Allgood Sarah, Levy Reena, Bubaris Despina, Riekert Kristin, Psoter Kevin J, Lechtzin Noah
Johns Hopkins University School of Nursing, USA.
Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Heliyon. 2023 Sep 16;9(9):e20110. doi: 10.1016/j.heliyon.2023.e20110. eCollection 2023 Sep.
Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a CFTR modulator that has led to large benefits in lung function, pulmonary exacerbation rates, and respiratory symptoms. Less is known about the effect of ETI on non-pulmonary symptoms. The objective of this study was to examine the changes in patient reported outcomes after starting ETI in multiple non-pulmonary symptoms.
This was a prospective cohort study of adults with CF. Participants completed questionnaires prior to starting ETI and then at weeks 2, 4, 6, 8, 10, 12, and 14 after starting ETI. They completed the following validated instruments: PROMIS Pain Intensity, PROMIS Pain Interference, FACIT Fatigue, SNOT22, PAC-SYM, PHQ8, GAD7 and Pittsburgh Sleep Quality Index. Longitudinal changes for outcomes were modelled using linear regression based on general estimating equations.
22 participants enrolled who answered questionnaires before and after starting ETI. The median age was 35.3 years (IQR 11.1) and 13 (59.1%) were male. In models adjusted for age, sex, and baseline value there were significant improvements in pain interference (β = -2.57; 95% CI -4.92, -0.23), sinus symptoms (β = -4.50; 95% CI -7.59, -1.41), and sleep disturbance (β = -1.90; 95% CI -2.71, -1.09) over 14 weeks after starting ETI. No symptom areas worsened over the study period.
In this prospective study we found statistically significant improvements in three different non-pulmonary symptom areas in people with CF started on ETI. While this was a small, uncontrolled study it suggests that use of highly effective CFTR modulators can result in benefits for patients beyond pulmonary symptoms.
依列卡福妥/替扎卡福妥/依伐卡托（ETI）是一种CFTR调节剂，已在肺功能、肺部恶化率和呼吸道症状方面带来了巨大益处。关于ETI对非肺部症状的影响，人们了解较少。本研究的目的是检查开始使用ETI后多种非肺部症状患者报告结局的变化。
这是一项针对成年囊性纤维化（CF）患者的前瞻性队列研究。参与者在开始使用ETI之前以及开始使用ETI后的第2、4、6、8、10、12和14周完成问卷。他们完成了以下经过验证的工具：患者报告结果测量信息系统疼痛强度量表、患者报告结果测量信息系统疼痛干扰量表、功能性评估慢性疾病治疗疲劳量表、鼻窦症状测试22项问卷、肺部症状评估量表、8项患者健康问卷、7项广泛性焦虑障碍量表和匹兹堡睡眠质量指数。基于一般估计方程，使用线性回归对结局的纵向变化进行建模。
22名参与者入组，他们在开始使用ETI前后均回答了问卷。中位年龄为35.3岁（四分位距11.1），13名（59.1%）为男性。在对年龄、性别和基线值进行调整的模型中，开始使用ETI后的14周内，疼痛干扰（β = -2.57；95%置信区间 -4.92，-0.23）、鼻窦症状（β = -4.50；95%置信区间 -7.59，-1.41）和睡眠障碍（β = -1.90；95%置信区间 -2.71，-1.09）有显著改善。在研究期间，没有症状领域恶化。
在这项前瞻性研究中，我们发现开始使用ETI的CF患者在三个不同的非肺部症状领域有统计学上的显著改善。虽然这是一项小型的、非对照研究，但它表明使用高效的CFTR调节剂可能会给患者带来除肺部症状之外的益处。
