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Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation.

Hind, Daniel and Parkin, James and Whitworth, Victoria and Rex, Saleema and Young, Tracey and Hampson, Lisa and Sheehan, Jennie and Maguire, Chin and Cantrill, Hannah and Scott, Elaine and Epps, Heather and Main, Marion and Geary, Michelle and McMurchie, Heather and Pallant, Lindsey and Woods, Daniel and Freeman, Jennifer and Lee, Ellen and Eagle, Michelle and Willis, Tracey and Muntoni, Francesco and Baxter, Peter (2017) Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation. Health technology assessment (Winchester, England), 21 (27). pp. 1-120. ISSN 2046-4924. This article is available to all HEFT staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their HEFT Athens login IDs

Full text not available from this repository.
Official URL: https://www.journalslibrary.nihr.ac.uk/hta/hta2127...

Abstract

BACKGROUND

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.

OBJECTIVES

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.

DESIGN

Parallel-group, single-blind, randomised pilot trial with nested qualitative research.

SETTING

Six paediatric neuromuscular units.

PARTICIPANTS

Children with DMD aged 7-16 years, established on corticosteroids, with a North Star Ambulatory Assessment (NSAA) score of 8-34 and able to complete a 10-m walk without aids/assistance. Exclusions: > 20% variation between baseline screens 4 weeks apart and contraindications.

INTERVENTIONS

Participants were allocated on a 1 : 1 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 (n = 8) and professionals (n = 8) 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.

MAIN OUTCOME MEASURES

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.

RESULTS

Over 6 months, 348 children were screened - most lived too 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 the AT (n = 8) and control (n = 2: attrition because of parental report) arms contributed outcome data. The mean change in NSAA score at 6 months was -5.5 [standard deviation (SD) 7.8] for LBT and -2.8 (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 £1970 and £2734 per patient.

LIMITATIONS

The focus on delivery in hospitals limits generalisability.

CONCLUSIONS

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.

TRIAL REGISTRATION

Current Controlled Trials ISRCTN41002956.

FUNDING

This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 21, No. 27. See the NIHR Journals Library website for further project information.

Item Type: Article
Additional Information: This article is available to all HEFT staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their HEFT Athens login IDs
Subjects: WS Paediatrics. Child health
Divisions: Womens and Childrens > Paediatrics
Related URLs:
Depositing User: Mrs Caroline Tranter
Date Deposited: 23 Jun 2017 15:36
Last Modified: 23 Jun 2017 15:36
URI: http://www.repository.heartofengland.nhs.uk/id/eprint/1427

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