| アブストラクト | BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, muscle-degenerative disease predominantly affecting males. Natural history models capture the full disease pathway under current care and combine with estimates of new interventions' effects to assess cost-effectiveness by health technology decision-makers. These models require mortality estimates throughout a patient's lifetime, but rare disease datasets typically contain relatively few patients with short follow-ups. Alternative (published) sources of mortality data may therefore be required. METHODS: The Clinical Practice Research Datalink (CPRD) was evaluated as a source of mortality and natural history data for future economic evaluations of health technologies for DMD and rare diseases in general in the UK population. This retrospective longitudinal cohort study provides flexible parametric estimates of mortality rates and survival probabilities in the current UK DMD population through primary/secondary records in the CPRD since 1990. It also investigates clinically significant milestones such as corticosteroid use, spinal surgery, and cardiomyopathy in these patients. RESULTS: A total of 1121 male patients were included in the study, observed from 0.7 to 48.9 years. Median life expectancy was 25.64 years (95% confidence interval 24.73, 26.47), consistent with previous global estimates. This has improved to 26.47 (25.16, 27.89) years in patients born after 1990. The median ages at corticosteroid initiation, spinal surgery, ventilation, and cardiomyopathy diagnosis were 6.06 years (5.77, 6.29), 14.79 years (14.29, 15.09), 16.97 years (16.50, 18.31), and 15.26 years (14.22, 16.70), respectively. CONCLUSIONS: Estimates of mortality in UK-based DMD patients are age-specific in a uniquely large and nationally representative sample from the CPRD. |
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| ジャーナル名 | Brain and behavior |
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| 投稿日 | 2023/11/14 |
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| 投稿者 | Broomfield, Jonathan; Abrams, Keith; Latimer, Nick; Guglieri, Michela; Rutherford, Mark; Crowther, Michael |
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| 組織名 | Department of Population Health Sciences, University of Leicester, Leicester, UK.;Department of Statistics, University of Warwick, Coventry, UK.;Centre for Health Economics, University of York, York, UK.;School of Health and Related Research (ScHARR), University of Sheffield,;Sheffield, UK.;John Walton Muscular Dystrophy Research Centre, Newcastle University and;Newcastle Hospitals NHS, Newcastle, UK.;Red Door Analytics, Stockholm, Sweden. |
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| Pubmed リンク | https://www.ncbi.nlm.nih.gov/pubmed/37957895/ |
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