Department of Respiratory and Sleep Medicine, Queensland Children's Hospital, Brisbane, Australia; Centre for Children's Health Research, School of Medicine, The University of Queensland, Australia.
Clinical Epidemiology and Biostatistics Unit, Murdoch Children's Research Institute, Melbourne, Australia; Department of Paediatrics, University of Melbourne, Melbourne, Australia.
J Cyst Fibros. 2020 May;19(3):483-491. doi: 10.1016/j.jcf.2020.02.022. Epub 2020 Mar 9.
The impact of early cystic fibrosis (CF) on health-related quality-of-life (HRQOL) in preschool children is poorly characterised, and data on relationships between HRQOL and health outcomes in young children with CF are limited. We aimed to characterise and compare parent-proxy and child-reported HRQOL and evaluate relationships with clinical outcomes at age 5-years.
Subjects were participating in the multi-centre Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) trial investigating BAL-directed versus standard CF therapy. Children aged 5-years and their parents rated HRQOL using the Pediatric Quality of Life Inventory (PedsQL™) and Cystic Fibrosis Questionnaire-Revised (CFQ-R) questionnaires.
PedsQL and CFQ-R questionnaires were completed by 141 primary caregivers and 135 and 130 children, respectively. There were no differences in HRQOL between children randomised to BAL-directed versus standard CF therapy. Children with CF rated worse HRQOL than healthy children and there was poor parent-child concordance across HRQOL domains. Nutritional status, CF-CT scan score, forced expiratory volume in 1-second (FEV), and pulmonary exacerbations correlated with HRQOL at age 5-years. FEV z-scores positively correlated with parent-proxy HRQOL in CFQ-R Respiratory (p = 0.018), Physical (<0.001), Emotional (p = 0.007) subscales and PedsQL Total-score (p = 0.021), Physical (p = 0.019) domains. Pulmonary exacerbation rates were inversely associated with parent-proxy CFQ-R Respiratory (p = 0.004), Physical (p = 0.022), PedsQL Total (p = 0.009) and Physical (p = 0.009) scores.
Parent-reported HRQOL is a meaningful clinical endpoint to evaluate interventions in young children. Parent and child HRQOL reports provide different, complementary information. A preschool version of the CFQ-R is needed to assess relationships between HRQOL and clinical outcomes in young children.
早期囊性纤维化（CF）对学龄前儿童健康相关生活质量（HRQOL）的影响特征描述不足，且有关 CF 幼儿 HRQOL 与健康结果之间关系的数据有限。我们旨在描述和比较父母代理和儿童报告的 HRQOL，并评估其与 5 岁时临床结局的关系。
参与多中心澳大利亚囊性纤维化支气管肺泡灌洗（ACFBAL）试验的研究对象正在研究 BAL 定向与标准 CF 治疗。5 岁儿童及其父母使用儿科生活质量量表（PedsQL）和囊性纤维化问卷修订版（CFQ-R）问卷评估 HRQOL。
141 名主要照顾者完成了 PedsQL 和 CFQ-R 问卷，分别有 135 名和 130 名儿童完成了这些问卷。BAL 定向与标准 CF 治疗之间的 HRQOL 无差异。CF 儿童的 HRQOL 评分差于健康儿童，且 HRQOL 各领域父母与儿童的一致性较差。营养状况、CF-CT 评分、1 秒用力呼气量（FEV）和肺部恶化与 5 岁时的 HRQOL 相关。FEV z 评分与 CFQ-R 呼吸（p=0.018）、身体（p<0.001）、情绪（p=0.007）分量表和 PedsQL 总分（p=0.021）以及身体（p=0.019）领域的父母代理 HRQOL 呈正相关。肺部恶化发生率与父母代理 CFQ-R 呼吸（p=0.004）、身体（p=0.022）、PedsQL 总分（p=0.009）和身体（p=0.009）评分呈负相关。
父母报告的 HRQOL 是评估幼儿干预措施的有意义的临床终点。父母和儿童的 HRQOL 报告提供了不同的、互补的信息。需要一个学前版的 CFQ-R 来评估幼儿 HRQOL 与临床结局之间的关系。
