HYDRO-DMD: Hydrotherapy for Duchenne Muscular Dystrophy (DMD): a pilot and feasibility randomised controlled trial in children

The Challenges

Physiotherapy is thought to help children with Duchenne muscular dystrophy (DMD) stay physically active. Aquatic therapy (hydrotherapy), physiotherapy in warm water, allows exercise and stretching which are not possible on land. It is not clear that aquatic therapy works, so NHS access is limited. This pilot study was funded to see if a controlled trial of aquatic therapy for DMD was feasible. We wanted to enrol 40 children with 20 having aquatic therapy. If we succeeded, we could say that a full-scale study was feasible.

The Research

Mixed methods feasibility study incorporating external pilot RCT, qualitative case study, cost-impact analysis. All children were asked to do stretching and exercise at home. We planned that half would have aquatic therapy in hospital pools for half an hour, twice a week for six months. We measured how well they could use their legs at the beginning and end. After this, children, parents and physiotherapists told us how they felt about aquatic therapy and the study.

The Results

Lay Summary Video

We screened 348 children with DMD for the study. Only twelve could enter the study. This is too few to show if aquatic therapy works or not. Those involved said it should be offered nearer to their homes than we managed. Not enough children could be found for a full-scale study run only from hospitals. Local aquatic therapy programmes should be designed by children, parents, physiotherapists and teaching assistants. Standard trial designs may not be feasible. Other research designs might be needed to test aquatic therapy.

What people said:
Some children who had aquatic therapy felt better:
"I couldn’t jump and now I can" (Child)
"the next day the body is more refreshed … it was nice way to start the day" (parent)

But some didn't improve.
"Unfortunately, I couldn’t find any kind of good positive result from hydrotherapy" (parent)

It wasn't just about how well your legs worked...
“He really did come out of himself and be excitable in the pool and actually chat to the physiotherapists… rather than being the reserved child he often is in public…" (Parent)

One child was very tired and had some pain after the aquatic therapy:
“Well I get really exhausted I feel like I wanna go to bed, but obviously I can’t go to bed because I have to go to school... when I get in class my legs are in a little bit of pain but then, when it’s the afternoon my legs feel better.” (Child)

Travelling to the hospital pools to do aquatic therapy was tough:
"It was probably only fifteen miles each way, but via the school and then rush hour traffic. Bear in mind my session was at four o’clock so sometimes it was an hour and a half on the way back, not a challenge unless you’ve got a five month old screaming baby in the back.”

Physiotherapists had different opinions about aquatic therapy.

"I mean I’ve often said ‘this child needs hydro'... but I think I would be even more dogmatic now and say ‘look it really is a good intervention’."
“All he’s done is get progressively worse and had more and more issues at school with fatigue and I think we’ve affected his quality of life very negatively”

The Impact

Before our study was neither robust data nor internationally-agreed guidelines on the effectiveness or optimal content of physical therapies for Duchenne Muscular Dystrophy. We created and tested manuals for land-based and aquatic therapy which are publicly available.

Publications

Hind D, Parkin J, Whitworth V, Rex S, Young T, Hampson L, et al. Aquatic Therapy for Children with Duchenne muscular dystrophy: a pilot-feasibility randomised controlled trial and mixed-methods process evaluation. Health Technol Assess 2017;21(XX). [In press]

Hind D, Parkin J, Whitworth V. Rex S. Young T, Hampson L. et al. Aquatic therapy for boys with Duchenne muscular dystrophy (DMD): an external pilot randomised controlled trial. Pilot and Feasibility Studies. DOI : 10.1186/s40814-017-0132-0 [In press]

Main M, Epps H, Hind D, Scott E, Sheehan J, Geary M, et al. Development and initial experience with aquatic therapy and land-based physiotherapy manuals for children with Duchenne Muscular Dystrophy. [Manuscript under consideration at journal]

HydroDMD Final Report

The HydroDMD Final Report can be found here.

Project Staff

Name Site Telephone Number Email
Dr Peter Baxter
Chief Investigator
Sheffield Children's NHS Foundation Trust 0114 2717612

peter.baxter@sch.nhs.uk

Elaine Scott
Local Co-Investigator
ScHARR, The University of Sheffield 0114 2224301 elaine.scott@sheffield.ac.uk
Dr Daniel Hind
CTRU Assistant Director
ScHARR, The University of Sheffield 0114 2220707 d.hind@sheffield.ac.uk
Chin Maguire
Study Manager
ScHARR, The University of Sheffield 0114 2220717 c.maguire@sheffield.ac.uk
Amanda Loban
Data Manager
ScHARR, The University of Sheffield 0114 2220685 a.loban@sheffield.ac.uk
Hannah Cantrill
Research Assistant
ScHARR, The University of Sheffield 0114 2224347 h.cantrill@sheffield.ac.uk

Ellen Lee
Statistician

ScHARR, The University of Sheffield 0114 2220805 e.lee@sheffield.ac.uk
Tracey Young
Senior Health Economist
ScHARR, The University of Sheffield 0114 2220837 t.a.young@sheffield.ac.uk

Katie Walker
Trial Support Officer

ScHARR, The University of Sheffield 0114 2220892 k.m.walker@sheffield.ac.uk
Jenny Freeman
Senior Statisitician
The University of Leeds

j.freeman@leeds.ac.uk

Gillian Gatenby
Sponsor
Sheffield Children's NHS Foundation Trust 0114 3053219 gillian.gatenby@sch.nhs.uk

Sites

Site Study Members Site Study Role

Sheffield

Noeleen Goulbourne Sheffield Children's Foundation Trust Principal Investigator

London

Marion Main Great Ormond Street Hospital for Children NHS Foundation Trust Principal Investigator

Leeds

Lindsey Pallant Leeds Teaching Hospitals NHS Foundation Trust Principal Investigator

Birmingham

Heather McMurchie Birmingham Children's Hospital NHS Foundation Trust Principal Investigator

Southampton

Michelle Geary Southampton Children's Hospital NHS Foundation Trust Principal Investigator

Oswestry

Tracey Willis Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust Principal Investigator