OBJECTIVES: A cost-effectiveness model was developed to compare UM171-expanded UCB (E-UCB) to unexpanded UCB (UCB) and unrelated donor transplant (URD) from a Canadian payer perspective.

METHODS: A lifetime-horizon Markov model was developed to evaluate various stem cell sources in patients with haematological malignancies requiring stem cell transplantation (SCT). Health states included: Diagnosis, Pre-progression (SCT recovery), Post-SCT (without GVHD), Post-SCT (with GVHD), and Graft failure/Relapse. UCB and URD estimation of patients transitioning to each health state was derived from published literature (pooled Kaplan-Meier data) and extrapolated using exponential functional forms. E-UCB efficacy data were based on the Kaplan-Meier estimator function derived from the patient level data. Model utilities varied by health state and were based on SCT-specific literature and included complication disutility. Resource utilization and cost calculations associated with each SCT source included costs of SCT, routine medical care, complications (acute and chronic GVHD, infection, and veno-occlusive disease), indirect and mortality (end of life) costs. The primary economic endpoints assessed were life years (LYs) and quality-adjusted life years (QALYs).

RESULTS: When published data were applied in the economic model, E-UCB showed economic dominance over both UCB and URD, with 1.50 and 1.46 LYs gained, respectively, and 1.23 and 1.19 QALYs gained, respectively. The total cost of treating one patient with E-UCB was estimated at $232,842, compared with $291,501 for UCB and $237,355 for URD over a lifetime horizon. While treatment cost was the highest for E-UCB, these costs were offset by lower relapse and GVHD costs in the E-UCB arm resulting in lower overall costs. Deterministic and probabilistic sensitivity analyses generally showed consistency with base case findings.

CONCLUSIONS: E-UCB appeared to be cost-saving and associated with a QALYs gain when compared to UCB and URD. Sensitivity analyses supported the findings and contributed to the robustness of the economic case.