Solem Caitlyn T, Vera-Llonch Montserrat, Liu Sizhu, Botteman Marc, Castiglione Brenda
Pharmerit International, 4350 East West Hwy, Suite 430, Bethesda, MD, 20814, USA.
Vertex Pharmaceuticals, 50 Northern Ave, Boston, MA, 02210, USA.
Health Qual Life Outcomes. 2016 Apr 21;14:63. doi: 10.1186/s12955-016-0465-z.
The analysis aimed to examine the impact of pulmonary exacerbations (PEs) and lung function on generic measures of HRQL in patients with cystic fibrosis (CF) using trial-based data.
In a 48-week randomized, placebo-controlled study of ivacaftor in patients ≥12 years with CF and a G551D-CFTR mutation the relationship between PEs, PE-related hospitalizations and percent predicted forced expiratory volume in one second (ppFEV1) with EQ-5D measures (index and visual analog scale [VAS]) was examined in post-hoc analyses. Multivariate mixed-effects models were employed to describe the association of PEs, PE-related hospitalizations, and ppFEV1 on EQ-5D measures.
One hundred sixty one patients (age: mean 25.5 [SD 9.5] years; baseline ppFEV1: 63.6 [16.4]) contributed 1,214 observations (ppFEV1: no lung dysfunction [n = 157], mild [n = 419], moderate [n = 572], severe [n = 66]). Problems were most frequently reported on pain/discomfort, anxiety/depression, and usual activities EQ-5D items. The mean (SE) EQ-5D index nominally decreased (worsened) with worsening severity of lung dysfunction (P = 0.070): 0.931 (0.023); mild: 0.923 (0.021); moderate: 0.904 (0.018); severe: 0.870 (0.020). 146 PEs were experienced by 72 patients, including 52 PEs (35.6 %) that required hospitalization. Mean EQ-5D index and VAS scores were lowest (worst) within 1 week (before or after PE start) for PEs requiring hospitalization. Pulmonary exacerbations, PE-related hospitalizations, and ppFEV1 were significant predictors of EQ-5D index and VAS.
In a clinical study of patients with CF (≥12 years of age and a G551D-CFTR mutation), PEs, primarily those requiring hospitalization, were associated with low EQ-5D index and VAS scores. The impact of ppFEV1 was relatively smaller. Reducing PEs, in particular those requiring hospitalization, would likely improve HRQL among these patients.
ClinicalTrials.gov, NCT00909532 ; URL: clinicaltrials.gov, May 26, 2009.
本分析旨在利用基于试验的数据，研究肺部加重（PEs）和肺功能对囊性纤维化（CF）患者健康相关生活质量（HRQL）通用指标的影响。
在一项针对年龄≥12岁、携带G551D - CFTR突变的CF患者进行的为期48周的依伐卡托随机、安慰剂对照研究中，在事后分析中研究了PEs、与PE相关的住院治疗以及一秒用力呼气容积预测值百分比（ppFEV1）与EQ - 5D指标（指数和视觉模拟量表[VAS]）之间的关系。采用多变量混合效应模型来描述PEs、与PE相关的住院治疗以及ppFEV1与EQ - 5D指标之间的关联。
161名患者（年龄：平均25.5[标准差9.5]岁；基线ppFEV1：63.6[16.4]）提供了1214次观察数据（ppFEV1：无肺功能障碍[n = 157]，轻度[n = 419]，中度[n = 572]，重度[n = 66]）。在疼痛/不适、焦虑/抑郁和日常活动的EQ - 5D项目上报告问题的频率最高。随着肺功能障碍严重程度的加重，EQ - 5D指数均值（标准误）名义上下降（恶化）（P = 0.070）：无肺功能障碍：0.931（0.023）；轻度：0.923（0.021）；中度：0.904（0.018）；重度：0.870（0.020）。72名患者经历了146次PEs，其中52次（35.6%）需要住院治疗。对于需要住院治疗的PEs，EQ - 5D指数和VAS评分在1周内（PE开始前或后）最低（最差）。肺部加重、与PE相关的住院治疗以及ppFEV1是EQ - 5D指数和VAS的显著预测因素。
在一项针对CF患者（≥12岁且携带G551D - CFTR突变）的临床研究中，PEs，主要是那些需要住院治疗的PEs，与低EQ - 5D指数和VAS评分相关。ppFEV1的影响相对较小。减少PEs，尤其是那些需要住院治疗 的PEs，可能会改善这些患者的HRQL。
ClinicalTrials.gov，NCT00909532；网址：clinicaltrials.gov，2009年5月26日。
