Division of Pulmonary, Critical Care, and Sleep Medicine, University Hospitals Cleveland Medical Center, 11100 Euclid Avenue, Bolwell Building 6174, Cleveland, OH 44106, USA; Rainbow Babies and Children's Hospital, Cleveland, OH, USA.
Department of Internal Medicine, National Jewish Medical Center, Denver, CO, USA; Department of Pediatrics, National Jewish Medical Center, Denver, CO, USA.
Clin Chest Med. 2022 Dec;43(4):677-695. doi: 10.1016/j.ccm.2022.06.009.
Based on the cystic fibrosis transmembrane conductance regulator (CFTR) genotype, approximately 90% of people with cystic fibrosis (CF) are candidates for highly effective modulator therapy (HEMT). Clinical trials conducted over the last 11 years have shown that these oral therapies substantially restore CFTR function, leading to improvements in lung function, nutritional status, and health-related quality of life. Here, we review safety and efficacy data from phase 3 clinical trials and observational studies which support the use of HEMT in most adults and children with CF. We also discuss opportunities for additional investigation in groups underrepresented or excluded from phase 3 clinical trials, and challenges in the evaluation of the safety and efficacy of HEMT at increasingly earlier stages of CFTR-mediated pathophysiology.
基于囊性纤维化跨膜电导调节因子（CFTR）基因型，约 90%的囊性纤维化（CF）患者是高效调节剂治疗（HEMT）的候选者。过去 11 年进行的临床试验表明，这些口服治疗方法可显著恢复 CFTR 功能，从而改善肺功能、营养状况和健康相关生活质量。在此，我们综述了来自 3 期临床试验和观察性研究的安全性和疗效数据，这些数据支持将 HEMT 用于大多数 CF 成人和儿童。我们还讨论了在 3 期临床试验中代表性不足或被排除的群体中进行进一步研究的机会，以及在 CFTR 介导的病理生理学的更早阶段评估 HEMT 的安全性和疗效所面临的挑战。
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