OBJECTIVES: Acute myeloid leukemia (AML) is an aggressive, fast-growing hematological cancer with associated infection, anemia, and bleeding. Due to the limited efficacy of current treatment, the five-year survival rate of AML patients who are ineligible for intensive chemotherapy (“unfit”) is only 5%. Venetoclax has demonstrated superior clinical efficacy in this group of patients. This study aims to assess the cost-effectiveness of venetoclax in combination with azacitidine (Ven+AZA) versus azacitidine (AZA) for patients newly diagnosed with unfit AML, from the healthcare perspective in China.

METHODS: A three-state partitioned survival model (event-free survival(EFS), progressive/relapsed, and death) was developed, in which composite complete remission(CR/CRi) was further introduced as a substate of EFS to reflect the clinical reality. Based on findings from real-world studies and guidelines in China, the baseline age was set at 60 years old. A 15-year time horizon was used to balance the long-term uncertainty of model stimulation and capturing most disease progression. Clinical inputs were obtained from VIALE-A trial Asian group (e.g., CR/CRi Ven+AZA 70.49% vs. AZA 28.13%) to better represent the efficacy in Chinese population. Cost inputs were informed by public prices and literature review. Utility inputs were derived from EQ-5D-5L data in VIALE-A using Chinese tariff. Sensitivity analyses were also performed.

RESULTS: Over a 15-year horizon, Ven+AZA resulted in 1.7 QALYs gained compared to AZA, which was driven by its improved CR/CRi and survival in Asian population (even numerically superior to the ones in overall population). Meanwhile, Ven+AZA could notably save the costs of blood transfusions, anti-infective therapy, ICU rescue and palliative care. The ICER result was 20,564 CNY/QALY, which is only a quarter of the willingness-to-pay threshold of 1 times GDP per capita in China in 2023. Sensitivity analyses results demonstrated the robustness

CONCLUSIONS: The study suggests that Ven+AZA is a cost-effective regimen for newly diagnosed unfit AML patients in China.