Ong Thida, Ramsey Bonnie W
Division of Pulmonary and Sleep Medicine, Department of Pediatrics, Seattle Children's Hospital, University of Washington, 4800 Sand Point Way Northeast, M/S OC.7.720, Seattle, WA 98105, USA.
Department of Pediatrics, Seattle Children's Research Institute, Center for Clinical and Translational Research, University of Washington, 2001 8th Avenue, Suite 400, M/S CW8-5B, Seattle, WA 98121, USA.
Pediatr Clin North Am. 2016 Aug;63(4):751-64. doi: 10.1016/j.pcl.2016.04.006.
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are clinically available personalized medicines approved for some individuals with cystic fibrosis (CF) to target the underlying defect of disease. This review summarizes strategies used to develop CFTR modulators as therapies that improve function and availability of CFTR protein. Lessons learned from dissemination of ivacaftor across the CF population responsive to this therapy and future approaches to predict and monitor treatment response of CFTR modulators are discussed. The goal remains to expand patient-centered and personalized therapy to all patients with CF, ultimately improving life expectancy and quality of life for this disease.
囊性纤维化跨膜传导调节因子（CFTR）调节剂是临床上可用的个性化药物，已被批准用于一些囊性纤维化（CF）患者，以针对疾病的潜在缺陷。本综述总结了开发CFTR调节剂作为改善CFTR蛋白功能和可用性的疗法所采用的策略。讨论了从依伐卡托在对此疗法有反应的CF患者群体中的传播中吸取的经验教训，以及预测和监测CFTR调节剂治疗反应的未来方法。目标仍然是将以患者为中心的个性化治疗扩展到所有CF患者，最终提高这种疾病患者的预期寿命和生活质量。
