Sepassi Aryana, Saunders Ila M, Bounthavong Mark, Taplitz Randy A, Logan Cathy, Watanabe Jonathan H
Department of Clinical Pharmacy Practice, University of California, Irvine School of Pharmacy and Pharmaceutical Sciences, Irvine, CA, USA.
Division of Clinical Pharmacy, University of California, San Diego Skaggs School of Pharmacy & Pharmaceutical Sciences, La Jolla, CA, USA.
Pharmacoecon Open. 2023 May;7(3):393-404. doi: 10.1007/s41669-023-00398-y. Epub 2023 Feb 25.
The aim of this study was to assess the cost effectiveness of letermovir prophylaxis with the option for subsequent pre-emptive therapy (PET) for the prevention of cytomegalovirus (CMV) infection compared with a PET-only scenario in adult allogeneic hematopoietic stem cell transplant (allo-HCT) recipients in the United States over a 10-year time horizon.
A publicly available decision tree model was constructed using a commercial third-party payer perspective to simulate an allo-HCT recipient's clinical trajectory in the first-year post-transplant, followed by entry to a Markov model to simulate years 2 through 10. Clinical inputs and utility estimates were derived from published literature. Costs were derived from published literature and US Department of Veterans Affairs Federal Supply Schedule drug pricing. Outcomes assessed included life expectancy, quality-adjusted life-years (QALYs), direct medical costs, and the incremental cost-effectiveness ratio (ICER). One-way and probabilistic sensitivity analyses (PSA) were performed to test the robustness of the findings.
Compared with PET alone, letermovir prophylaxis was projected to increase life-years per person (4.99 vs. 4.70 life-years), and increase QALYs (3.29 vs. 3.08) and costs (US$83.411 vs. US$70,698), yielding an ICER of US$59,356 per QALY gained. One-way sensitivity analyses indicated our model was sensitive to mortality (ICER: $164,771/QALY) and utility (letermovir ICER: $117,447/QALY; PET ICER: $107,290/QALY) in the first-year post-transplant. In 57.1% of the PSA simulations, letermovir was a cost-effective option using a willingness-to-pay threshold of US$100,000 per QALY.
Letermovir prophylaxis is cost effective compared with PET alone with a willingness-to-pay threshold of US$100,000 per QALY gained. Sensitivity analysis results indicate future research is required to understand the impact of mortality and quality of life in the first-year post-transplant to arrive at a conclusive decision on letermovir adoption.
本研究旨在评估在美国成人异基因造血干细胞移植（allo-HCT）受者中，与仅采用抢先治疗（PET）方案相比，来特莫韦预防治疗并结合后续抢先治疗（PET）预防巨细胞病毒（CMV）感染的成本效益，研究时间跨度为10年。
使用商业第三方支付方视角构建一个公开可用的决策树模型，以模拟allo-HCT受者移植后第一年的临床轨迹，随后进入马尔可夫模型模拟第2年至第10年的情况。临床输入数据和效用估计值来自已发表的文献。成本来自已发表的文献和美国退伍军人事务部联邦供应时间表药品定价。评估的结果包括预期寿命（LE）、质量调整生命年（QALY）、直接医疗成本和增量成本效益比（ICER）。进行了单因素和概率敏感性分析（PSA）以检验研究结果的稳健性。
与仅采用PET相比，来特莫韦预防治疗预计可增加人均生命年（4.99对4.70生命年），增加QALY（3.29对3.08）并增加成本（83,411美元对70,698美元），每获得一个QALY的ICER为59,356美元。单因素敏感性分析表明，我们的模型对移植后第一年的死亡率（ICER：164,771美元/QALY）和效用（来特莫韦ICER：117,447美元/QALY；PET ICER：107,290美元/QALY）敏感。在57.1%的PSA模拟中，采用每个QALY支付意愿阈值为100,000美元时，来特莫韦是具有成本效益的选择。
与仅采用PET相比，来特莫韦预防治疗具有成本效益，每个获得的QALY支付意愿阈值为100,000美元。敏感性分析结果表明，需要进一步研究以了解移植后第一年死亡率和生活质量的影响，从而就来特莫韦的应用做出最终决策。
