source: Pilot and Feasibility Studies
Hind D, Parkin J, Whitworth V, Rex S, Young T, Hampson L, Sheehan J, Maguire C, Cantrill H, Scott E, Epps H, Main M, Geary M, McMurchie H, Pallant L, Woods D, Freeman J, Lee E, Eagle M, Willis T, Muntoni F, Baxter P
Standard treatment of Duchenne muscular dystrophy (DMD) includes regular physiotherapy. There are no data to show whether adding aquatic therapy (AT) to land-based exercises helps maintain motor function. We assessed the feasibility of recruiting and collecting data from boys with DMD in a parallel-group pilot randomised trial (primary objective), also assessing how intervention and trial procedures work.
Ambulant boys with DMD aged 7-16 years established on steroids, with North Star Ambulatory Assessment (NSAA) score >=8, who were able to complete a 10-m walk test without aids or assistance, were randomly allocated (1:1) to 6 months of either optimised land-based exercises 4 to 6 days/week, defined by local community physiotherapists, or the same 4 days/week plus AT 2 days/week. Those unable to commit to a programme, with >20% variation between NSAA scores 4 weeks apart, or contraindications to AT were excluded. The main outcome measures included feasibility of recruiting 40 participants in 6 months from six UK centres, clinical outcomes including NSAA, independent assessment of treatment optimisation, participant/therapist views on acceptability of intervention and research protocols, value of information (VoI) analysis and cost-impact analysis.
Over 6 months, 348 boys were screened: most lived too far from centres or were enrolled in other trials; 12 (30% of the targets) were randomised to AT (n = 8) or control (n = 4). The mean change in NSAA at 6 months was -5.5 (SD 7.8) in the control arm and -2.8 (SD 4.1) in the AT arm. Harms included fatigue in two boys, pain in one. Physiotherapists and parents valued AT but believed it should be delivered in community settings. Randomisation was unattractive to families, who had already decided that AT was useful and who often preferred to enrol in drug studies. The AT prescription was considered to be optimised for three boys, with other boys given programmes that were too extensive and insufficiently focused. Recruitment was insufficient for VoI analysis.
Neither a UK-based RCT of AT nor a twice weekly AT therapy delivered at tertiary centres is feasible. Our study will help in the optimisation of AT service provision and the design of future research.
University of Sheffield, UK; School of Health and Related Research, UK; University of Lancaster, UK; Evelina London Childrens Hospital, Guy's and St Thomas' NHS Foundation Trust, UK; Aquaepps, UK; The UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust, UK; University Hospital Southampton NHS Foundation Trust, UK; Heart of England NHS Foundation Trust, UK; Leeds Teaching Hospitals NHS Trust, UK; Sheffield Children's Hospital, UK; University of Leeds, UK; Newcastle University, UK; Orthopaedic Hospital NHS Foundation Trust, UK
10.1186/s40814-017-0132-0 read more
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