依伐卡托特治疗非 G551D 门控突变的囊性纤维化患者的疗效和安全性。
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation.
机构信息
University Hospital Gasthuisberg, Leuven, Belgium.
Assistance publique-Hôpitaux de Paris, Hôpital Robert Debré, Paris, France.
出版信息
J Cyst Fibros. 2014 Dec;13(6):674-80. doi: 10.1016/j.jcf.2014.09.005. Epub 2014 Sep 26.
BACKGROUND
Ivacaftor is used to treat patients with CF and a G551D gating mutation; the KONNECTION study assessed the efficacy and safety of ivacaftor in patients with CF and a non-G551D gating mutation.
METHODS
Patients with CF ≥6-years- old with non-G551D gating mutations received ivacaftor 150mg q12h or placebo for 8weeks in this 2-part, double-blind crossover study (Part 1) with a 16-week open-label extension (Part 2). The primary efficacy outcome was absolute change in FEV1 through 8 and 24weeks of ivacaftor treatment; secondary outcomes were changes in BMI, sweat chloride, and CFQ-R and safety through 8 and 24weeks of treatment.
RESULTS
Eight weeks of ivacaftor resulted in significant improvements in percent predicted FEV1, BMI, sweat chloride, and CFQ-R scores that were maintained through 24weeks. Ivacaftor was generally well tolerated.
CONCLUSIONS
Ivacaftor was efficacious in a group of patients with CF who had selected non-G551D gating mutations.
背景
依伐卡托用于治疗 CF 伴有 G551D 门控突变的患者；KONNECTION 研究评估了依伐卡托治疗 CF 伴有非 G551D 门控突变患者的疗效和安全性。
方法
这项两部分、双盲交叉研究（第 1 部分）纳入年龄≥6 岁、伴有非 G551D 门控突变的 CF 患者，患者在第 1 部分接受依伐卡托 150mg，每日 2 次或安慰剂治疗 8 周，随后进入为期 16 周的开放标签扩展期（第 2 部分）。主要疗效结局为依伐卡托治疗 8 周和 24 周时的 FEV1 绝对值变化；次要结局为治疗 8 周和 24 周时 BMI、汗液氯和 CFQ-R 的变化以及安全性。
结果
依伐卡托治疗 8 周可显著改善预测值 FEV1、BMI、汗液氯和 CFQ-R 评分，且这些改善在 24 周时得到维持。依伐卡托总体耐受良好。
结论
依伐卡托对一组伴有特定非 G551D 门控突变的 CF 患者有效。
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