Aquatic therapy for children with Duchenne muscular dystrophy

152 Pages·2017·2.33 MB·English

by  Hind D

HEALTH TECHNOLOGY ASSESSMENT
VOLUME 21 ISSUE 27 MAY 2017
ISSN 1366-5278
Aquatic therapy for children with Duchenne muscular
dystrophy: a pilot feasibility randomised controlled
trial and mixed-methods process evaluation
Daniel Hind, James Parkin, Victoria Whitworth, Saleema Rex, Tracey Young,
Lisa Hampson, Jennie Sheehan, Chin Maguire, Hannah Cantrill, Elaine Scott,
Heather Epps, Marion Main, Michelle Geary, Heather McMurchie,
Lindsey Pallant, Daniel Woods, Jennifer Freeman, Ellen Lee, Michelle Eagle,
Tracey Willis, Francesco Muntoni and Peter Baxter
DOI 10.3310/hta21270

Aquatic therapy for children with
Duchenne muscular dystrophy: a pilot
feasibility randomised controlled trial and
mixed-methods process evaluation
Daniel Hind,1* James Parkin,1 Victoria Whitworth,1
Saleema Rex,1 Tracey Young,2 Lisa Hampson,3
Jennie Sheehan,4 Chin Maguire,1 Hannah Cantrill,1
Elaine Scott,2 Heather Epps,5 Marion Main,6
Michelle Geary,7 Heather McMurchie,8
Lindsey Pallant,9 Daniel Woods,10 Jennifer Freeman,11
Ellen Lee,1 Michelle Eagle,12 Tracey Willis,13
Francesco Muntoni6 and Peter Baxter14
1Sheffield Clinical Trials Research Unit, University of Sheffield, Sheffield, UK
2School of Health and Related Research, University of Sheffield, Sheffield, UK
3Department of Mathematics and Statistics, University of Lancaster, Lancaster, UK
4Evelina London Children’s Hospital, Guy’s & St Thomas’ NHS Foundation Trust,
London, UK
5Aquaepps, Dorking, UK
6Dubowitz Neuromuscular Centre (DNC), Great Ormond Street Hospital for
Children NHS Foundation Trust, London, UK
7Children’s Therapy Department, University Hospital Southampton NHS
Foundation Trust, Southampton, UK
8Paediatric Physiotherapy, Heart of England NHS Foundation Trust, Birmingham, UK
9Regional Paediatric Neuromuscular Team, Leeds Teaching Hospitals NHS Trust,
Leeds, UK
10PT Kids, Doncaster, UK
11Leeds Institute of Health Sciences, University of Leeds, Leeds, UK
12Newcastle upon Tyne Hospitals NHS Trust, Newcastle, UK
13The Oswestry Inherited Neuromuscular Service, The Robert Jones and Agnes
Hunt Orthopaedic Hospital NHS Foundation Trust, Oswestry, UK
14Paediatric Neurology, Sheffield Children’s Hospital, Sheffield, UK
*Corresponding author
Declared competing interests of authors: LisaHampsonreportsgrantsfromtheMedicalResearch
Council(MRC)andthepharmaceuticalindustryoutsidethesubmittedwork.FrancescoMuntonireports
grantsfromtheEuropeanUnion7thFrameworkProgramme,MRC,IonisPharmaceuticals/Biogen,Inc.,PTC
Therapeutics,SummitPharmaceuticalInternational,Roche,L’AssociationFrançaisecontrelesMyopathiesand
MuscularDystrophyUKandpersonalfeesfromPfizer,Biogen,Inc.andSummitoutsidethesubmittedwork.

Published May 2017
DOI: 10.3310/hta21270
This reportshould be referenced as follows:
Hind D, ParkinJ, Whitworth V, Rex S, Young T,Hampson L, et al. Aquatic therapy for children
withDuchenne muscular dystrophy: apilotfeasibilityrandomisedcontrolledtrialand
mixed-methodsprocessevaluation.HealthTechnolAssess2017;21(27).
HealthTechnology Assessment isindexed and abstracted in IndexMedicus/MEDLINE, Excerpta
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DOI:10.3310/hta21270 HEALTHTECHNOLOGYASSESSMENT2017 VOL.21 NO.27
Abstract
Aquatic therapy for children with Duchenne muscular
dystrophy: a pilot feasibility randomised controlled trial and
mixed-methods process evaluation
Daniel Hind,1* James Parkin,1 Victoria Whitworth,1 Saleema Rex,1
Tracey Young,2 Lisa Hampson,3 Jennie Sheehan,4 Chin Maguire,1
Hannah Cantrill,1 Elaine Scott,2 Heather Epps,5 Marion Main,6
Michelle Geary,7 Heather McMurchie,8 Lindsey Pallant,9
Daniel Woods,10 Jennifer Freeman,11 Ellen Lee,1 Michelle Eagle,12
Tracey Willis,13 Francesco Muntoni6 and Peter Baxter14
1Sheffield Clinical Trials Research Unit, University ofSheffield, Sheffield, UK
2Schoolof Health andRelated Research, University ofSheffield, Sheffield, UK
3DepartmentofMathematicsandStatistics,UniversityofLancaster,Lancaster,UK
4Evelina London Children’s Hospital, Guy’s& St Thomas’ NHS FoundationTrust, London, UK
5Aquaepps, Dorking, UK
6Dubowitz Neuromuscular Centre (DNC), Great Ormond StreetHospital for Children NHS
FoundationTrust, London, UK
7Children’sTherapy Department, University Hospital Southampton NHS FoundationTrust,
Southampton,UK
8PaediatricPhysiotherapy,HeartofEnglandNHSFoundationTrust,Birmingham,UK
9Regional Paediatric Neuromuscular Team, LeedsTeaching Hospitals NHS Trust, Leeds, UK
10PT Kids, Doncaster, UK
11Leeds Institute ofHealth Sciences, University ofLeeds, Leeds, UK
12Newcastle upon Tyne Hospitals NHS Trust, Newcastle, UK
13TheOswestry Inherited Neuromuscular Service,The Robert Jonesand Agnes Hunt Orthopaedic
Hospital NHS FoundationTrust, Oswestry, UK
14Paediatric Neurology, Sheffield Children’s Hospital, Sheffield, UK
*Corresponding author [email protected]
Background: Duchenne muscular dystrophy (DMD) isa rare diseasethat causesthe progressive loss of
motor abilities such as walking. Standardtreatment includes physiotherapy. Notrial hasevaluated whether
or not adding aquatic therapy (AT) to land-basedtherapy (LBT) exercises helpsto keep muscles strong and
children independent.
Objectives: To assess the feasibility of recruiting boys with DMD to arandomised trial evaluating AT
(primary objective) andto collect datafrom them; to assess how, and howwell, the intervention and trial
procedures work.
Design: Parallel-group, single-blind, randomised pilot trial with nested qualitative research.
Setting: Six paediatric neuromuscular units.
©Queen’sPrinterandControllerofHMSO2017.ThisworkwasproducedbyHindetal.underthetermsofacommissioningcontractissuedbytheSecretaryofStateforHealth.
Thisissuemaybefreelyreproducedforthepurposesofprivateresearchandstudyandextracts(orindeed,thefullreport)maybeincludedinprofessionaljournalsprovidedthat vii
suitableacknowledgementismadeandthereproductionisnotassociatedwithanyformofadvertising.Applicationsforcommercialreproductionshouldbeaddressedto:NIHR
JournalsLibrary,NationalInstituteforHealthResearch,Evaluation,TrialsandStudiesCoordinatingCentre,AlphaHouse,UniversityofSouthamptonSciencePark,Southampton
SO167NS,UK.
ABSTRACT
Participants: Children with DMD aged 7–16years, established on corticosteroids, with aNorth Star
Ambulatory Assessment (NSAA) scoreof 8–34 and ableto complete a10-m walk without aids/assistance.
Exclusions: >20% variation between baseline screens 4weeks apart andcontraindications.
Interventions: Participants were allocated on a1:1ratio to (1) optimised, manualised LBT (prescribed by
specialist neuromuscular physiotherapists) or (2) the sameplus manualised AT (30minutes, twiceweekly
for 6 months: active assisted and/or passive stretching regime; simulated or real functional activities;
submaximal exercise). Semistructured interviews with participants, parents (n=8) and professionals (n=8)
were analysed usingFramework analysis. An independent rater reviewed patient recordsto determine the
extent to which treatment was optimised. Acost-impact analysis was performed. Quantitative and
qualitative data were mixed usingatriangulation exercise.
Main outcome measures: Feasibility of recruiting 40participants in 6months, participant and therapist
views on theacceptability of theintervention and research protocols, clinical outcomes including NSAA,
independent assessment of treatment optimisation and intervention costs.
Results: Over 6months, 348 children were screened –most livedtoo far from centres or were enrolled in
other trials. Twelve (30% of target) were randomised to AT(n=8) or control(n=4). People in theAT
(n=8) and control (n=2: attrition becauseof parentalreport) arms contributed outcome data. Themean
change in NSAA scoreat 6 months was –5.5[standard deviation(SD) 7.8] for LBT and –2.8(SD 4.1) in the
AT arm. Oneboy suffered painand fatigue after AT, which resolved thesame day.Physiotherapists and
parents valued AT and believedthat itshould be delivered in community settings. The independent rater
considered AT optimised for three outof eight children, with other children givenprogrammes that were
too extensive and insufficiently focused. The estimated NHS costs of 6-month service were between £1970
and £2734 perpatient.
Limitations: The focus ondelivery inhospitals limits generalisability.
Conclusions: Neither afull-scale frequentist randomised controlled trial (RCT) recruiting in the UK alone
nor atwice-weekly open-ended AT course delivered at tertiary centres isfeasible. Further intervention
development research is neededto identify howcommunity-based poolscan beaccessed, and how
families can link with eachother and community physiotherapists to access tailored ATprogrammes
guided by highly specialised physiotherapists. Bayesian RCTs maybe feasible; otherwise, time seriesdesigns
are recommended.
Trial registration: Current Controlled Trials ISRCTN41002956.
Funding: This project was funded bythe National Institute for Health Research (NIHR) HealthTechnology
Assessment programme and will bepublished in full in HealthTechnology Assessment; Vol. 21,No. 27.
See theNIHR Journals Library website for further project information.
viii
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