Real-World Experience With Venetoclax-Based Therapy in Acute Myeloid Leukemia: Insights From the Czech Republic

Venetoclax (VEN) combined with azacitidine (AZA) has become the standard treatment for patients (pts) with newly diagnosed (ND) acute myeloid leukemia (AML) who are unfit for intensive chemotherapy. This study reports on a real-world cohort of 163 patients with ND AML treated with VEN/AZA across 7 hospitals in the Czech Republic.

The median age at diagnoses was 72 (39-98) years. Standard VEN/AZA (7 + 28; ie, AZA 75mg/m² for 7 days + VEN for 28 days) was used in 89% of patients (145/163), with 73% (119/163) receiving a reduced dose of venetoclax (100 mg or 200 mg) due to concomitant azole antifungal prophylaxis. Treatment was considered palliative or nonintensive (including potential allogeneic transplant candidates) based on the clinician's decision.

Among 150 evaluable patients, 71% (107/150) achieved composite complete remission (CCR = CR + CRi), with 28% (32/115) achieving MRD negativity. Median overall survival (OS) was 5 months for the palliative cohort and 21 months for the nonintensive cohort. Day 30 and day 60 mortality rate were 7% and 13%, respectively. Allogeneic transplantation (HSCT) was performed in 15% (25/163) of pts, with the median OS not reached in this group. Mutations in NPM1, and IDH1/2 were associated with improved survival, while FLT3, ASXL1, TP53, deletion/monosomy 7, and complex karyotype were linked to poorer outcomes.

Data suggest that TP53 patients benefit from treatment if they achieve CR within the first 2 cycles. Discontinuing VEN/AZA in low-risk MRD-negative patients does not appear to worsen overall survival, though long-term outcomes remain to be seen.