Fajac Isabelle, Daines Cori, Durieu Isabelle, Goralski Jennifer L, Heijerman Harry, Knoop Christiane, Majoor Christof, Bruinsma Bote G, Moskowitz Samuel, Prieto-Centurion Valentin, Van Brunt Kate, Zhang Yaohua, Quittner Alexandra
APHP.Centre-Université de Paris, Paris, France.
University of Arizona, Banner University Medical Center, Tucson, AZ, US.
J Cyst Fibros. 2023 Jan;22(1):119-123. doi: 10.1016/j.jcf.2022.08.018. Epub 2022 Sep 14.
Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) heterozygous for F508del and a minimal function mutation (F/MF) or homozygous for F508del (F/F) in two pivotal Phase 3 trials, significantly improving percentage predicted forced expiratory volume in 1 second, Cystic Fibrosis Questionnaire-Revised, Respiratory Domain (CFQ-R RD) scores, and sweat chloride concentration. Here, we analyzed the 11 non-respiratory domains (non-RDs) of the CFQ-R, which assess general health-related quality of life (i.e., Physical Functioning, Role Functioning, Vitality, Health Perceptions, Emotional Functioning, and Social Functioning) and quality of life impacted by CF (i.e., Body Image, Eating Problems, Treatment Burden, Weight, and Digestive Symptoms), for participants in these two Phase 3 trials. ELX/TEZ/IVA treatment led to higher scores in all CFQ-R non-RDs, with improvements in most domains compared with control treatments. These findings demonstrate that ELX/TEZ/IVA improves a range of CF-specific symptoms and general functioning and well-being.
在两项关键的3期试验中，依列卡福妥/替扎卡福妥/依伐卡福妥（ELX/TEZ/IVA）对携带F508del杂合子和最小功能突变（F/MF）或F508del纯合子（F/F）的囊性纤维化（CF）患者显示出安全有效，显著提高了1秒用力呼气量预测百分比、囊性纤维化问卷修订版呼吸领域（CFQ-R RD）评分和汗液氯化物浓度。在此，我们分析了这两项3期试验参与者的CFQ-R的11个非呼吸领域（非RD），这些领域评估与总体健康相关的生活质量（即身体功能、角色功能、活力、健康认知、情绪功能和社会功能）以及受CF影响的生活质量（即身体形象、饮食问题、治疗负担、体重和消化症状）。与对照治疗相比，ELX/TEZ/IVA治疗使所有CFQ-R非RD的得分更高，大多数领域都有改善。这些发现表明，ELX/TEZ/IVA改善了一系列CF特异性症状以及总体功能和幸福感。
