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首页> 外文期刊>Value in health: the journal of the International Society for Pharmacoeconomics and Outcomes Research >Forecasting the Long-Term Clinical and Economic Outcomes of Lumacaftor/Ivacaftor in Cystic Fibrosis Patients with Homozygous phe508del Mutation
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Forecasting the Long-Term Clinical and Economic Outcomes of Lumacaftor/Ivacaftor in Cystic Fibrosis Patients with Homozygous phe508del Mutation

机译：纯合PHE508del突变患者岩手/ IVAcafetor的长期临床和经济效应

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Abstract Objectives To forecast lifetime outcomes and cost of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis (CF) with homozygous phe508del mutation from the US payer perspective. Methods A lifetime Markov model was developed from a US payer perspective. The model included five health states: 1) mild lung disease (percent predicted forced expiratory volume in 1 second [FEV 1 ] >70%), 2) moderate lung disease (40% ≤ FEV 1 ≤ 70%), 3) severe lung disease (FEV 1 Results Lumacaftor/ivacaftor was associated with additional 2.91 life-years (95% credible interval 2.55–3.56) and additional 2.42 quality-adjusted life-years (QALYs) (95% credible interval 2.10–2.98). Lumacaftor/ivacaftor was associated with improvements in survival and QALYs equivalent to 27.6% and 20.7%, respectively, for the survival and QALY gaps between CF usual care and their non-CF peers. The incremental lifetime cost was $2,632,249. Conclusions Lumacaftor/ivacaftor increased life-years and QALYs in CF patients with the homozygous phe508del mutation and moved morbidity and mortality closer to that of their non-CF peers but it came with higher cost.
机译：摘要目的预测患有杂志纤维化（CF）的Lumacafetor / Ivacafactor组合治疗的终身结果和成本与美国付款人的纯合PHE508del突变。方法从美国付款人的角度开发了一生马尔可夫模型。该模型包括五个健康状态：1）轻度肺病（百分比预测强制呼气量1秒[FEV 1]> 70％），2）中度肺病（40％≤FEV1≤70％），3）严重肺疾病（FEV 1结果Lumacaftor / Ivacafacter与额外的2.91寿命相关（95％可靠的间隔2.55-3.56）和额外的2.42质量调整的寿命 - 年（Qalys）（95％可靠的间隔2.10-2.98）。Lumacafactor / Ivacaftor与QALYS的改善有关，分别为27.6％和20.7％，分别用于CF通常护理及其非CF对等体之间的生存和QALY差距。增量终身费用为2,632,249美元。结论Lumacafactor / Ivacafeor uploce和qalys在CF患者中纯合PHE508DEL突变，移动发病率和死亡率更接近他们的非CF同行，但它具有更高的成本。

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