Seid Michael, Limbers Christine A, Driscoll Kimberly A, Opipari-Arrigan Lisa A, Gelhard Leticia Reyes, Varni James W
Division of Pulmonary Medicine and Center for Health Care Quality, Division of Health Policy and Clinical Effectiveness, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
J Asthma. 2010 Mar;47(2):170-7. doi: 10.3109/02770900903533966.
Patient-reported outcomes such as health-related quality of life (HRQOL) are increasingly used as primary endpoints in clinical trials. The Pediatric Quality of Life Inventory (PedsQL) is widely used as a measure of HRQOL and may be a particularly suitable primary outcome in pediatric asthma clinical trials.
To examine the reliability, validity, and responsiveness to clinical change of the PedsQL 4.0 Generic Core Scales and PedsQL Asthma Module Asthma Symptoms Scale in a sample of vulnerable children with persistent asthma recruited from Federally Qualified Health Centers.
Children (N = 252; ages 3 to 14 years) with persistent asthma (27% mild, 40.9% moderate, 32.1% severe) and their parents (93.7% mother, 83.3% Hispanic, 76.9% Spanish-speaking, 72.6% less than a high school diploma) enrolled in a clinical trial completed the PedsQL 4.0 Generic Core Scales, the PedsQL 3.0 Asthma Module Asthma Symptoms Scale, and a measure of asthma symptom frequency (used as an indicator of clinical change) at baseline and 3-month follow-up.
The PedsQL demonstrated adequate internal consistency reliability and convergent and discriminative validity. Based on intra- and intersubject change, effect sizes, and standard errors of measurement, the PedsQL demonstrated responsiveness to clinical change. Conclusions. For both child self-report and parent proxy-report, the PedsQL Generic Core Scales Total Scale score and the PedsQL Asthma Symptoms Scale are suitable for use as primary asthma clinical trial outcomes.
患者报告的结果，如健康相关生活质量（HRQOL），在临床试验中越来越多地被用作主要终点。儿童生活质量量表（PedsQL）被广泛用作HRQOL的衡量指标，可能是儿科哮喘临床试验中特别合适的主要结局。
在从联邦合格健康中心招募的易患持续性哮喘的儿童样本中，检验PedsQL 4.0通用核心量表和PedsQL哮喘模块哮喘症状量表的信度、效度以及对临床变化的反应性。
患有持续性哮喘（27%轻度、40.9%中度、32.1%重度）的儿童（N = 252；年龄3至14岁）及其父母（93.7%为母亲，83.3%为西班牙裔，76.9%说西班牙语，72.6%学历低于高中）参加了一项临床试验，在基线和3个月随访时完成了PedsQL 4.0通用核心量表、PedsQL 3.0哮喘模块哮喘症状量表以及哮喘症状频率测量（用作临床变化指标）。
PedsQL表现出足够的内部一致性信度以及收敛效度和区分效度。基于个体内和个体间变化、效应量和测量标准误差，PedsQL表现出对临床变化的反应性。结论。对于儿童自评和家长代理报告，PedsQL通用核心量表总分和PedsQL哮喘症状量表均适合用作哮喘临床试验的主要结局。
