Abstract Introduction: Decitabine is not approved in the United States (US) for acute myeloid leukemia (AML) because it did not improve overall survival compared with standard conventional induction treatment with cytarabine and daunorubicin (AD). We asked what would be the cost effectiveness of decitabine versus AD in AML patients older than 60 years of age. Methods: A semi-Markov model compiling survival and cost data was used based on survival probabilities from the literature. Data accounted for re-induction therapy with idarubicin, fludarabine, cytarabine and granulocyte colony-stimulating factor and consolidation therapy with high-dose cytarabine (HiDAC) but not for stem cell transplantation. The assumption-based model considered a maximum of four cycles of HiDAC and continuing decitabine until loss of benefit. Results: Assuming 1,000 patients for each treatment arm in a semi-Markov model over one year time horizon, the quality-adjusted life year (QALY) for AD vs. decitabine were 0.47 and 0.61. The percentage survival for AD and decitabine were 45.2% and 50.5%. Their costs were $168,863 and $108,084. The incremental cost-effectiveness ratio was -$60,779/0.14 = -$433,756 per QALY. By sensitivity analysis, decitabine was superior to AD in all parameters. Conclusion: Decitabine is a more cost-effective therapy for patients older than 60 years of age than AD. While cost effectiveness is certainly important, decitabine may be arguably considered for elderly newly diagnosed AML patients given the economic pressures in the US health system; however, this is not a criterion for drug approval.
Keywords: acute myeloid leukemia; elderly; cytarabine; daunorubicin and decitabine; cost effectiveness analysis