Protocol for a feasibility randomised controlled trial of a musculoskeletal exercise intervention versus usual care for children with haemophilia

Abstract

IntroductionHaemophilia is a rare, inherited disorder in which blood does not clot normally, resulting in bleeding into joints and muscles. Long-term consequence is disabling joint pain, stiffness, muscle weakness, atrophy and reduced mobility. The purpose of this proposed feasibility of a randomised controlled trial (RCT) is to test the feasibility of an age-appropriate physiotherapy intervention designed to improve muscle strength, posture and the way boys use their joints during walking and everyday activities.Methods and analysisA small-scale two-centre RCT of a 12-week muscle strengthening exercise intervention versus usual care for young children with haemophilia will be conducted. Primary outcomes will be safety and adherence to the exercise intervention. Secondary outcomes will include recruitment, retention and adverse event rates, clinical data, muscle strength, joint biomechanics and foot loading patterns during walking, 6 min timed walk, timed-up-and-down-stairs, EQ-5D-Y, participants’ perceptions of the study, training requirements and relevant costs. Recruitment, follow-up, safety and adherence rates will be described as percentages. Participant diary and interview data will be analysed using a framework analysis. Demographic and disease variable distributions will be analysed for descriptive purposes and covariant analysis. Estimates of differences between treatment arms (adjusted for baseline) and 75% and 95% CIs will be calculated.Ethics and disseminationThe study has ethical approval from the London—Fulham Research Ethics Committee (17/LO/2043) as well as Health Research Authority approval. As well as informing the design of the definitive trial, results of this study will be presented at local, national and international physiotherapy and haemophilia meetings as well as manuscripts submitted to peer-reviewed journals. We will also share the main findings of the study to all participants and the Haemophilia Society.

Influência do tratamento fisioterapêutico na independência funcional de pessoas com hemofilia submetidas à sinovectomia radioativa

“…Procedimentos como eletroterapia, crioterapia e cinesioterapia foram relatados, intervenções que apresentam evidências publicadas na utilização em pessoas com hemofilia. 9 Quanto ao local de realização da SR, nossa amostra total difere dos estudos em geral, que apontam a articulação do joelho como a mais comum, 12,16,18 na presente pesquisa o cotovelo foi segmento mais prevalente. A literatura relata que os resultados da SR para a articulação dos joelhos são piores em relação às outras articulações menores, como tornozelo e cotovelo, respectivamente; o que faz com que as articulações dos joelhos sejam mais prevalentes e necessitem de mais de uma aplicação, muitas vezes devido à magnitude da articulação.…”

“…6 Dessa maneira, a sinovite crônica e hemartroses recorrentes em uma articulação-alvo geram quadro clínico de dor e aumento do volume articular, evoluindo para rigidez, fraqueza muscular, deformidades e mobilidade reduzida com danos irreversíveis à cartilagem e perda funcional importante, caracterizando a artropatia hemofílica. 8,9 Em vista disso, o melhor manejo para evitar a artropatia hemofílica é a prevenção das hemartroses recidivantes. Para isto, o aspecto central do tratamento da hemofilia é a administração regular intravenosa profilática de concentrado de fator de coagulação e fisioterapia com objetivo de evitar sangramentos repetitivos e prevenir a degeneração progressiva da articulação.…”

Muscle strengthening intervention for boys with haemophilia: Developing and evaluating a best‐practice exercise programme with boys, families and health‐care professionals

“…The current findings discussed in this paper report upon a recently completed feasibility study funded by the UK’s National Institute for Health Research (NIHR) known as the ‘DOLPHIN’ study or D evelopment O f a haemophi L ia P hysiotherapy IN tervention for optimum musculoskeletal health (PB‐PG‐0215‐36091) 15 . This paper describes how a muscle strengthening intervention was designed, developed and refined for boys with haemophilia.…”

Results of feasibility and safety of randomised controlled trial of a musculoskeletal exercise intervention versus usual care for children with haemophilia

“…The purpose of this two-centre feasibility randomized controlled trial (RCT) was to test the feasibility of an age-appropriate physiotherapy intervention co-designed by healthcare professionals and patients9 to improve muscle strength and physical function in pre-adolescent boys with haemophilia. Favourable ethical approval was obtained from the Health Research Authority and London-Fulham Research Ethics Committee and (17/LO/2043).…”