Development of a health-related quality of life instrument for children with cerebral palsy

脑瘫儿童健康相关生活质量测试仪的开发

• 批准号: nhmrc : 284514
• 负责人: Prof Andrew Mackinnon
• 金额: $ 7.6万
• 依托单位: Deakin University
• 依托单位国家: 澳大利亚
• 项目类别: NHMRC Project Grants
• 财政年份: 2004
• 资助国家: 澳大利亚
• 起止时间: 2004-01-01 至 2005-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/development-health-related-cerebral-palsy/101417
• 关键词: Development; health; related; quality; life

This project aims to develop and test a measure of quality of life for children with cerebral palsy (CP). This is a new project of international significance that has been recommended as the highest research priority of the United Cerebral Palsy Association with the strong support of CP researchers and clinicians internationally. CP remains the most common cause of physical disability in childhood, with an incidence of 2-2.0-2.5 per 1,000 live births. Described as a 'non-progressive motor impairment of central origin recognised in infancy or childhood', CP presents as a static lesion on the brain characterised by progressive muscoskeletal deformity. Its impact on children and families is profound, resulting in extensive and life-long burden of care for families, and significant limitations to children's development and wellbeing. The management of the neuromuscular sequelae and health problems is a considerable cost to the health system because children require frequent visits for medical management, surgical procedures and rehabilitation. Trials of CP management effectiveness are hampered by the absence of patient outcome measures. Whilst new treatment options aim to provide substantial improvements in impairment and functioning they have disadvantages. For example, spasticity management includes Botulinum toxin A and intrathecal baclofen, both may improve function but are costly and invasive; treatments for ambulation (multi-level orthopaedic surgery) offer improved gait and mobility but require extensive rehabilitation; treatments for severe eating difficulties and poor growth (gastrostomy) may improve survival but result in aggravation of gastro-oesophageal reflux; and surgery for intractable epilepsy may improve seizure disorder but result in functional deficits. Quality of life is now a mandatory component of clinical trial research; valid and reliable tools sensitive to detecting change are urgently required.

该项目旨在开发和测试脑瘫儿童（CP）生活质量的衡量标准。这是一个具有国际意义的新项目，在国际脑瘫研究人员和临床医生的大力支持下，被推荐为联合脑瘫协会的最高研究优先事项。CP仍然是儿童期身体残疾的最常见原因，发病率为每1，000名活产中2-2.0-2.5例。CP被描述为“在婴儿期或儿童期发现的中枢起源的非进行性运动障碍”，表现为大脑上的静态病变，其特征为进行性肌肉骨骼畸形。它对儿童和家庭的影响是深远的，给家庭带来了广泛和终身的照顾负担，并严重限制了儿童的发展和福祉。神经肌肉后遗症和健康问题的治疗对卫生系统来说是一笔相当大的费用，因为儿童需要经常就诊，以便进行医疗管理、外科手术和康复。CP管理有效性的试验因缺乏患者结局指标而受到阻碍。虽然新的治疗选择旨在提供损伤和功能的实质性改善，但它们具有缺点。例如，痉挛管理包括肉毒杆菌毒素A和鞘内巴氯芬，两者都可以改善功能，但昂贵且侵入性;（多节段骨科手术）提供改善的步态和活动能力，但需要广泛的康复;治疗严重的进食困难和生长不良（胃造口术）可能会提高生存率，但会导致胃食管反流加重;顽固性癫痫的手术可能会改善癫痫发作，但会导致功能障碍。生活质量现在是临床试验研究的一个强制性组成部分;迫切需要对检测变化敏感的有效和可靠的工具。