Department of Thoracic Medicine, The Prince Charles Hospital and QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia.
Jena University Hospital, Jena, Germany.
BMC Pulm Med. 2019 Aug 13;19(1):146. doi: 10.1186/s12890-019-0887-6.
Clinical studies demonstrate that ivacaftor (IVA) improves health-related quality of life (HRQoL) in patients aged ≥6 years with cystic fibrosis (CF). The real-world impact of IVA and standard of care (SOC) in groups of patients with G551D and F508del mutations, respectively, was assessed using a survey comprising disease-specific and generic HRQoL measures.
Patients with CF aged ≥12 years, or aged 6-11 years with caregiver support, with either (1) a G551D mutation and receiving IVA (G551D/IVA) for ≥3 months, or (2) homozygous for F508del and receiving SOC before lumacaftor/IVA availability (F508del/SOC), were eligible to participate in a cross-sectional survey. Demographic and clinical characteristics, and HRQoL measures were compared between patient groups, and multiple regression analyses were conducted.
After differences in patient demographic and clinical characteristics were controlled for, significantly better scores were observed in the G551D/IVA group than in the F508del/SOC group on multiple domains of the validated Cystic Fibrosis Questionnaire-Revised and the EuroQol 5-dimensions 5-level questionnaire.
G551D/IVA patients reported better HRQoL than F508del/SOC patients on generic and disease-specific measures in a real-world setting.
临床研究表明，依伐卡托（IVA）可改善年龄≥6 岁囊性纤维化（CF）患者的健康相关生活质量（HRQoL）。使用包含疾病特异性和通用 HRQoL 测量的调查评估了 IVA 和标准治疗（SOC）分别在 G551D 和 F508del 突变患者群体中的实际影响。
年龄≥12 岁且有 CF 的患者，或有照料者支持且年龄为 6-11 岁的患者，满足以下条件之一即可参与横断面调查：（1）G551D 突变并接受 IVA（G551D/IVA）治疗≥3 个月，或（2）纯合 F508del 突变并在 lumacaftor/IVA 上市前接受 SOC（F508del/SOC）治疗。调查比较了患者群体的人口统计学和临床特征以及 HRQoL 测量值，并进行了多元回归分析。
在控制了患者人口统计学和临床特征的差异后，在验证后的囊性纤维化问卷修订版和欧洲五维健康量表 5 级问卷的多个领域，G551D/IVA 组的评分明显高于 F508del/SOC 组。
在真实环境中，G551D/IVA 患者在通用和疾病特异性测量上报告的 HRQoL 优于 F508del/SOC 患者。
