首页> 美国卫生研究院文献>Therapeutic Advances in Respiratory Disease >Modeling long-term health outcomes of patients with cystic fibrosis homozygous for F508del-CFTR treated with lumacaftor/ivacaftor
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Modeling long-term health outcomes of patients with cystic fibrosis homozygous for F508del-CFTR treated with lumacaftor/ivacaftor

机译:为lumacaftor / ivacaftor治疗的F508del-CFTR纯合的囊性纤维化患者的长期健康结局建模

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摘要

Background:Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. However, long-term survival benefits of lumacaftor/ivacaftor (LUM/IVA) cannot yet be quantified. Simulation models can provide predictions about long-term health outcomes. In this study, we aimed to project long-term health outcomes of LUM/IVA plus standard care (SC) in patients with CF homozygous for F508del-CFTR.
机译:背景:Lumacaftor / ivacaftor联合疗法对F508del-CF跨膜电导调节剂(CFTR)突变的纯合性囊性纤维化(CF)患者有效且通常安全。但是,尚不能量化lumacaftor / ivacaftor(LUM / IVA)的长期生存益处。模拟模型可以提供有关长期健康结果的预测。在这项研究中,我们旨在预测F508del-CFTR纯合CF患者的LUM / IVA加标准治疗(SC)的长期健康结果。

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