The combination of venetoclax with hypomethylating agents (VEM-HM) is considered standard of care for unfit or elderly patients with acute myeloid leukemia (AML). There are several real world studies describing complications and outcome of patients treated with venetoclax-based regimens, but none in Brazil.

To describe the clinical characteristics and outcome of patients with AML or MDS treated with venetoclax-based regimens in Brazil.

In this retrospective, multicentric study, we evaluated clinical characteristics and outcome of the first cycle of venetoclax-based regimens in the treatment of patients with AML and MDS.

A total of 114 patients were analyzed, 71% were male and the median age was 66 years (range 19-95). Most of the patients had AML (91%) with high risk disease (61%) and 4.4% had MDS. The main indication for venetoclax use was unfit patient (60%) and the most common regimen used was VEN-HM (92 patients, 81%). Intensive chemotherapy was given in to patients. Almost all patients (94%) developed neutropenia during the first cycle and 27% did not recovery from neutropenia. Among patients receiving VEN-HM, 13% received quinolone prophylaxis and 6.5% received an anti-Aspergillus azole as prophylaxis. Febrile neutropenia occurred in 54% of patients and proven or probable invasive fungal disease (IFD) was diagnosed in 7%. At the end of the first cycle, 50% patients had complete response (CR) or complete response with incomplete hematologic recovery (CRi). Among patients receiving VEN-HM, CR + CRi was 58% in first line therapy and 38% in RR. The death rate after the first cycle was 8% and 67% received a second cycle.

This is the first real world study in Brazil evaluating the use o venetoclax-based regimens in patients with AML and MDS. These results show that antibacterial and antimold prophylaxis was uncommon; yet, the rates of bacteremia and IFD was low. The 58% CR + CRi rate after the first cycle in patients receiving VEN-HMA in first line is similar to the rates observed in the randomized trial.