J Pediatr Health Care. 2019 May-Jun;33(3):356-364. doi: 10.1016/j.pedhc.2018.08.013.
The literature surrounding the use of cystic fibrosis transmembrane conductance regulator-targeted pharmacotherapies in pediatric patients continues to evolve. These therapies represent a departure from symptom management and infection prevention, which have been the mainstay of cystic fibrosis management in pediatrics, to targeting the genetic defect present within these patients. This article reviews the clinical studies evaluating the safety and efficacy of ivacaftor, ivacaftor/lumacaftor, and ivacaftor/tezacaftor. These medications were initially studied in adults and adolescents but have begun to be studied in younger populations. Further investigation into the use of these drugs with different CFTR mutations and in younger age groups will continue to expand the number of patients who can benefit from these therapies.
有关囊性纤维化跨膜电导调节因子靶向治疗在儿科患者中的应用的文献不断发展。这些治疗方法不同于症状管理和感染预防，后者一直是儿科囊性纤维化管理的主要手段，而是针对这些患者中存在的遗传缺陷。本文综述了评估伊伐卡托、伊伐卡托/卢美卡托和伊伐卡托/特扎卡托安全性和有效性的临床研究。这些药物最初在成人和青少年中进行了研究，但已开始在更年轻的人群中进行研究。进一步研究这些药物在不同 CFTR 突变和更年轻年龄组中的应用将继续扩大受益于这些治疗方法的患者数量。
