De la Hoz Diana, Villamil Osorio Milena, Restrepo-Gualteros Sonia M
Universidad Nacional de Colombia, Departamento de Pediatría, Bogotá DC, Colombia.
Fundación Hospital Pediátrico de la Misericordia, Bogotá DC, Colombia.
Arch Argent Pediatr. 2019 Apr 1;117(2):e131-e136. doi: 10.5546/aap.2019.eng.e131.
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are the present and future of drug management for patients with cystic fibrosis. The objective of this article is to review this therapeutic option. Scientific articles were reviewed by searching the MedLine database, which is available through the Cystic Fibrosis Foundation's official website, from 2009 to 2018, in English. Twelve articles about the current status of research in CFTR modulators were selected without restrictions regarding the type of study. To date, the United States Food and Drug Administration has approved three modulators: ivacaftor, lumacaftor + ivacaftor, and tezacaftor + ivacaftor, while other 11 drugs are being studied in different investigation phases. CFTR modulator therapy is a developing reality aimed at the highest goal of personalized medicine and promises to improve the quality of life of cystic fibrosis patients.
囊性纤维化跨膜传导调节因子（CFTR）调节剂是目前及未来用于囊性纤维化患者药物治疗的关键。本文旨在对这一治疗选择进行综述。通过检索可从囊性纤维化基金会官方网站获取的MedLine数据库，筛选了2009年至2018年期间的英文科学文献。选取了12篇关于CFTR调节剂研究现状的文章，研究类型不限。迄今为止，美国食品药品监督管理局已批准了三种调节剂：依伐卡托、鲁马卡托+依伐卡托和泰扎卡托+依伐卡托，另有11种药物正处于不同的研究阶段。CFTR调节剂疗法是朝着精准医疗这一最高目标发展的现实手段，有望改善囊性纤维化患者的生活质量。
