Cost-Effectiveness of Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation for Older Patients With High-Risk Myelodysplastic Syndrome: Analysis of BMT CTN 1102

PURPOSE BMT CTN 1102 was a phase III trial comparing reduced-intensity allogeneic hematopoietic cell transplantation (RIC alloHCT) to standard of care for persons with intermediate- or high-risk myelodysplastic syndrome (MDS). We report results of a cost-effectiveness analysis conducted alongside the clinical trial. METHODS Three hundred eighty-four patients received HCT (n = 260) or standard of care (n = 124) according to availability of a human leukocyte antigen–matched donor. Cost-effectiveness was calculated from US commercial and Medicare perspectives over a 20-year time horizon. Health care utilization and costs were estimated using propensity score–matched cohorts of HCT recipients in the OptumLabs Data Warehouse (age 50-64 years) and Medicare (age 65 years and older). EuroQol 5 Dimension (EQ-5D) surveys of trial participants were used to derive health state utilities. RESULTS Extrapolated 20-year overall survival for those age 50-64 years was 29% for HCT (n = 105) versus 13% for usual care (n = 44) and 31% for HCT (n = 155) versus 12% for non-HCT (n = 80) for those age 65 years and older. HCT was more effective (+2.36 quality-adjusted life-years [QALYs] for age 50-64 years and +2.92 QALYs for age 65 years and older) and more costly (+$452,242 in US dollars (USD) for age 50-64 years and +$233,214 USD for age 65 years and older) than usual care, with incremental cost-effectiveness ratios of $191,487 (USD)/QALY and $79,834 (USD)/QALY, respectively. For persons age 50-64 years, there was a 29% chance that HCT was cost-effective using a willingness-to-pay (WTP) threshold of $150K (USD)/QALY and 51% at a $200K (USD)/QALY. For persons age 65 years and older, the probability was 100% at a WTP >$150K (USD)/QALY. CONCLUSION Among patients age 65 years and older with high-risk MDS, RIC HCT is a high-value strategy. For those age 50-64 years, HCT is a lower-value strategy but has similar cost-effectiveness to other therapies commonly used in oncology. For patients with high-risk myelodysplastic syndrome, bone marrow transplant is a cost-effective therapy option.

目的 BMT CTN 1102是一项Ⅲ期试验，比较了减低强度异基因造血细胞移植（RIC alloHCT）与中危或高危骨髓增生异常综合征（MDS）患者的标准治疗。我们报告了与临床试验同时进行的成本 - 效果分析结果。 方法 384例患者根据是否有人类白细胞抗原匹配的供者，接受了造血细胞移植（n = 260）或标准治疗（n = 124）。从美国商业保险和医疗保险的角度，在20年的时间跨度内计算成本 - 效果。利用OptumLabs数据仓库（年龄50 - 64岁）和医疗保险（年龄65岁及以上）中倾向评分匹配的造血细胞移植受者队列估计医疗保健利用情况和成本。使用试验参与者的欧洲五维健康量表（EQ - 5D）调查来推导健康状态效用。 结果 年龄50 - 64岁人群外推的20年总生存率，造血细胞移植组（n = 105）为29%，常规治疗组（n = 44）为13%；年龄65岁及以上人群，造血细胞移植组（n = 155）为31%，非造血细胞移植组（n = 80）为12%。与常规治疗相比，造血细胞移植更有效（年龄50 - 64岁为+2.36质量调整生命年[QALYs]，年龄65岁及以上为+2.92 QALYs）且成本更高（年龄50 - 64岁增加452,242美元，年龄65岁及以上增加233,214美元），增量成本 - 效果比分别为191,487美元/QALY和79,834美元/QALY。对于年龄50 - 64岁的人群，当支付意愿阈值为150,000美元/QALY时，造血细胞移植具有成本 - 效果的可能性为29%，当阈值为200,000美元/QALY时为51%。对于年龄65岁及以上的人群，当支付意愿＞150,000美元/QALY时，概率为100%。 结论 在年龄65岁及以上的高危MDS患者中，RIC造血细胞移植是一种高价值的策略。对于年龄50 - 64岁的人群，造血细胞移植是一种价值较低的策略，但与肿瘤学中常用的其他疗法具有相似的成本 - 效果。对于高危骨髓增生异常综合征患者，骨髓移植是一种具有成本 - 效果的治疗选择。