Abstract

Objectives

Hematopoietic stem cell transplantation is an accepted treatment of hematological malignancies, but the cost-effectiveness of this technology has not been fully explored. This study aims to assess the cost-effectiveness of stem cell transplantation from either cord blood or bone marrow/peripheral blood compared with no transplantation in adult patients with acute leukemias not expected to be cured with chemotherapy.

Methods

A systematic review was performed to estimate the efficacy of unrelated cord blood and bone marrow/peripheral blood stem cells (BM/PBSC) transplantations in adults with acute leukemia. A Markov decision analysis model using Monte Carlo simulations was used to calculate the incremental cost-effectiveness ratio (ICER) and 95% confidence intervals (CIs).

Results

The estimated cumulative survival at 1 and 10 years were 27.9% and 14%, respectively, for cord blood recipientsand 47% and 17.7%, respectively, for BM/PBSC recipients. Using conservative assumptions, the cost per life-year gained compared with no transplantation was US $16,346 (95% CI $8695, $38,006) for BM/PBSC transplantation and US $34,360 (95% CI $23,101, $89,417) for cord blood transplantation.

Conclusion

Although both types of stem cell transplantations are associated with a high short-term mortality and high cost, the cumulative gains in life-years of survivors can be substantial, resulting in ICERs compared with no transplantation that are usually considered acceptable. However there is less certainty about this conclusion with cord blood transplantation.