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

该项目旨在开发和测试脑瘫(CP)儿童的生活质量指标。这是一个具有国际意义的新项目，在国际CP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重点。脑性瘫痪仍然是导致儿童肢体残疾的最常见原因，每1000名活产儿中的发病率为2-2.0-2.5。脑性瘫痪被描述为“在婴儿期或儿童期发现的非进行性中枢运动障碍”，脑性瘫痪表现为脑部的静态损害，其特征是进行性肌肉骨骼畸形。它对儿童和家庭的影响是深远的，造成照顾家庭的广泛和终生负担，并严重限制儿童的发展和福祉。神经肌肉后遗症和健康问题的管理对卫生系统来说是一笔相当大的费用，因为儿童需要经常前往医院进行医疗管理、外科手术和康复。CP管理有效性的试验因缺乏患者结局衡量标准而受阻。虽然新的治疗方案旨在提供损害和功能的实质性改善，但它们也有缺点。例如，痉挛的治疗包括A型肉毒杆菌毒素和鞘内巴氯芬，两者都可以改善功能，但代价昂贵，且具有侵入性；对下地活动的治疗(多节段矫形手术)可改善步态和灵活性，但需要广泛的康复；对严重进食困难和生长不良的治疗(胃造瘘术)可能会提高存活率，但会导致胃食道反流加重；顽固性癫痫的手术可能会改善癫痫发作障碍，但会导致功能障碍。生活质量现在是临床试验研究的强制性组成部分；迫切需要有效和可靠的工具来检测变化。

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.