{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,6]],"date-time":"2026-09-06T10:53:10Z","timestamp":1788691990200,"version":"build-2803163510"},"reference-count":31,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2023,11,13]],"date-time":"2023-11-13T00:00:00Z","timestamp":1699833600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000272","name":"National Institute for Health and Care Research","doi-asserted-by":"publisher","award":["NIHR300984"],"award-info":[{"award-number":["NIHR300984"]}],"id":[{"id":"10.13039\/501100000272","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Brain and Behavior"],"published-print":{"date-parts":[[2023,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Duchenne muscular dystrophy (DMD) is a rare, muscle\u2010degenerative disease predominantly affecting males. Natural history models capture the full disease pathway under current care and combine with estimates of new interventions\u2019 effects to assess cost\u2010effectiveness by health technology decision\u2010makers. These models require mortality estimates throughout a patient's lifetime, but rare disease datasets typically contain relatively few patients with short follow\u2010ups. Alternative (published) sources of mortality data may therefore be required.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Estimates of mortality in UK\u2010based DMD patients are age\u2010specific in a uniquely large and nationally representative sample from the CPRD.<\/jats:p><\/jats:sec>","DOI":"10.1002\/brb3.3331","type":"journal-article","created":{"date-parts":[[2023,11,14]],"date-time":"2023-11-14T06:10:57Z","timestamp":1699942257000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Natural history of Duchenne muscular dystrophy in the United Kingdom: A descriptive study using the Clinical Practice Research Datalink"],"prefix":"10.1002","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1846-2150","authenticated-orcid":false,"given":"Jonathan","family":"Broomfield","sequence":"first","affiliation":[{"name":"Department of Population Health Sciences University of Leicester  Leicester UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keith","family":"Abrams","sequence":"additional","affiliation":[{"name":"Department of Statistics University of Warwick  Coventry UK"},{"name":"Centre for Health Economics University of York  York UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nick","family":"Latimer","sequence":"additional","affiliation":[{"name":"School of Health and Related Research (ScHARR) University of Sheffield  Sheffield UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Guglieri","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre Newcastle University and Newcastle Hospitals NHS  Newcastle UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Rutherford","sequence":"additional","affiliation":[{"name":"Department of Population Health Sciences University of Leicester  Leicester UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Crowther","sequence":"additional","affiliation":[{"name":"Red Door Analytics  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,11,13]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(21)00581-6"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30024-3"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.21203\/rs.3.rs\u20102405860\/v1"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000012910"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70272-8"},{"key":"e_1_2_9_8_1","unstructured":"Clinical Practice Research Datalink. (2021).CPRD GOLD August\u00a02021\u00a0(Version 2021.08.001) [Data set]. Clinical Practice Research Datalink.https:\/\/doi.org\/10.48329\/dfcr\u2010ap05"},{"key":"e_1_2_9_9_1","unstructured":"Clinical Practice Research Datalink. (2021).CPRD Aurum June\u00a02021\u00a0(Version 2021.06.001) [Data set]. Clinical Practice Research Datalink.https:\/\/doi.org\/10.48329\/pyc2\u2010we97"},{"key":"e_1_2_9_10_1","unstructured":"ClinicalTrials.gov. (2005).Identifier NCT00468832 Longitudinal study of the relationship between impairment activity limitation participation and quality of life in persons with confirmed Duchenne Muscular Dystrophy (DMD).http:\/\/clinicaltrials.gov\/ct\/show\/NCT00468832?order=1"},{"key":"e_1_2_9_11_1","unstructured":"Critical Path Institute. (2016).Duchenne regulatory science consortium.https:\/\/c\u2010path.org\/programs\/d\u2010rsc\/"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0960-8966(02)00140-2"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2007.03.002"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2019-034024"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.09.006"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2017.11.019"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rehab.2013.06.002"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000669"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10654-020-00613-8"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/pds.4204"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"e_1_2_9_22_1","unstructured":"Morgan C. Crossley E. &Perry M.(2021).Epidemiology and healthcare resource utilisation associated with Duchenne muscular dystrophy.https:\/\/cprd.com\/protocol\/epidemiology\u2010and\u2010healthcare\u2010resource\u2010utilisation\u2010associated\u2010duchenne\u2010muscular\u2010dystrophy"},{"key":"e_1_2_9_23_1","unstructured":"National Health Service. (2022).Muscular dystrophy.https:\/\/www.nhs.uk\/conditions\/muscular\u2010dystrophy\/"},{"key":"e_1_2_9_24_1","unstructured":"NICE. (2016).Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (NICE Guideline).https:\/\/www.nice.org.uk\/guidance\/hst3"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.slsci.2016.07.004"},{"issue":"4","key":"e_1_2_9_26_1","first-page":"477","article-title":"Survival of patients with Duchenne muscular dystrophy","volume":"89","author":"San Martin P. 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