Medical University of South Carolina, 96 Jonathan Lucas Street, 812-CSB, Charleston, SC 29425, USA.
Formerly of Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA 02210, USA.
J Cyst Fibros. 2019 Sep;18(5):737-742. doi: 10.1016/j.jcf.2018.12.004. Epub 2018 Dec 23.
We explored the time-dependent impact of pulmonary exacerbations (PEx) on health-related quality of life (HRQoL) using Cystic Fibrosis Questionnaire-Revised (CFQ-R) data from 2 large cystic fibrosis (CF) trials.
This exploratory post-hoc analysis evaluated the impact of PEx on CFQ-R domains of functioning in 80 patients with CF (homozygous for F508del-CFTR), aged ≥14 years randomized to placebo in the TRAFFIC and TRANSPORT trials who experienced 1 PEx.
Scores on the CFQ-R were significantly lower within 1 week of PEx start in 8 out of 12 domains (Respiratory Symptoms, Physical Functioning, Emotional Functioning, Health Perceptions, Role Functioning, Social Functioning, Eating, and Vitality). Patients whose PEx was treated with hospitalization or intravenous antibiotics had greater reductions in some domains of HRQoL compared with those treated with oral antibiotics. In the immediate weeks post-PEx, improvement was seen on Emotional Functioning, Respiratory Symptoms, and Health Perceptions, while further decline was seen for Eating, Physical Functioning, Role Functioning, Vitality, and Weight. For some measures (Physical Functioning, Vitality), full recovery to pre-PEx levels took several weeks.
Pulmonary exacerbations have significant effects on multiple domains of HRQoL, and recovery across multiple domains post-PEx can take several weeks. These findings provide insight into the impact of PEx on patient HRQoL and recovery post-PEx.
ClinicalTrials.gov identifiers, NCT01807923 and NCT01807949.
我们利用来自两项大型囊性纤维化（CF）试验的囊性纤维化问卷修订版（CFQ-R）数据，探索了肺部恶化（PEx）对健康相关生活质量（HRQoL）的时间依赖性影响。
这项探索性的事后分析评估了 80 名携带 F508del-CFTR 纯合子 CF 患者（年龄≥14 岁）在 TRAFFIC 和 TRANSPORT 试验中接受安慰剂治疗，经历了 1 次 PEx 后对 CFQ-R 功能领域的影响。
在 12 个领域中的 8 个领域中，在 PEx 开始后 1 周内，CFQ-R 的评分明显降低（呼吸道症状、身体机能、情绪功能、健康感知、角色功能、社会功能、饮食和活力）。与接受口服抗生素治疗的患者相比，接受住院或静脉用抗生素治疗的患者在某些 HRQoL 领域的评分下降幅度更大。在 PEx 后立即的几周内，情绪功能、呼吸道症状和健康感知有所改善，而饮食、身体机能、角色功能、活力和体重则进一步下降。对于某些指标（身体机能、活力），需要几周时间才能完全恢复到 PEx 前的水平。
肺部恶化对 HRQoL 的多个领域有显著影响，而 PEx 后多个领域的恢复可能需要数周时间。这些发现为了解 PEx 对患者 HRQoL 的影响和 PEx 后恢复情况提供了深入的见解。
ClinicalTrials.gov 标识符，NCT01807923 和 NCT01807949。
