Division of Respiratory Medicine, The Hospital for Sick Children, Toronto, Ontario, Canada; Department of Paediatrics, University of Toronto, Toronto, Canada; Translational Medicine Program, SickKids Research Institute, Toronto, Canada; Royal College of Surgeons in Ireland, Dublin, Ireland; National Children's Research Centre, Crumlin, Dublin, Ireland.
Division of Respiratory Medicine, The Hospital for Sick Children, Toronto, Ontario, Canada; Department of Paediatrics, University of Toronto, Toronto, Canada; Translational Medicine Program, SickKids Research Institute, Toronto, Canada.
J Cyst Fibros. 2020 May;19(3):492-498. doi: 10.1016/j.jcf.2020.02.017. Epub 2020 Mar 2.
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory score is a validated and widely used patient-reported outcome. This study aimed to establish changes in the score with acute respiratory events in preschool children with CF and to investigate its' relationship with physiological outcomes.
The Parent CFQ-R, multiple breath washout test and spirometry were performed at six study visits over one year. The clinical status of participants, stable or symptomatic, was defined by the patient's physician. Linear regression and distribution-based statistical methods were used to examine the changes in the CFQ-R from the last stable visit and to investigate its relationship with physiological outcomes.
There were 272 stable and 115 symptomatic visits from 78 participants. The mean CFQ-R Respiratory score did not change between consecutive stable visits (-0.73, SD 20.4). The mean (SD) score deteriorated by 15.5 (20.7) points between stable and symptomatic visits and improved by 14.8 (20.1) points between symptomatic and stable follow-up visits. When a clinically important change is defined as 0.5SD change (10-points), the positive predictive value (PPV) was 45% and the negative predictive value (NPV) was 84%. For visits with a 10-point worsening in the CFQ-R Respiratory score and a 15% increase in LCI, the PPV was better (81%) than using either measure alone.
The CFQ-R Respiratory score is responsive to acute respiratory events in preschool children with CF and its utility to monitor individual patients is improved when combined with LCI.
囊性纤维化问卷修订版（CFQ-R）呼吸评分是一种经过验证且广泛使用的患者报告结局。本研究旨在确定 CF 学龄前儿童急性呼吸事件后评分的变化，并探讨其与生理结局的关系。
在一年的六次研究访问中，进行了父母 CFQ-R、多次呼吸冲洗试验和肺活量测定。参与者的临床状况（稳定或有症状）由患者的医生定义。线性回归和基于分布的统计方法用于检查从最后一次稳定访问到 CFQ-R 的变化，并研究其与生理结果的关系。
来自 78 名参与者的 272 次稳定和 115 次有症状访问。连续稳定访问之间的平均 CFQ-R 呼吸评分没有变化（-0.73，SD 20.4）。在稳定和有症状的访问之间，评分平均恶化 15.5（20.7）点，在有症状和稳定的随访访问之间，评分平均改善 14.8（20.1）点。当将临床重要变化定义为 0.5SD 变化（10 分）时，阳性预测值（PPV）为 45%，阴性预测值（NPV）为 84%。对于 CFQ-R 呼吸评分恶化 10 分且 LCI 增加 15%的访问，PPV 优于单独使用任何一种测量方法（81%）。
CFQ-R 呼吸评分对 CF 学龄前儿童的急性呼吸事件敏感，当与 LCI 结合使用时，其监测个体患者的效果得到改善。
