囊性纤维化患者Tezacaftor-Ivacaftor Phe508del纯合子。

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Taylor-Cousar杰,Munck McKone EF, van der Ent CK, Moeller, Simard C,王LT, Ingenito EP,麦基C,陆Y, Lekstrom-Himes J, Elborn JS

囊性纤维化患者Tezacaftor-Ivacaftor Phe508del纯合子。

郑传经地中海J。2017年11月23日,377 (21):2013 - 2023。doi: 10.1056 / NEJMoa1709846。Epub 2017年11月3。

PubMed ID
29099344 (在PubMed
]
文摘

背景:联合治疗与囊性纤维化跨膜电导调节(雌性生殖道)调节器tezacaftor (vx - 661)和ivacaftor (vx - 770)设计目标疾病的根本原因在囊性纤维化患者。方法:在这个阶段3、随机、双盲、多中心、安慰剂对照、平行对照试验中,我们与tezacaftor评估联合治疗,ivacaftor 12岁以上的病人谁有囊性纤维化和纯合子的雌性生殖道Phe508del突变。病人被随机分配在1:1的比例获得100毫克的tezacaftor一旦每日150毫克的ivacaftor每日两次或匹配的安慰剂24周。主要终点是绝对的百分比变化预计在1秒用力呼气量(FEV1)通过24周(百分比计算);相对变化量的百分比预测FEV1通过一周的24(百分比计算)是一个重要的次要终点。结果:510个病人的随机化,509收到tezacaftor-ivacaftor或安慰剂,475年完成了24周的试验方案。平均FEV1在基线的60.0%的预测价值。上的效果绝对和相对比例的变化预测残赞成tezacaftor-ivacaftor安慰剂分别4.0和6.8%,分别为比较(P < 0.001)。肺恶化的速度降低了35% tezacaftor-ivacaftor组比安慰剂组(P = 0.005)。不良事件的发生率在两组相似。 Most adverse events were of mild severity (in 41.8% of patients overall) or moderate severity (in 40.9% overall), and serious adverse events were less frequent with tezacaftor-ivacaftor (12.4%) than with placebo (18.2%). A total of 2.9% of patients discontinued the assigned regimen owing to adverse events. Fewer patients in the tezacaftor-ivacaftor group than in the placebo group had respiratory adverse events, none of which led to discontinuation. CONCLUSIONS: The combination of tezacaftor and ivacaftor was efficacious and safe in patients 12 years of age or older who had cystic fibrosis and were homozygous for the CFTR Phe508del mutation. (Funded by Vertex Pharmaceuticals; EVOLVE ClinicalTrials.gov number, NCT02347657 .).

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药物
药物靶点
药物 目标 类 生物 药理作用 行动
Tezacaftor 囊性纤维化跨膜电导调节 蛋白质 人类
是的
积极的变构调制器
 细节