| アブストラクト | BACKGROUND: 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. METHODS: A retrospective, longitudinal, cohort study was conducted of patients in England aged >/= 18 years with treatment-refractory or non-refractory MG, using data recorded during 1997-2016 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 t tests for normally distributed data and Mann-Whitney U 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. RESULTS: 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 2-3 years of diagnosis. Patients with MG refractory to conventional treatment (n = 66) experienced more exacerbations and MG-related hospitalizations than patients with non-refractory disease (n = 1083). 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. CONCLUSIONS: These results show that conventional treatments for MG are not adequately managing patients' 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. |
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| ジャーナル名 | BMC neurology |
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| 投稿日 | 2022/5/10 |
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| 投稿者 | Harris, Linda; Graham, Sophie; MacLachlan, Sharon; Exuzides, Alex; Jacob, Saiju |
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| 組織名 | Biohaven Pharmaceuticals, 215 Church Street, New Haven, CT, 06510, USA.;, The Ark, 201 Talgarth Road, London, W6 8DL, UK.;, 450 Sansome Street, Suite 650, San Francisco, CA, 94111, USA.;Department of Neurology and Centre for Rare Diseases, Institute of Immunology and;Imunotherapy, University Hospitals Birmingham and University of Birmingham,;Mindelsohn Way, Edgbaston, Birmingham, B15 2GW, UK. Saiju.Jacob@uhb.nhs.uk. |
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| Pubmed リンク | https://www.ncbi.nlm.nih.gov/pubmed/35534810/ |
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