Cystic fibrosis (CF) is a progressive, life-limiting genetic disease caused by a dysfunctional CF transmembrane conductance regulator (CFTR) protein that leads to mucus clearance abnormalities, the development of chronic endobronchial infections and progressive, irreversible lung damage. Historically, treatments for CF have largely been supportive in nature and have focused on symptomatic improvement from the many CF-related disease manifestations, including pulmonary, gastrointestinal and sinus-related morbidities. The use of acute and chronic medications to address these disease-related complications has undoubtedly improved the lives of people with CF (PwCF) and extended life expectancy. The approval of ivacaftor by the US Food and Drug Administration on 31 January 2012 for PwCF 6 years and older with a G551D mutation has heralded in a new era of CF care. For the first time, a CF therapy (termed CFTR modulator) was developed to target the underlying CFTR protein defect itself. Since its approval, several other CFTR modulators have been deployed in the clinical arena; the most recent (and arguably the most effective) of which is elexacaftor/tezacaftor/ivacaftor, a CFTR modulator currently approved for all PwCF 12 years and older with at least once copy of the Phe508del mutation. While the approval of CFTR modulators has revolutionised the care of PwCF and might even improve life expectancy, there remains a number of potential barriers to CFTR modulator adherence, including cost, perceived medication benefit, delays in prior authorisations, decreased access to care and individual factors …

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调节剂的临床益处能否有效地“调节”CF 患者的依从性？

囊性纤维化 (CF) 是一种进行性、限制生命的遗传疾病，由功能失调的 CF 跨膜电导调节剂 (CFTR) 蛋白引起，导致粘液清除异常、慢性支气管内感染的发展和进行性、不可逆的肺损伤。从历史上看，CF 的治疗在本质上主要是支持性的，并且侧重于许多 CF 相关疾病表现的症状改善，包括肺部、胃肠道和鼻窦相关疾病。使用急性和慢性药物治疗这些疾病相关并发症无疑改善了 CF (PwCF) 患者的生活并延长了预期寿命。美国食品和药物管理局于 2012 年 1 月 31 日批准 ivacaftor 用于 6 岁及以上具有 G551D 突变的 PwCF，预示着 CF 护理的新时代。首次开发了一种 CF 疗法（称为 CFTR 调节剂）以针对潜在的 CFTR 蛋白缺陷本身。自批准以来，其他几种 CFTR 调节剂已被部署到临床领域；最新的（可以说是最有效的）是 elexacaftor/tezacaftor/ivacaftor，一种 CFTR 调节剂，目前已批准用于所有 12 岁及以上的 PwCF，至少有一次 Phe508del 突变副本。虽然 CFTR 调节剂的批准彻底改变了 PwC​​F 的护理，甚至可能提高预期寿命，但 CFTR 调节剂的依从性仍然存在许多潜在障碍，包括成本、感知的药物益处、