依伐卡托：一种用于囊性纤维化的新型基因治疗方法。
Ivacaftor: a novel gene-based therapeutic approach for cystic fibrosis.
作者信息
Condren Michelle E, Bradshaw Marquita D
机构信息
Department of Pharmacy Clinical and Administrative Sciences-Tulsa, University of Oklahoma College of Pharmacy, Tulsa, Oklahoma ; Department of Pediatrics, University of Oklahoma School of Community Medicine, Tulsa, Oklahoma.
出版信息
J Pediatr Pharmacol Ther. 2013 Jan;18(1):8-13. doi: 10.5863/1551-6776-18.1.8.
Abstract
Ivacaftor is a new therapeutic agent that acts at the cystic fibrosis transmembrane conductance regulator (CFTR) channel to alter activity. It is approved for use in patients 6 years and older with cystic fibrosis who have at least 1 G551D mutation in the CFTR gene. It is unlike any other current pharmacologic agent for cystic fibrosis in that it specifically targets the gene defect associated with cystic fibrosis as opposed to treating resulting symptomology. Mucoactive agents, antibiotics, inhaled beta agonists, and other anti-inflammatory agents are currently the mainstay of cystic fibrosis treatment but can be associated with several side effects in addition to cumbersome frequency of administration. Ivacaftor's oral dosing regimen offers a more convenient treatment option. However, it is associated with significant drug-drug interactions.
摘要
依伐卡托是一种新型治疗药物，作用于囊性纤维化跨膜传导调节因子（CFTR）通道以改变其活性。它被批准用于6岁及以上患有囊性纤维化且CFTR基因至少有1个G551D突变的患者。它与目前用于囊性纤维化的任何其他药物不同，因为它专门针对与囊性纤维化相关的基因缺陷，而不是治疗由此产生的症状。黏液活性药物、抗生素、吸入性β受体激动剂和其他抗炎药物目前是囊性纤维化治疗的主要手段，但除了给药频率繁琐外，还可能伴有多种副作用。依伐卡托的口服给药方案提供了一种更方便的治疗选择。然而，它会引起显著的药物相互作用。
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