Hospital Episode Statistics databases. A control cohort of patients without MG, matched to the patients in the treatment-refractory MG cohort, was also identified. Outcome measures included myasthenic crises, MG exacerbations, MG-related hospitalizations, comorbidities, and all-cause mortality. Descriptive statistics were calculated for the overall MG population. For continuous variables, between-cohort comparisons were made using\n                      <jats:italic>t<\/jats:italic>\n                      \u00a0tests for normally distributed data and Mann\u2013Whitney\n                      <jats:italic>U<\/jats:italic>\n                      tests for non-normally distributed data. For categorical data, the comparisons were made by chi-squared tests. Differences in clinical outcomes between cohorts were modeled using negative binomial regression.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      A total of 1149 patients with MG were included. Overall, 18.4% of patients experienced myasthenic crises, 24.6% experienced exacerbations, and 38.6% underwent MG-related hospitalizations. Most of these events occurred within 2\u20133\u00a0years of diagnosis. Patients with MG refractory to conventional treatment (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200966) experienced more exacerbations and MG-related hospitalizations than patients with non-refractory disease (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20091083). Patients with refractory MG experienced a higher frequency of renal disease and hypertension compared with patients with non-refractory MG, and with matched patients without MG. They were also more likely to have diabetes and congestive heart failure than the matched controls. Rates of all-cause mortality during the follow-up period did not differ between patients with refractory MG and non-refractory MG.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>These results show that conventional treatments for MG are not adequately managing patients\u2019 symptoms and that patients with refractory MG are more likely to experience certain comorbidities than those with non-refractory MG or matched controls without MG. Future research should focus on the impact of newer targeted therapies on long-term clinical outcomes and comorbid conditions.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s12883-022-02692-4","type":"journal-article","created":{"date-parts":[[2022,5,9]],"date-time":"2022-05-09T02:03:33Z","timestamp":1652061813000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":54,"title":["A retrospective longitudinal cohort study of the clinical burden in myasthenia gravis"],"prefix":"10.1186","volume":"22","author":[{"given":"Linda","family":"Harris","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Graham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon","family":"MacLachlan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alex","family":"Exuzides","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saiju","family":"Jacob","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2022,5,9]]},"reference":[{"issue":"5","key":"2692_CR1","doi-asserted-by":"publisher","first-page":"419","DOI":"10.1055\/s-0036-1586265","volume":"36","author":"DB Drachman","year":"2016","unstructured":"Drachman DB. 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The ethics committee that waivered the need for informed consent was the Independent Scientific Advisory Committee for Medicines and Healthcare Products Regulatory Agency database research. This committee also approved the conduct of the study (17_111). All authors confirm that the research was conducted in accordance with the Declaration of Helsinki.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"LH disclosed that, at the time the study was conducted, she was an employee of Alexion Pharmaceuticals, the study sponsor, and owns shares in the company. SG and SM are current employees of Evidera and AE is a former Evidera employee; Evidera received payment from Alexion Pharmaceuticals for work on this study and for medical writing support. SJ has disclosed that he received honoraria and expenses from Alexion Pharmaceuticals as an international advisory board member for the Eculizumab in Myasthenia trial, has received honoraria and speaker fees from Alnylam pharmaceuticals, Terumo BCT, and Eisai Ltd, and research support from Momenta pharmaceuticals, argenX pharma and UCB Ltd.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"172"}}