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Hind D, Parkin J, Whitworth V, et al. Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation. Southampton (UK): NIHR Journals Library; 2017 May. (Health Technology Assessment, No. 21.27.)

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

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Chapter 10Conclusions

A full-scale RCT based on frequentist methods is not feasible based on the trial protocol piloted in this study. Before a full-scale RCT can be conducted, further formative work is required to develop an intervention that can be delivered safely by community physiotherapists in settings that are more convenient for families of boys with DMD. If such an intervention is delivered, specialist physiotherapists must help focus clinical problems, propose related exercises and collaborate in the monitoring of intervention safety and the disease condition’s development. Although absence of parent equipoise, access to pools and the prohibition on study co-enrolment are likely to preclude a full-scale randomised trial run in the UK alone, a RCT employing one of a number of novel Bayesian designs, designed for use in rare populations, may be feasible in the future.

Copyright © Queen’s Printer and Controller of HMSO 2017. This work was produced by Hind et al. under the terms of a commissioning contract issued by the Secretary of State for Health. This issue may be freely reproduced for the purposes of private research and study and extracts (or indeed, the full report) may be included in professional journals provided that suitable acknowledgement is made and the reproduction is not associated with any form of advertising. Applications for commercial reproduction should be addressed to: NIHR Journals Library, National Institute for Health Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK.

Included under terms of UK Non-commercial Government License.

Bookshelf ID: NBK436190
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