{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T21:43:24Z","timestamp":1791582204021,"version":"4.3.4"},"reference-count":278,"publisher":"National Institute for Health and Care Research","issue":"27","license":[{"start":{"date-parts":[[2017,5,1]],"date-time":"2017-05-01T00:00:00Z","timestamp":1493596800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/www.nationalarchives.gov.uk\/doc\/non-commercial-government-licence\/non-commercial-government-licence.htm"}],"funder":[{"DOI":"10.13039\/501100000664","name":"Health Technology Assessment programme","doi-asserted-by":"publisher","award":["12\/144\/04"],"award-info":[{"award-number":["12\/144\/04"]}],"id":[{"id":"10.13039\/501100000664","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Health Technol Assess"],"abstract":"<jats:sec id=\"abs1-1\"><jats:title>Background<\/jats:title><jats:p>Duchenne muscular dystrophy (DMD) is a rare disease that causes the progressive loss of motor abilities such as walking. Standard treatment includes physiotherapy. No trial has evaluated whether or not adding aquatic therapy (AT) to land-based therapy (LBT) exercises helps to keep muscles strong and children independent.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-2\"><jats:title>Objectives<\/jats:title><jats:p>To assess the feasibility of recruiting boys with DMD to a randomised trial evaluating AT (primary objective) and to collect data from them; to assess how, and how well, the intervention and trial procedures work.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-3\"><jats:title>Design<\/jats:title><jats:p>Parallel-group, single-blind, randomised pilot trial with nested qualitative research.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-4\"><jats:title>Setting<\/jats:title><jats:p>Six paediatric neuromuscular units.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-5\"><jats:title>Participants<\/jats:title><jats:p>Children with DMD aged 7\u201316 years, established on corticosteroids, with a North Star Ambulatory Assessment (NSAA) score of 8\u201334 and able to complete a 10-m walk without aids\/assistance. Exclusions: &gt;\u200920% variation between baseline screens 4 weeks apart and contraindications.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-6\"><jats:title>Interventions<\/jats:title><jats:p>Participants were allocated on a 1\u2009:\u20091 ratio to (1) optimised, manualised LBT (prescribed by specialist neuromuscular physiotherapists) or (2) the same plus manualised AT (30 minutes, twice weekly for 6 months: active assisted and\/or passive stretching regime; simulated or real functional activities; submaximal exercise). Semistructured interviews with participants, parents (<jats:italic>n<\/jats:italic>\u2009=\u20098) and professionals (<jats:italic>n<\/jats:italic>\u2009=\u20098) were analysed using Framework analysis. An independent rater reviewed patient records to determine the extent to which treatment was optimised. A cost-impact analysis was performed. Quantitative and qualitative data were mixed using a triangulation exercise.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-7\"><jats:title>Main outcome measures<\/jats:title><jats:p>Feasibility of recruiting 40 participants in 6 months, participant and therapist views on the acceptability of the intervention and research protocols, clinical outcomes including NSAA, independent assessment of treatment optimisation and intervention costs.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-8\"><jats:title>Results<\/jats:title><jats:p>Over 6 months, 348 children were screened \u2013 most lived too far from centres or were enrolled in other trials. Twelve (30% of target) were randomised to AT (<jats:italic>n<\/jats:italic>\u2009=\u20098) or control (<jats:italic>n<\/jats:italic>\u2009=\u20094). People in the AT (<jats:italic>n<\/jats:italic>\u2009=\u20098) and control (<jats:italic>n<\/jats:italic>\u2009=\u20092: attrition because of parental report) arms contributed outcome data. The mean change in NSAA score at 6 months was \u20135.5 [standard deviation (SD) 7.8] for LBT and \u20132.8 (SD 4.1) in the AT arm. One boy suffered pain and fatigue after AT, which resolved the same day. Physiotherapists and parents valued AT and believed that it should be delivered in community settings. The independent rater considered AT optimised for three out of eight children, with other children given programmes that were too extensive and insufficiently focused. The estimated NHS costs of 6-month service were between \u00a31970 and \u00a32734 per patient.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-9\"><jats:title>Limitations<\/jats:title><jats:p>The focus on delivery in hospitals limits generalisability.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-10\"><jats:title>Conclusions<\/jats:title><jats:p>Neither a full-scale frequentist randomised controlled trial (RCT) recruiting in the UK alone nor a twice-weekly open-ended AT course delivered at tertiary centres is feasible. Further intervention development research is needed to identify how community-based pools can be accessed, and how families can link with each other and community physiotherapists to access tailored AT programmes guided by highly specialised physiotherapists. Bayesian RCTs may be feasible; otherwise, time series designs are recommended.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-11\"><jats:title>Trial registration<\/jats:title><jats:p>Current Controlled Trials ISRCTN41002956.<\/jats:p><\/jats:sec><jats:sec id=\"abs1-12\"><jats:title>Funding<\/jats:title><jats:p>This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in<jats:italic>Health Technology Assessment<\/jats:italic>; Vol. 21, No. 27. See the NIHR Journals Library website for further project information.<\/jats:p><\/jats:sec>","DOI":"10.3310\/hta21270","type":"journal-article","created":{"date-parts":[[2017,6,19]],"date-time":"2017-06-19T10:50:06Z","timestamp":1497869406000},"page":"1-120","update-policy":"https:\/\/doi.org\/10.3310\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":25,"title":["Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation"],"prefix":"10.3310","volume":"21","clinical-trial-number":[{"clinical-trial-number":"isrctn41002956","registry":"10.18810\/isrctn","type":"results"}],"author":[{"given":"Daniel","family":"Hind","sequence":"first","affiliation":[{"name":"Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Parkin","sequence":"additional","affiliation":[{"name":"Sheffield 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