RCT of a physiotherapy intervention for children with haemophilia
Research type
Research Study
Full title
Development Of a haemophiLia PHysiotherapy INtervention for optimum musculoskeletal health in children (DOLPHIN-II Trial)
IRAS ID
282478
Contact name
David Stephensen
Contact email
Sponsor organisation
East Kent Hospitals University NHS Foundation Trust
ISRCTN Number
ISRCTN13911947
Duration of Study in the UK
2 years, 3 months, 1 days
Research summary
Research Summary:
What the study was about?
Haemophilia is a rare, inherited condition where blood doesn't clot properly causing bleeding in joints and muscles, leading to long-term damage, pain, and movement problems. Some people think special exercises to make muscles stronger might help. Stronger muscles could help children move better, protect their joints, and bleed less. But we don't really know if this works.
How the study was done?
We split children with haemophilia into two groups. One group did the special muscle-strengthening exercises online. The other group had their usual physiotherapy. We also talked to children, their families, and physiotherapists about the exercise program.
What the study found?
38 children took part (our aim was 132). 18 did the exercises. The children were around 9 years old. Six/thirty-eight children dropped out. After 3 and 6 months, we didn't see any big differences between the two groups in how strong their muscles were, how well they could move, or their quality of life. We did find that we could safely and reliably check how well children could move using video calls.
What the results mean?
We didn't get as many children in the study as we wanted. Hospitals told us this was because there weren't enough physiotherapists. We didn't find any clear benefits from the exercises. This might be because the exercises were done online and not 'in person' as originally intended. We need to be careful about these results because of the small number of children, some dropped out, their joint problems weren't too bad, and age might have affected the results.
What's next?
We are still looking at how active the children were and how age might have affected the results. We also want to do this study in poorer countries where children often have worse joint problems from haemophilia.Haemophilia is an inherited condition affecting 1:10000 people where the blood does not clot normally, leading to bleeding into muscles and joints. As a result, muscles become weak. Joints become painful and difficult to move. “Being able to participate in games and activities with their friends” is one of the things that matters most to boys with haemophilia. “What is the role of exercise for both prevention and treatment of joint damage in haemophilia?” is one of the top unanswered questions that concern patients, carers and healthcare professionals most. At present, there is a lack of evidence whether muscle strengthening exercise improves or negatively affects outcomes for young children with haemophilia. With the help of boys with haemophilia, their parents and physiotherapists we developed an exercise programme designed to increase muscle strength. We recently showed the exercise programme had no harmful effects and was acceptable to children with haemophilia. To answer the question, “does muscle strengthening help improve the long-term health of children with haemophilia?”, we will allocate 66 boys with haemophilia to a group that is asked to complete the 12-week exercise routine to strengthen their leg muscles and another 66 boys to a group that does not do the exercises. The boys will be allocated at random, so that each boy has an equal chance of being in either group. We will monitor the boys throughout the study and for another 12 weeks afterwards by measuring their muscle strength, how far they can walk in six-minutes, how far they can hop, how high they can jump and time taken to ascend and descend 12 steps. We will also record how physically active the boys are using a wrist band as well as how satisfied they are with their health.
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
20/SW/0154
Date of REC Opinion
30 Oct 2020
REC opinion
Favourable Opinion