Wei Yi, Speechley Kathy, Campbell Craig
Department of Pediatrics and Epidemiology and Biostatistics, Western University.
J Neuromuscul Dis. 2015 Sep 2;2(3):313-324. doi: 10.3233/JND-150071.
In pediatric chronic illness, improving health-related quality of life (HRQOL) has become one of the most important goals of disease management. Duchenne muscular dystrophy (DMD) is a debilitating, progressive and chronic neuromuscular disorder affecting boys. The purpose of this review is to provide an overview of published research on HRQOL in the pediatric DMD population, describe the instruments used and summarize the study findings. The databases searched were Medline, Embase and PsycInfo. The literature search yielded 167 articles, of which 19 were included in this review. The studies were published between 2005 and 2013 across nine countries. Thirteen different generic and disease-specific measures were used, the most common being the Pediatric Quality of Life 4.0 Generic Core module.HRQOL in boys with DMD is worse than that of healthy peers and children with other chronic illnesses, especially in the physical domains. Boys who are at a more severe stage of the disease reported worse physical HRQOL but not necessarily psychosocial HRQOL than boys at a less severe stage. Traditional clinical outcome measures correlated well only with physical HRQOL. Parents' proxy-reports of their sons' HRQOL and the boys' self-reports had poor concordance. More research is needed to assess trends in HRQOL over time and to elucidate factors that affect HRQOL.
在儿科慢性病中，改善健康相关生活质量（HRQOL）已成为疾病管理的最重要目标之一。杜氏肌营养不良症（DMD）是一种影响男孩的使人衰弱、进行性的慢性神经肌肉疾病。本综述的目的是概述已发表的关于儿科DMD患者HRQOL的研究，描述所使用的工具并总结研究结果。检索的数据库有Medline、Embase和PsycInfo。文献检索产生了167篇文章，其中19篇被纳入本综述。这些研究在2005年至2013年间发表于9个国家。使用了13种不同的通用和疾病特异性测量方法，最常用的是儿童生活质量4.0通用核心模块。DMD男孩的HRQOL比健康同龄人及其他慢性病患儿更差，尤其是在身体领域。疾病处于更严重阶段的男孩报告的身体HRQOL比病情较轻阶段的男孩更差，但心理社会HRQOL不一定更差。传统的临床结局测量仅与身体HRQOL有很好的相关性。父母对儿子HRQOL的代理报告与男孩的自我报告一致性较差。需要更多研究来评估HRQOL随时间的趋势，并阐明影响HRQOL的因素。
