对于IDH2突变型难治性/复发性急性髓细胞性白血病老年患者,依那西尼与常规治疗的成本效益对比。

IF 2.2 4区 医学 Q3 HEMATOLOGY
Abdulrahman Alhajahjeh, Kishan K Patel, Rory M Shallis, Nikolai A Podoltsev, Tariq Kewan, Jessica M Stempel, Lourdes Mendez, Scott F Huntington, Maximilian Stahl, George Goshua, Jan Philipp Bewersdorf, Amer M Zeidan
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引用次数: 0

摘要

在随机III期IDHENTIFY试验中,与常规治疗方案(CCR)相比,IDH2抑制剂依那西尼(ENA)改善了复发/难治性(R/R)、IDH2突变急性髓细胞白血病患者的无事件生存期,但未改善总生存期。我们构建了一个分区生存模型,以评估依那西尼治疗复发/难治性和IDH2突变型老年急性髓细胞性白血病患者的成本效益。在基础方案中,与CCR相比,ENA的增量有效性为0.234质量调整生命年(QALYs),增量成本为12.68万美元,增量成本效益比为54.03万美元/QALY(95% CI:19.78万美元-477.7万美元/QALY)。在概率敏感性分析中,99.8% 的模拟结果均支持 CCR。在 150,000 美元/QALY 的支付意愿阈值下,ENA 的成本需要降低 72%,才具有成本效益。我们的研究结果表明,在目前的定价条件下,ENA不太可能成为治疗老年IDH2突变R/R急性髓细胞白血病患者的一种具有成本效益的疗法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Cost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML.

In the randomized phase III IDHENTIFY trial, the IDH2 inhibitor enasidenib (ENA) showed improvement in event-free but not overall survival compared with conventional care regimens (CCR) among patients with relapsed/refractory (R/R), IDH2-mutant AML. We constructed a partitioned survival model to evaluate the cost-effectiveness of enasidenib for the treatment of older patients with R/R, and IDH2-mutant AML. In the base-case scenario, ENA exhibited an incremental effectiveness of 0.234quality-adjusted life-years (QALYs) compared to CCR, and an incremental cost of $126,800, leading to an incremental cost-effectiveness ratio of $540,300/QALY(95% CI: $197,800-$4,777,000/QALY). In probabilistic sensitivity analysis, CCR was favored in 99.8% of simulations. The cost of ENA would need to be decreased by 72% to be cost-effective at a willingness-to-pay threshold of $150,000/QALY. Our findings suggest that ENA is unlikely to be a cost-effective treatment for older patients with IDH2-mutant R/R AML under current pricing.

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来源期刊
Leukemia & Lymphoma
Leukemia & Lymphoma 医学-血液学
CiteScore
4.10
自引率
3.80%
发文量
384
审稿时长
1.8 months
期刊介绍: Leukemia & Lymphoma in its fourth decade continues to provide an international forum for publication of high quality clinical, translational, and basic science research, and original observations relating to all aspects of hematological malignancies. The scope ranges from clinical and clinico-pathological investigations to fundamental research in disease biology, mechanisms of action of novel agents, development of combination chemotherapy, pharmacology and pharmacogenomics as well as ethics and epidemiology. Submissions of unique clinical observations or confirmatory studies are considered and published as Letters to the Editor
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