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

Abstract

BackgroundDuchenne 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.ObjectivesTo 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.DesignParallel-group, single-blind, randomised pilot trial with nested qualitative research.SettingSix paediatric neuromuscular units.ParticipantsChildren 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.InterventionsParticipants 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 measuresFeasibility 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.ResultsOver 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.LimitationsThe focus on delivery in hospitals limits generalisability.ConclusionsNeither 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 registrationCurrent Controlled Trials ISRCTN41002956.FundingThis project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 21, No. 27. See the NIHR Journals Library website for further project information.

Using Normalization Process Theory in feasibility studies and process evaluations of complex healthcare interventions: a systematic review

“…Godfrey et al [ 36 ] UK NPT Delirium prevention in hospital Systematic review [ 158 ] Yes Process evaluation Qualitative Prospective Yes Yes Yes 15. Hind et al [ 37 ] UK NPT Aquatic therapy for children with Duchenne muscular Dystrophy Yes Feasibility Study Qualitative Prospective Yes No No 16. Hooker et al [ 38 – 42 ] Australia NPT Identifying women at risk of intimate partner violence Yes Process evaluation Mixed Prospective Yes Yes Yes 17.…”

“…They used NPT in ‘identifying and describing the components and sub-components of the intervention’ to understand ‘the nuances associated with collective implementation’. The next most numerous group of studies focused on the implementation of diagnostic and therapeutic interventions ( n = 28, 25.9% [ 19 , 24 – 26 , 28 – 31 , 36 , 37 , 47 , 48 , 55 – 57 , 67 , 69 , 78 , 80 , 90 , 95 , 97 , 98 , 103 , 104 , 111 , 117 , 123 , 126 , 137 , 142 , 143 ]). For example, in the USA, Hoberg et al [ 98 ] examined the implementation of a new form of group therapy for people with mental health problems, while Leon et al [ 48 ] showed how provider initiated testing and counselling for HIV was successfully normalised in a South African setting.…”

Timed immersion expiration measures in patients with muscular dystrophies

“…Recent research protocols in the aquatic environment aim at optimizing trunk control, due to the hydrostatic pressure, which also improves body perception and even proprioception [27]. Aquatic therapy can be beneficial for strength, endurance and range of motion maintenance.…”

Are pilot trials useful for predicting randomisation and attrition rates in definitive studies: A review of publicly funded trials

“…External pilot trials have demonstrated clear value where there are many unknown factors including whether the intervention or outcome measures are acceptable to participants and where uncertainties exist around the implementation of the intervention. 7 , 23 – 26 In such cases, an external pilot trial is likely to be more appropriate than an internal pilot. However, based on the findings of this study, we would recommend that where the key unknown factors are randomisation and retention rates alone, investigators should consider using an internal pilot rather than an external pilot.…”