Cost effectiveness of reduced intensity conditioning and transplantation of unrelated umbilical cord blood versus HLA haploidentical related bone marrow for adults with hematologic malignancies.
Journal of Clinical Oncology(2022)
摘要
6591 Background: BMT CTN 1101 was a Phase III randomized controlled trial evaluating the comparative effectiveness of unrelated umbilical cord blood (UCB) versus HLA-haploidentical related donor bone marrow (haplo-BM) cell sources for hematopoietic cell transplantation (HCT) in patients with high-risk hematologic malignancies (leukemias, lymphomas). We report results of an economic evaluation conducted as part of the clinical trial. Methods: 368 patients (90% of planned accrual) enrolled from 33 centers in the U.S. were randomly assigned to unrelated UCB (n=186) or haplo-BM (n=182) transplant. Healthcare utilization and costs were estimated using propensity-score matched cohorts of BMT patients in the OptumLabs Data Warehouse for trial participants <65 years, and Medicare claims for trial participants ≥65 years. Cost-effectiveness was calculated from payer perspectives (commercial, Medicare) over a 20-year time horizon from time of transplant. Weibull models (best fit based on AIC/BIC) were used to extrapolate survival from 5-year trial follow-up data. Trial participant surveys (EQ-5D) were used to derive health state utilities for estimating Quality-Adjusted Life Years (QALYs). One-way and probabilistic sensitivity analyses were conducted to assess uncertainty in results. Outcomes were discounted at 3% annually. Results: At 5-year follow-up, overall survival was 42% for haplo-BM versus 36% for UCB (P=.06). Over a 20-year time horizon, haplo-BM is expected to be more effective and more costly for <65 year-olds and in ≥65 year-olds it is expected to be more effective and less costly. In one-way uncertainty analyses, for persons <65, the cost/QALY result was most sensitive to life years and health state utilities. For persons ≥65, life years were more influential than costs and health state utilities. Using probabilistic sensitivity analysis, for persons <65 there was a 43% chance that haplo-BM was cost-effective using a willingness to pay threshold of $150k/QALY and 52% at a $200K/QALY threshold. Conclusions: Results from a large national clinical trial indicate that compared to UCB, haplo-BM was moderately cost-effective for patients aged <65 years, and less costly and more effective for persons ≥65 years. Haplo-BM is a fair value choice for commercially insured patients with high-risk leukemia and lymphoma who require HCT. For Medicare enrollees, haplo-BM is a preferred choice when considering costs and outcomes. [Table: see text]
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关键词
hematologic malignancies,unrelated umbilical cord blood,bone marrow,cost effectiveness,transplantation
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