西班牙急性髓细胞白血病或骨髓增生异常综合征患者中使用泊沙康唑片预防侵袭性真菌感染的成本效益分析。

Cost-Effectiveness of Posaconazole Tablets for Invasive Fungal Infections Prevention in Acute Myelogenous Leukemia or Myelodysplastic Syndrome Patients in Spain.

机构信息

La Princesa Hospital, Madrid, Spain.

Outcomes10, Castellon, Spain.

出版信息

Adv Ther. 2017 Sep;34(9):2104-2119. doi: 10.1007/s12325-017-0600-1. Epub 2017 Aug 14.

INTRODUCTION

Posaconazole is superior to fluconazole (FLU) and itraconazole (ITRA) in the prevention of invasive fungal diseases (IFDs) in neutropenic patients with acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS). A new tablet formulation of posaconazole with improved pharmacokinetic and pharmacodynamic properties compared to posaconazole oral solution has recently been approved. The objective of this study is to estimate the cost-effectiveness of the newly developed posaconazole tablets versus FLU oral suspension or ITRA oral solution for preventing IFDs in high-risk neutropenic patients with AML or MDS and from the perspective of the Spanish National Health System (NHS).

METHODS

A previously validated economic model was used. The probabilities of experiencing an IFD, an IFD-related death or death from other causes over 100 days were based on clinical trial data and input into a decision tree. Surviving patients were entered into a Markov model to calculate total costs, number of IFDs and number of life-years gained per patient over a lifetime horizon in each disease and treatment group. Two health states, alive and dead, were considered. Health effects were discounted using a rate of 3%. Univariate and probabilistic sensitivity analyses were conducted.

RESULTS

During the first 100 days, posaconazole tablets were associated with a lower risk of IFDs (0.046 vs. 0.111), longer life expectancy (2.92 vs. 2.69 years) and lower total costs (€5906.06 vs. €7847.20 per patient) over the patients' lifetimes compared to FLU or ITRA treatments. Thus, posaconazole tablets were more effective and less costly than FLU or ITRA. Probabilistic sensitivity analysis indicated that there was a 79.9% probability of posaconazole tablets being cost-saving compared to FLU or ITRA.

CONCLUSION

From the Spanish NHS perspective, posaconazole tablets are cost-effective compared to FLU or ITRA in AML or MSD patients with chemotherapy-induced neutropenia and at high risk for IFDs.

FUNDING

MSD Sharp & Dohme.

简介

泊沙康唑优于氟康唑（FLU）和伊曲康唑（ITRA），可预防中性粒细胞减少的急性髓系白血病（AML）和骨髓增生异常综合征（MDS）患者侵袭性真菌感染（IFD）。最近批准了一种泊沙康唑新片剂制剂，与泊沙康唑口服溶液相比，具有改善的药代动力学和药效学特性。本研究旨在从西班牙国家卫生系统（NHS）的角度评估新开发的泊沙康唑片剂与 FLU 口服混悬剂或 ITRA 口服溶液预防 AML 或 MDS 伴高危中性粒细胞减少的患者 IFD 的成本效益。

方法

使用了先前验证的经济模型。IFD、IFD 相关死亡或其他原因死亡的概率在 100 天内基于临床试验数据并输入决策树。存活患者进入马尔可夫模型，以计算每个疾病和治疗组的每位患者终生的总成本、IFD 数量和获得的生命年数。考虑了两种健康状况，存活和死亡。使用 3%的贴现率对健康影响进行贴现。进行了单变量和概率敏感性分析。

结果

在最初的 100 天内，与 FLU 或 ITRA 治疗相比，泊沙康唑片剂与较低的 IFD 风险（0.046 比 0.111）、更长的预期寿命（2.92 比 2.69 年）和更低的总费用（每位患者 5906.06 欧元比 7847.20 欧元）相关。因此，与 FLU 或 ITRA 相比，泊沙康唑片剂更有效且成本更低。概率敏感性分析表明，与 FLU 或 ITRA 相比，泊沙康唑片剂具有 79.9%的成本节约可能性。