The costs and cost-effectiveness of allogeneic peripheral blood stem cell transplantation versus bone marrow transplantation in pediatric patients with acute leukemia

Abstract

In a retrospective study, we evaluated the cost and cost-effectiveness of allogeneic peripheral blood stem cell transplantation (PBSCT) (n = 30) compared with bone marrow transplantation (BMT) (n = 110) in children with acute leukemia after 1 year of follow-up. Treatment success was defined as disease-free survival at 1 year posttransplantation. For patients at standard risk for disease, the treatment success rate was 57.1% for PBSCT recipients and 80.3% for BMT recipients (P = not significant [NS]). The average total cost per treatment success at 1 year in the standard-risk disease group was $512,294 for PBSCT recipients and $352,885 for BMT recipients (P = NS). For patients with high-risk disease, the treatment success rate was 18.8% for PBSCT recipients and 23.5% for BMT recipients (P = NS). The cumulative average cost was $457,078 in BMT recipients and $377,316 in PBSCT recipients (P = NS). Point estimates of the incremental cost-effectiveness ratio (ICER) indicate that in patients with standard-risk disease, allogeneic BMT had lower costs and greater effectiveness than PBSCT (ICER, -$687,108; 95% confidence interval [CI], $2.4 million to dominated). For patients with high-risk disease, BMT was more effective and more costly, and it had an ICER of $1.69 million (95% CI, $29.7 million to dominated) per additional treatment success. The comparative economic evaluation provides support for BMT in standard-risk patients, but much uncertainty precludes a clear advantage of either treatment option in patients with high-risk disease. More studies using larger and randomized controlled trials are needed to confirm the long-term cost-effectiveness of each procedure.

Figure 1. Incremental Cost-Effectiveness (ICE) of Peripheral Blood Stem Cell Transplantation (A) and Bone Marrow Transplantation (B) – Standard Risk Group

Incremental Cost-Effectiveness was performed using 1000 Monte Carlo simulations to estimate the 95% confidence ellipse. Most points (77%) fell in quadrant II, indicating a higher probability that PBSCT (A) was less effective and more costly and being dominated by BMT.

Figure 2. Incremental Cost-Effectiveness (ICE) of Peripheral Blood Stem Cell Transplantation (A) and Bone Marrow Transplantation (B) – High Risk Group

Incremental Cost-Effectiveness was performed using 1000 Monte Carlo simulations to estimate the 95% confidence ellipse. There is a 37% probability that PBSCT (A) would be less costly but also less effective compared to BMT (B) (quadrant III). PBSCT has an equivalent opportunity (24%) of being the dominant option (quadrant IV) or being the dominated choice (quadrant II) over BMT.