title: Aquatic therapy for boys with Duchenne muscular dystrophy (DMD): an external pilot randomised controlled trial.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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.
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
  
  
  BACKGROUND
  ISRCTN41002956.
authors.affiliation: Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK.
authors.forename: Daniel
authors.initials: D
authors.lastname: Hind
authors.affiliation: Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK.
authors.forename: James
authors.initials: J
authors.lastname: Parkin
authors.affiliation: Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK.
authors.forename: Victoria
authors.initials: V
authors.lastname: Whitworth
authors.affiliation: Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK.
authors.forename: Saleema
authors.initials: S
authors.lastname: Rex
authors.affiliation: School of Health and Related Research, Sheffield, UK.
authors.forename: Tracey
authors.initials: T
authors.lastname: Young
authors.affiliation: Department of Mathematics and Statistics, University of Lancaster, Lancaster, UK.
authors.forename: Lisa
authors.initials: L
authors.lastname: Hampson
authors.affiliation: Evelina London Childrens Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK.
authors.forename: Jennie
authors.initials: J
authors.lastname: Sheehan
authors.affiliation: Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK.
authors.forename: Chin
authors.initials: C
authors.lastname: Maguire
authors.affiliation: Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK.
authors.forename: Hannah
authors.initials: H
authors.lastname: Cantrill
authors.affiliation: School of Health and Related Research, Sheffield, UK.
authors.forename: Elaine
authors.initials: E
authors.lastname: Scott
authors.affiliation: Aquaepps, Dorking, Surrey UK.
authors.forename: Heather
authors.initials: H
authors.lastname: Epps
authors.affiliation: Dubowitz Neuromuscular Centre (DNC), The UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors.forename: Marion
authors.initials: M
authors.lastname: Main
authors.affiliation: Children's Therapy Department, University Hospital Southampton NHS Foundation Trust, Southampton, UK.
authors.forename: Michelle
authors.initials: M
authors.lastname: Geary
authors.affiliation: Paediatric Physiotherapy, Heart of England NHS Foundation Trust, Birmingham, UK.
authors.forename: Heather
authors.initials: H
authors.lastname: McMurchie
authors.affiliation: Regional Paediatric Neuromuscular Team, Leeds Teaching Hospitals NHS Trust, Leeds, UK.
authors.forename: Lindsey
authors.initials: L
authors.lastname: Pallant
authors.affiliation: Paediatric Neurology, Sheffield Children's Hospital, Sheffield, UK.
authors.forename: Daniel
authors.initials: D
authors.lastname: Woods
authors.affiliation: Leeds Institute of Health Sciences, University of Leeds, Leeds, UK.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Freeman
authors.affiliation: Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK.
authors.forename: Ellen
authors.initials: E
authors.lastname: Lee
authors.affiliation: Newcastle Muscle Centre, Newcastle University, Newcastle, UK.
authors.forename: Michelle
authors.initials: M
authors.lastname: Eagle
authors.affiliation: The Oswestry Inherited Neuromuscular Service, The Robert Jones and Agnes Hunt, Orthopaedic Hospital NHS Foundation Trust, Oswestry, UK.
authors.forename: Tracey
authors.initials: T
authors.lastname: Willis
authors.affiliation: Dubowitz Neuromuscular Centre (DNC), The UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors.forename: Francesco
authors.initials: F
authors.lastname: Muntoni
authors.affiliation: Paediatric Neurology, Sheffield Children's Hospital, Sheffield, UK.
authors.forename: Peter
authors.initials: P
authors.lastname: Baxter
keywords: Aquatic therapy; Duchenne muscular dystrophy; Feasibility study; Hydrotherapy; Physical therapy; Pilot study