Assessment of health-related quality of life in children: A review of conceptual, methodological, and regulatory issues
Assessment of health-related quality of life in children: A review of conceptual, methodological, and regulatory issues
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DOI：
10.1111/j.1524-4733.2004.71273.x
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发表时间：
2004-01-01
期刊：
影响因子：
4.5
通讯作者：
Leidy, NK
中科院分区：
文献类型：
--
作者：
Matza, LS;Swensen, AR;Leidy, NK
最近，FDA鼓励在药物开发期间在儿科患者中进行药物测试。制药公司通过在儿童中进行更多的临床试验做出了回应，研究人员开始意识到评估儿科健康结果的独特挑战，包括健康相关的生活质量（HRQL）。与成人一样，儿童经历的疾病和治疗的影响超出了生理结果。进一步的儿科HRQL研究是必要的，以检查这些更广泛的心理社会结果，并提供一个彻底的了解治疗对儿童的健康状况的影响。目前审查的目的是讨论关键的监管和方法的发展，并为未来的研究提供指导儿科HRQL。儿科HRQL评估的这篇综述包括五个部分：1）美国最近儿科监管的发展; 2）定义和概念化儿科HRQL的问题，包括家庭和同伴系统等背景变量的重要性; 3）方法学问题（例如，代理问题，发展差异，反应集），并建议在临床试验中解决这些问题; 4）验证通用和特定条件的儿科HRQL措施;和5）建议对儿童精神疾病的HRQL影响进行额外的研究。建议定期对儿童的HRQL进行评估，作为药物开发的一个组成部分。
Recently, the FDA has encouraged testing of medications among pediatric patients during drug development. Pharmaceutical companies have responded by conducting more clinical trials among children, and researchers are becoming aware of the unique challenges of assessing pediatric health outcomes, including health-related quality of life (HRQL). Like adults, children experience effects of illness and treatment beyond physiologic outcomes. Further pediatric HRQL research is necessary to examine these broader psychosocial outcomes and provide a thorough understanding of the effects of treatment on children's health status. The purpose of the current review is to discuss key regulatory and methodologic developments and provide guidance for future research on pediatric HRQL. This review of pediatric HRQL assessment includes five sections: 1) recent pediatric regulatory developments in the United States; 2) issues in defining and conceptualizing pediatric HRQL, including the importance of contextual variables such as family and peer systems; 3) methodologic issues (e.g., the proxy question, developmental differences, response sets) with recommendations for addressing these issues in clinical trials; 4) validated generic and condition-specific pediatric HRQL measures; and 5) a recommendation for additional research on the HRQL impact of childhood psychiatric disorders. It is advocated that assessment of HRQL among children should be conducted regularly as an integral part of drug development.