Highly effective modulator therapies (HEMTs) have revolutionised the management approach of most patients living with cystic fibrosis (CF) who have access to these therapies. Clinical trials have reported significant improvements across multiorgan systems, with patients surviving longer. However, there are accumulating case reports and observational data describing various adverse events following initiation of HEMTs including drug-to-drug interactions, drug induced liver injury, Stevens-Johnson syndrome, and neurocognitive symptoms including psychosis and depression, which have required discontinuation of therapy. Current clinical trials are assessing efficacy in younger patients with CF, yet long-term studies are also required to better understand the safety profile in the real-world setting across all ages and the impact of HEMT dose alteration or discontinuation.

高效调节剂疗法 (HEMT) 彻底改变了大多数能够接受这些疗法的囊性纤维化 (CF) 患者的治疗方法。临床试验报告了多器官系统的显着改善，患者的生存时间更长。然而，越来越多的病例报告和观察数据描述了开始 HEMT 后的各种不良事件，包括药物间相互作用、药物诱发的肝损伤、史蒂文斯-约翰逊综合征以及包括精神病和抑郁症在内的神经认知症状，这些症状需要停止治疗。目前的临床试验正在评估年轻 CF 患者的疗效，但还需要进行长期研究，以更好地了解所有年龄段的现实环境中的安全性以及 HEMT 剂量改变或停药的影响。