Centre for Health Service Studies, University of Kent, Canterbury, Kent, UK.
Haemophilia Centre, Great Ormond Street Hospital For Children NHS Foundation Trust, London, UK.
BMJ Open. 2019 Aug 1;9(8):e029474. doi: 10.1136/bmjopen-2019-029474.
Haemophilia 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.
A 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.
The 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.
血友病是一种罕见的遗传性疾病，血液无法正常凝结，导致关节和肌肉出血。长期后果是关节疼痛、僵硬、肌肉无力、萎缩和活动能力下降。本研究旨在测试一项针对血友病儿童的适合年龄的物理治疗干预措施的可行性，该措施旨在改善肌肉力量、姿势以及男孩在行走和日常活动中使用关节的方式。
将进行一项为期 12 周的肌肉强化运动干预与常规护理的小型两中心 RCT，以评估该干预措施对患有血友病的幼儿的安全性和依从性。主要结果将是安全性和对运动干预的依从性。次要结果将包括招募、保留和不良事件率、临床数据、肌肉力量、关节生物力学和行走时的足部负荷模式、6 分钟计时步行、上下楼梯计时、EQ-5D-Y、参与者对研究的看法、培训要求和相关成本。将以百分比形式描述招募、随访、安全性和依从性的比率。将使用框架分析对参与者日记和访谈数据进行分析。将对人口统计学和疾病变量分布进行分析，以进行描述性分析和协变量分析。将计算治疗组之间的差异估计值（根据基线进行调整）和 75%和 95%CI。
该研究已获得伦敦富勒姆研究伦理委员会（17/LO/2043）以及卫生保健研究管理局的伦理批准。除了为确定试验的设计提供信息外，本研究的结果还将在当地、国家和国际物理治疗和血友病会议上以及提交给同行评审期刊的手稿中进行介绍。我们还将向所有参与者和血友病协会分享该研究的主要发现。
