Cost-Effectiveness Analysis of Treating Acute Promyelocytic Leukemia Patients With Arsenic Trioxide and Retinoic Acid in the United States

Abstract

Introduction This study estimated the cost-effectiveness of arsenic trioxide (ATO) added to all-trans retinoic acid (ATRA) when used in first-line acute promyelocytic leukemia (APL) treatment. Methods A Markov cohort model was developed with three states: stable disease (during first- or second-line treatment), disease event, and death. Newly diagnosed patients with low/intermediate risk APL were included and each month could remain in their current health state or move to another. Treatment consisted of ATO + ATRA, ATRA + idarubicin (IDA), or ATRA + cytarabine (AraC) + additional chemotherapy. After an initial disease event, patients discontinued first-line and switched to a second-line ATO regimen. Efficacy/safety data were obtained from published trials; quality of life/utility estimates were obtained from the literature; costs were obtained from US data sources. Costs and outcomes over time were used to calculate incremental cost-effectiveness ratios (ICERs). Deterministic and probabilistic sensitivity analyses were conducted. Results Compared to ATRA + AraC + additional chemotherapy, ATRA + IDA treatment had ICERs of $2,933 per life year (LY) saved and $3,122 per quality-adjusted life year (QALY) gained. Compared to the ATRA + IDA regimen, first-line ATO + ATRA treatment had ICERs of $4,512 per LY saved and $5,614 per QALY gained. Results were sensitive to changes in pharmacy costs of the ATO + ATRA regimen during consolidation. Conclusion The ATO + ATRA regimen is highly cost-effective compared to ATRA + AraC + additional chemotherapy or ATRA + IDA in the treatment of newly diagnosed low to intermediate risk APL patients.

The Survival of Patients with t(15;17)(q22;q12) Positive Acute Promyelocytic Leukemia: A Study in North-East of Iran

“…Rarely, RARA may be fused with genes other than PML. The alternative variant partners of RARA include PLZF (ZBTB16), NPM1 and STAT5b (7,8 While ATO generally promotes favorable responses only in cases with PML-RARA fusion, ATRA is also effective in cases with NPM1, NUMA and FIP1L1 fusion partners (9)(10)(11). Survival studies in patients with hematologic malignancies provide valuable insights into the identification of parameters associated with high mortalities and also provide us with opportunities to improve life expectancy of at-risk patients.…”

“…ATRA and arsenic trioxide (ATO) are current medications for t(15;17)(q22;q12) positive AML. While ATO generally promotes favorable responses only in cases with PML-RARA fusion, ATRA is also effective in cases with NPM1, NUMA and FIP1L1 fusion partners ( 9 - 11 ).…”

Resource utilization and cost effectiveness of treating acute promyelocytic leukaemia using generic arsenic trioxide

“…This corroborates our results where the ATO‐based regimen has an advantage in terms of a significant reduction in the need for supportive care. In another analysis (Tallman et al , ), total costs per patient ranged from $96 940 for the ATRA + chemotherapy regimen to $136 170 for the ATO + ATRA regimen. However, compared to the ATRA + chemotherapy regimen, incremental cost‐effectiveness ratios (ICER) for ATO + ATRA were $4512 per life year (LY) saved and $5614 per quality‐adjusted life year (QALY) gained.…”

“…ATO‐based regimens thus have become the standard of care and replaced the more intensive chemotherapy‐based strategy. However, the cost of patented ATO is prohibitive, and remains a major hurdle in widespread use of ATO for treatment of APL (Lo‐Coco et al , ; Tallman et al , ; Philip et al , ).…”

“…As a result, ATO is now the major driver of cost of treatment of APL with ATO‐based regimens ($50 000 for the entire treatment). The expenditure on pharmacy itself comprises about 50–80% of the total cost of treatment with ATO compared to 12–15% with the all‐ trans ‐retinoic acid (ATRA) + chemotherapy‐based regimen (Kruse et al , ; Tallman et al , ). Despite these high costs, patented ATO remains cost effective because of its excellent clinical efficacy (Schlenk et al , ; Kruse et al , ).…”