Supplementary Material for: “An amazing transformation!” The lived experiences of elexacaftor/tezacaftor/ivacaftor in adults with CF

Introduction: Cystic fibrosis (CF) is associated with a high respiratory symptom and treatment burden. Elexacaftor/tezacaftor/ivacaftor (ETI) provides substantial improvements in physiological outcomes such as respiratory function and sweat chloride. This study sought to comprehensively examine an area of limited research in this field: the lived experiences of adults with CF after ETI initiation.
Methods: Adults with CF completed semi-structured interviews at least six weeks following the initiation of ETI. Participants shared their experiences regarding respiratory-related symptoms, airway clearance routines, and their capacity to participate in physical activity. Interviews were audio-recorded, transcribed verbatim and analysed using the Framework Method.
Results: Seventeen participants completed interviews (FEV1 range before ETI initiation 31 to 87 %pred). All participants reported improvements in respiratory-related symptoms including cough, sputum expectoration, dyspnoea, and chest tightness. As a result, there was a reduction in participants airway clearance treatment burden and improvements in their capacity to participate in physical activity, health-related quality of life (HRQoL) and outlook for the future. The overall theme highlighted in this study was that ETI had “completely changed everything” and they can now do “everything I wasn’t able to do before” due to the reductions in symptom and treatment burden.
Conclusion: Participants consistently described their experiences with ETI in positive terms. The results of this study confirm that ETI is in fact perceived to be a “miracle drug” and has had a transformative effect on the everyday lives of adults with CF through improved HRQoL and overall wellbeing, which led to positive views regarding their future with CF and ongoing medical care.

引言：囊性纤维化（cystic fibrosis, CF）与较高的呼吸道症状负担及治疗负担密切相关。依来卡托/泰泽卡托/伊伐卡托（Elexacaftor/tezacaftor/ivacaftor，ETI）可显著改善肺功能、汗液氯离子水平等生理结局指标。本研究旨在全面探索该领域尚未得到充分研究的方向：即囊性纤维化成人患者启动ETI治疗后的真实生活体验。
方法：囊性纤维化成人患者在启动ETI治疗至少六周后接受半结构化访谈。参与者分享了其在呼吸道相关症状、气道廓清治疗流程以及参与体力活动能力方面的体验。访谈内容采用音频录制、逐字转录，并借助框架分析法（Framework Method）进行分析。
结果：共17名参与者完成访谈（ETI启动前的一秒用力呼气容积（FEV1）占预计值百分比范围为31%~87%）。所有参与者均报告呼吸道相关症状得到改善，包括咳嗽、咳痰、呼吸困难与胸闷。受此影响，参与者的气道廓清治疗负担有所降低，参与体力活动的能力、健康相关生活质量（health-related quality of life, HRQoL）以及对未来的展望均得到改善。本研究凸显的核心主题为：ETI已"彻底改变了一切"，患者因症状与治疗负担的减轻，如今能够完成"此前无法做到的所有事情"。
结论：参与者均以积极的口吻描述了其使用ETI的体验。本研究结果证实，ETI实际上被参与者视为"神药"，其通过改善HRQoL与整体健康状况，对囊性纤维化成人患者的日常生活产生了颠覆性影响，进而让患者对自身罹患CF后的未来以及后续医疗护理持有积极态度。