Alpern Adrianne N, Brumback Lyndia C, Ratjen Felix, Rosenfeld Margaret, Davis Stephanie D, Quittner Alexandra L
University of Miami, Department of Psychology, 5665 Ponce De Leon Blvd., Coral Gables, FL 33146, USA.
University of Washington, Campus Box 354922, Seattle, WA, USA.
J Cyst Fibros. 2015 May;14(3):403-11. doi: 10.1016/j.jcf.2014.11.002. Epub 2014 Nov 28.
There is an urgent need to evaluate treatments for young children with cystic fibrosis (CF); however, efforts have been hampered by a lack of reliable, practical endpoints. To examine whether a patient-reported outcome could be reliable in children 4 to 60 months of age, we assessed the psychometric properties of the modified Parent Cystic Fibrosis Questionnaire--Revised (CFQ-R) using data from the Infant Study of Inhaled Saline (ISIS). We also characterized patterns of symptom presentation and daily functioning in children in this age range to inform future measure development.
Parents (N=314) completed the CFQ-R and Treatment Adherence Questionnaire (TAQ) at five quarterly visits, as well as a weekly Parent Symptom Diary.
The Parent CFQ-R demonstrated good construct validity and adequate internal consistency (α's .58-.75). Associations with age, TAQ, and Parent Symptom Diary were observed. The Treatment Burden scale demonstrated responsiveness to change.
Parents were reliable observers of young children's symptoms and daily functioning, and PROs show promise for this age group. Research is needed to identify key symptoms in infants and young children with CF, and to develop a parent proxy PRO according to FDA/EMA guidelines.
迫切需要评估针对患有囊性纤维化（CF）的幼儿的治疗方法；然而，由于缺乏可靠、实用的终点指标，相关工作受到了阻碍。为了检验患者报告结局在4至60个月大的儿童中是否可靠，我们使用吸入盐水婴儿研究（ISIS）的数据评估了修订后的改良版家长囊性纤维化问卷（CFQ-R）的心理测量特性。我们还描述了这个年龄范围内儿童的症状表现和日常功能模式，以为未来的测量工具开发提供参考。
314名家长在五次季度访视时完成了CFQ-R和治疗依从性问卷（TAQ），以及每周一次的家长症状日记。
家长CFQ-R显示出良好的结构效度和足够的内部一致性（α系数为0.58 - 0.75）。观察到与年龄、TAQ和家长症状日记之间的关联。治疗负担量表显示出对变化的反应性。
家长是幼儿症状和日常功能的可靠观察者，患者报告结局对这个年龄组有应用前景。需要开展研究以确定患有CF的婴幼儿的关键症状，并根据美国食品药品监督管理局/欧洲药品管理局的指南开发家长代理的患者报告结局。
