Safety and Efficacy of Low-Dose Venetoclax Plus Voriconazole in Patients With Acute Myeloid Leukemia Unfit for Intensive Chemotherapy

Low-dose venetoclax plus strong CYP3A4 inhibitor voriconazole were commonly used for acute myeloid leukemia (AML) patients who were unfit for intensive chemotherapy in China. However, the efficacy and safety of this schedule have not been well investigated. We analyzed clinical data from 54 patients with a median age of 67 years. Thirty patients received a standard dose of venetoclax plus azacitidine (cohort 1), whereas another 24 patients received low-dose venetoclax plus voriconazole plus azacitidine (cohort 2). The composite complete remission (complete remission or complete remission with incomplete hematologic recovery; CR/CRi) rate was 76.7% (23/30) in cohort 1 and 87.5% (21/24) in cohort 2 (p = 0.483). At a median follow-up of 16 months, the median progression-free survival was 12 months in cohort 1 and 18 months in cohort 2 (p = 0.241). The median overall survival was 14 months in cohort 1 and 19 months in cohort 2 (p = 0.453). Key adverse events included cytopenia and infections. Grade 3 or higher infections occurred in 36.7% of the patients in cohort 1 and 20.8% of those in cohort 2. In conclusion, this study demonstrated the safety and effectiveness of the combination of low-dose venetoclax and voriconazole in unfit AML.