Reduced Duration and Dosage of Venetoclax Is Efficient in Newly Diagnosed Patients with Acute Myeloid Leukemia

Abstract

venetoclax (VEN) and Azacitidine (AZA) combination have shown improved survival and excellent responses in patients with untreated Acute myeloid leukemia (AML). However, frequent dose reduction (or discontinuation) occurred due to therapy-related toxicity and may limit triplet combination tolerance. This study retrospectively analyzed the characteristics and outcomes of newly diagnosed AML patients who received 100mg VEN for 14 days combined with AZA at our institution. Overall, 36 patients were enrolled, the median age at diagnosis was 64 years; after a median follow-up of 15(range 4–29) months, median overall survival (OS) and progression-free survival (PFS) for the total cohort were 17 (4–29) months and 12 (1–28) months, and for patients achieving composite complete remission (CRc) were not reached. Overall response rate (ORR) was 69.4% and CRc rate were 66.7% for the total cohort. Subgroup analysis revealed that NPM1 mutations and FAB-M5 were associated higher response rates, while adverse ELN risk group predicted inferior response. ASXL1, NPM1 and IDH1/2 mutation was statistically significant impact the PFS duration. The most common adverse events were hematological, with grade 3 or 4 neutropenia, thrombocytopenia, and anemia reported in 36%, 25%, 44% of patients, respectively. In summary, the VEN combination regimen remains effective for newly diagnosed AML in the real world despite VEN dose reductions.