Université de Paris, Institut Cochin, Inserm U1016, Paris, France; Respiratory Medicine and National Reference Cystic Fibrosis Reference Center, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France; ERN-Lung CF network.
Respiratory Medicine and National Reference Cystic Fibrosis Reference Center, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France; ERN-Lung CF network.
Respir Med Res. 2021 Nov;80:100829. doi: 10.1016/j.resmer.2021.100829. Epub 2021 May 17.
Elexacaftor-tezacaftor-ivacaftor partially restores cystic fibrosis transmembrane conductance regulator function, and has been shown to induce significant clinical improvement in patients with at least one Phe508del allele. Yet little data exist on patient perspectives following elexacaftor-tezacaftor-ivacaftor initiation.
A mixed methods study was conducted using an online 13-item questionnaire (including 9 closed questions and 4 open questions), submitted from July 10 to August 21 2020 to French patients aged 12 years and older with advanced CF who were treated with elexacaftor-tezacaftor-ivacaftor. Their responses were summarized as numbers (%), and free-text items were analysed using a grounded theory approach.
Of 245 patients who started elexacaftor-tezacaftor-ivacaftor in France, 101 (41%) participated. Median [IQR] age was 35 [28-41] years and duration of elexacaftor-tezacaftor-ivacaftor treatment was 4.3 [3.0-5.6] months. Patients generally reported a rapid impact on respiratory symptoms, sleep quality, general well-being and physical self-esteem, and a reduction in overall treatment burden. The majority of patients contrasted treatment burden, symptom severity, depression and a closed future marked by death or transplantation before elexacaftor-tezacaftor-ivacaftor, to renewed and unexpected physical strength, leading to greater self-confidence, autonomy and long-term planning, after treatment initiation. A small number of patients expressed concerns, mainly regarding changes in body representation and/or the fear of becoming dependent on the treatment.
After initiation of elexacaftor-tezacaftor-ivacaftor, CF patients with advanced disease reported rapid and positive physical, psychological and social effects, which translated into improved quality of life and the formulation of new life goals.
依伐卡托与泰比卡托联合艾克卡托治疗能部分恢复囊性纤维化跨膜电导调节因子的功能，并且已被证明可显著改善至少有一个 F508del 等位基因的患者的临床症状。然而，在开始依伐卡托与泰比卡托联合艾克卡托治疗后，患者的观点相关数据很少。
本研究采用混合方法，于 2020 年 7 月 10 日至 8 月 21 日期间，通过在线 13 项问卷（包括 9 个封闭问题和 4 个开放问题），对法国年龄在 12 岁及以上的晚期 CF 患者进行调查，这些患者接受依伐卡托与泰比卡托联合艾克卡托治疗。对患者的回答进行了总结，以数字（%）表示，对自由文本项目采用扎根理论方法进行分析。
在法国开始接受依伐卡托与泰比卡托联合艾克卡托治疗的 245 例患者中，有 101 例（41%）参与了研究。中位[四分位距]年龄为 35[28-41]岁，依伐卡托与泰比卡托联合艾克卡托治疗的时间为 4.3[3.0-5.6]个月。患者普遍报告称，呼吸道症状、睡眠质量、整体健康状况和身体自尊迅速改善，整体治疗负担减轻。大多数患者在接受依伐卡托与泰比卡托联合艾克卡托治疗前后，将治疗负担、症状严重程度、抑郁和死亡或移植标记的封闭未来进行了对比，认为治疗后获得了意想不到的体力，从而增强了自信心、自主性和长期规划。少数患者表达了一些担忧，主要是关于身体形象的变化和/或对依赖治疗的恐惧。
接受依伐卡托与泰比卡托联合艾克卡托治疗后，晚期 CF 患者报告称，他们的身体、心理和社会状况迅速得到改善，并产生了积极的影响，从而提高了生活质量，并制定了新的生活目标。
