Upfront HSCT More Cost-Effective for Patients With AML in Remission

Upfront hematopoietic stem cell transplantation (HSCT) is the cost-effective option for patients with intermediate-risk acute myeloid leukemia (AML) who are in remission.

Research found that proceeding with HSCT is preferable to delaying transplantation until after consolidation chemotherapy from a system-level economic perspective. The study was published in Blood Advances.¹

The new study builds on the results from the ETAL-1 trial, which was published in 2023.² That phase 3 trial included 143 patients with intermediate-risk AML who had achieved complete remission or complete remission with incomplete blood cell count recovery after conventional induction therapy. The patients, all of whom were 60 years old or younger, were assigned in a randomized fashion to receive either allogeneic HSCT or high-dose cytarabine for consolidation. In the latter group, HSCT was delayed unless patients experienced relapse. The results showed that, at 2 years, overall survival was marginally higher in the consolidation chemotherapy group compared to the primary HSCT group (84% versus 74%). However, disease-free survival at 2 years was higher among patients who received primary HSCT (69% versus 40%).

Yet, while ETAL-1 helped shed light on the impact of the 2 treatment approaches on patient outcomes, it did not delve into the financial implications of the different approaches.

In the new report Jan Bewersdorf, MD, of Yale University, and colleagues used data from the earlier trial to develop a partitioned survival analysis model that compared the cost-effectiveness of upfront HSCT versus “delayed” HSCT in intermediate-risk patients with AML in their first remission.

“By incorporating AML-specific clinical outcomes, costs, and quality of life data, this research seeks to provide guidance for optimizing treatment decisions in intermediate-risk AML in both the United States (US) and the United Kingdom (UK),” Bewersdorf and colleagues wrote.

The study’s primary outcome was the incremental net monetary benefit (INMB) for the US and UK, using willingness-to-pay thresholds of $50,000 to $150,000 per quality-adjusted life year (QALY) and £20,000 to 30,000 per QALY.

The investigators’ findings favored upfront HSCT. They found the INMB for upfront HSCT was $497,100 (95% CI, $259,800-$719,600) and £235,600 (95% CI, £166,800-£298,500) using the higher end of the willingness-to-pay thresholds. When the authors performed sensitivity analyses, they found that upfront HSCT was consistently cost-effective and was more cost-effective than delayed HSCT in 90% of scenarios.

“Our findings show that upfront HSCT dominated delayed HSCT, meaning that it is less costly and more effective for intermediate-risk AML in both the US and UK healthcare settings,” Bewersdorf and colleagues wrote.

The main reason for the cost advantage of upfront HSCT was the high rates of disease relapse in patients who delay transplantation, the authors explained. Those relapses lead to excess costs, such as the need for reinduction chemotherapy and an accompanying risk of complications and healthcare utilization. The authors pointed to a study finding that 60% of intermediate-risk patients who did not undergo HSCT in their first complete remission went on to relapse, and just 54% of those patients went on to achieve a second complete remission.³

Still, Bewersdorf and colleagues wrote that the decision to proceed with HSCT or delay it is one that must be informed by an individual patient's clinical context, including factors such as the availability of a donor and the patient’s comorbidities. They added that their report can be seen as a tool to inform decision-making from a health-system perspective.

References:

1. Alhajahjeh A, Patel K, Podoltsev NA, et al. Cost-effectiveness of upfront vs delayed allogeneic hematopoietic stem cell transplant for intermediate-risk AML. Blood Adv. Published online July 28, 2025. doi:10.1182/bloodadvances.2025016532
2. Bornhäuser M, Schliemann C, Schetelig J, et al. Allogeneic hematopoietic cell transplantation vs standard consolidation chemotherapy in patients with intermediate-risk acute myeloid leukemia: a randomized clinical trial. JAMA Oncol. 2023;9(4):519-526. doi:10.1001/jamaoncol.2022.7605
3. Burnett AK, Goldstone A, Hills RK, et al. Curability of patients with acute myeloid leukemia who did not undergo transplantation in first remission. J Clin Oncol. 2013;31(10):1293-1301. doi:10.1200/JCO.2011.40.5977