Respiratory and Sleep Medicine, Perth Children's Hospital, Nedlands, Western Australia, Australia
Cerebral Palsy Alliance, Forestville, New South Wales, Australia.
BMJ Open. 2024 Feb 15;14(2):e076730. doi: 10.1136/bmjopen-2023-076730.
The most common cause of morbidity and mortality in children with severe cerebral palsy (CP) is respiratory disease. BREATHE-CP (Better REspiratory and Airway Treatment and HEalth in Cerebral Palsy) is a multidisciplinary research team who have conducted research on the risk factors associated with CP respiratory disease, a systematic review on management and a Delphi study on the development of a consensus for the prevention and management of respiratory disease in CP. These strategies have not been investigated; therefore, it is not known if implementation is feasible, if they improve patient outcomes or if they are acceptable for families.
Mixed-method feasibility pilot randomised controlled trial with economic analysis. Twenty children with CP aged 0-12 years who are at risk of respiratory disease will be followed up for 1 year. All children will receive baseline assessments for comparison. The control group will receive usual care from their treating teams. The intervention group will receive comprehensive assessments from physiotherapy, speech pathology and respiratory medicine. An individualised investigation and treatment plan will then be made. Participants in both groups will complete fortnightly patient-reported outcome surveys to assess symptoms and health service use. Analysis will include assessments of acceptability through qualitative interviews, implementation by ability to recruit, randomise and retain, practicality including costs of intervention and hospitalisation, and explore efficacy through quality-of-life surveys and decreased health service use for respiratory-related symptoms.
Ethics and governance approvals have been obtained through Child and Adolescent Health Service Human Research Ethics Committee. At completion, this study will lead to the design of the definitive protocol to test intervention efficacy that maximises recruitment, retention and adherence to interventions.
Australian New Zealand Clinical Trials Registry (ACTRN12620000114943).
严重脑瘫（CP）儿童发病率和死亡率最高的原因是呼吸道疾病。BREATHE-CP（更好的呼吸和气道治疗以及脑瘫健康）是一个多学科研究团队，他们对与 CP 呼吸道疾病相关的危险因素进行了研究，对管理进行了系统评价，并通过 Delphi 研究就 CP 呼吸道疾病的预防和管理达成了共识。这些策略尚未得到调查；因此，尚不清楚实施是否可行，是否能改善患者的结局，或者是否能被家庭接受。
混合方法可行性先导随机对照试验，同时进行经济分析。20 名 0-12 岁有呼吸道疾病风险的 CP 儿童将接受为期 1 年的随访。所有儿童都将进行基线评估以作比较。对照组将接受他们治疗团队的常规护理。干预组将接受来自物理治疗师、言语病理学家和呼吸内科医生的全面评估。然后将制定个体化的调查和治疗计划。两组参与者都将完成每两周一次的患者报告结果调查，以评估症状和卫生服务的使用情况。分析将包括通过定性访谈评估可接受性、通过招募、随机化和保留能力评估实施情况、通过干预和住院费用的实用性评估、通过生活质量调查和减少与呼吸道相关症状的卫生服务使用情况评估疗效。
已通过儿童和青少年健康服务人体研究伦理委员会获得伦理和治理批准。完成后，本研究将设计出最大程度提高招募、保留和坚持干预措施的确定性方案，以测试干预的疗效。
澳大利亚新西兰临床试验注册中心（ACTRN12620000114943）。
