Cost-Effectiveness of Blinatumomab for Pediatric High-Risk Relapsed Acute Lymphoblastic Leukemia in Brazil: An Economic Evaluation Using Real-World Data.

OBJECTIVES

To evaluate the cost-effectiveness of blinatumomab compared with consolidation chemotherapy in pediatric patients with high-risk relapsed B-cell acute lymphoblastic leukemia from the perspective of the Brazilian Unified Health System.

METHODS

A partitioned survival model with 3 health states (event-free survival, postevent survival, and death) was developed over a lifetime horizon. Parametric survival models were fitted to clinical trial data from Locatelli et al, and treatment effects were incorporated using hazard ratios under the proportional hazards assumption. Real-world data from a retrospective cohort of pediatric patients treated at a tertiary public hospital were used to inform cost inputs, resource utilization, and dosing assumptions. Health-state utility values were obtained from published literature. Costs and outcomes were discounted at an annual rate of 5%. Parameter uncertainty was assessed through deterministic and probabilistic sensitivity analyses (5000 Monte Carlo simulations).

RESULTS

Blinatumomab was associated with higher costs and improved outcomes compared with consolidation chemotherapy. Patients treated with blinatumomab achieved 11.73 life-years and 9.96 quality-adjusted life-years (QALYs), compared with 8.36 life-years and 6.74 QALYs with chemotherapy, resulting in incremental gains of 3.37 life-years and 3.22 QALYs. The incremental cost was R$ 340 490.07, yielding an incremental cost-effectiveness ratio of R$ 101 035.63 per life-year gained and an incremental cost-utility ratio of R$ 105 742.26 per QALY gained. Sensitivity analyses confirmed the robustness of these findings, with most probabilistic simulations falling below the willingness-to-pay threshold.

CONCLUSIONS

Blinatumomab is associated with improved clinical outcomes and represents a cost-effective alternative to consolidation chemotherapy for pediatric patients with high-risk relapsed B-cell ALL within the Brazilian Unified Health System under commonly referenced willingness-to-pay thresholds.