Compassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience
Compassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience
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DOI：
10.1016/j.arbres.2018.05.004
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发表时间：
2018-12-01
影响因子：
8
通讯作者：
Javier Gomez-de-Terreros-Caro, Francisco
中科院分区：
文献类型：
--
作者：
Diab-Caceres, Layla;Maria Giron-Moreno, Rosa;Javier Gomez-de-Terreros-Caro, Francisco
背景：最常见的导致囊性纤维化的突变是F508(F508del)，它存在于28%的西班牙囊性纤维化患者中。虽然基于对接受Lumacaftor/iVacaftor治疗的纯合子F508del的CF患者的实际研究的文献有限，但它表明需要更好的策略来预防相关不良事件(AEs)以及新药的开发。方法：我们在西班牙进行了一项多中心、回顾性、观察性研究，以描述Lumacaftor/iVacaftor治疗在现实生活中的效果。研究对象为20名6岁及以上的CF患者，均为ppFEV1
Background: The most common cystic fibrosis (CF)-causing mutation is deltaF508 (F508del), which is present in 28% of CF Spanish patients. While the literature based on real-life studies on CF patients homozygous F508del treated with lumacaftor/ivacaftor is limited, it demonstrates the need for better strategies to prevent related adverse events (AEs) as well as the development of newer drugs.Methods: We conducted a multicenter, retrospective, observational study to describe the effects of lumacaftor/ivacaftor treatment in real-life in Spain. 20 CF patients were included, all aged 6 and upwards and presented with ppFEV1