Objective  To rapidly evaluate the efficacy, safety, and cost-effectiveness of venetoclax (Ven) in non-M3 acute myeloid leukemia (AML), and to provide an evidence-based basis for rational clinical treatment.

Methods  PubMed, Cochrane Library, Embase, CNKI, WanFang Data databases, and relevant health technology assessment (HTA) websites were searched to collect relevant literature and reports on Ven treatment for non-M3 AML, with a search timeframe from the establishment of the database/website to November 1st, 2024. Two researchers independently screened literature, extracted data, and assessed quality, and then qualitatively described and analyzed the results.

Results  A total of 11 pieces of literature were included, including 5 systematic reviews/Meta-analysis, 4 pharmacoeconomic studies, and 2 HTA reports. In terms of efficacy, compared with the control group, non-M3 AML patients receiving Ven treatment had a higher clinical remission rate (P<0.05), a longer event-free survival (EFS) (P<0.05) and a similar or longer overall survival (OS) (P<0.05). Regarding safety, compared to Azacitidine(Aza) monotherapy, Ven+Aza resulted in a higher likelihood of febrile neutropenia in non-M3 AML patients (P<0.05). Non-M3 AML patients receiving Ven+low-dose cytarabine (LDAC) had a higher risk of developing thrombocytopenia compared with LDAC monotherapy (P<0.05). However, the early 30-day mortality rate was lower in the Ven+chemotherapy group than that in the chemotherapy alone group (P<0.05), presenting an acceptable security profile overall. In terms of cost-effectiveness, Ven was cost-effective in non-M3 AML patients compared with the control group.

Conclusion  Ven has manifested remarkable efficacy and acceptable security profile among patients with non-M3 AML, thus proving to be a medium to long-term cost-effective treatment modality.

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