https://orcid.org/0000-0001-5264-8990
Key Points
Upfront HSCT for intermediate-risk AML is always cost-effective in the United States and the United Kingdom, compared to delayed HSCT
The respective incremental net monetary benefits are $497,100 and £235,600, respectively, for the United States and the United Kingdom
The ETAL-1 trial demonstrated that upfront allogeneic hematopoietic stem cell transplant (HSCT) improved disease-free survival (DFS) but not overall survival (OS) when compared to consolidation chemotherapy (CCT) followed by "delayed" HSCT on relapse in patients with intermediate-risk acute myeloid leukemia (AML). However, the health-economic implications of upfront HSCT compared to delayed HSCT are unknown. We developed a partitioned survival analysis model using derived survival data, probabilities of salvage treatments, utilities, and costs from the ETAL-1 trial and published literature. The primary outcome was the incremental net monetary benefit (INMB) from the perspective of the United States (US) and the United Kingdom (UK) healthcare systems, at all accepted willingness-to-pay (WTP) thresholds: $50,000-$150,000 per quality-adjusted life year (QALY) and £20,000-30,000/QALY, respectively. The respective INMBs favored upfront HSCT and were $497,100 (95% CI: $259,800 - $719,600) and £235,600 (95% CI: £166,800 - £298,500) at WTP thresholds of $150,000/QALY and £30,000/QALY. Across deterministic sensitivity analyses, no model input changed the conclusion that upfront HSCT is the cost-effective strategy in either jurisdiction. Probabilistic sensitivity analysis showed that upfront HSCT was cost-effective in 100% of iterations and was less costly and more effective (i.e., 'dominant') in over 90% of iterations in both healthcare systems. In conclusion, we conducted a partitioned survival analysis based on the ETAL-1 trial and showed that proceeding to HSCT in first remission is the cost-effective strategy in the care of intermediate-risk AML patients in both the US and the UK, as compared to delayed HSCT.
