Venetoclax in Patients with Relapsed or Refractory Acute Myeloid Leukemia - A Retrospective Study of a Real-World Setting

Context In November 2018, the FDA approved the use of venetoclax combined with hypomethylating agents (HMA) or low-dose cytarabine (LDAC) in patients with newly diagnosed acute myeloid leukemia (AML) of age ≥ 75 years, or with comorbidities that preclude the use of intensive chemotherapy. However, the evidence in relapse/ refractory (R/R-AML) patients is scarce. Objective To analyze the efficacy and safety of the off-label use of venetoclax in patients with R/R-AML. Design Retrospective, multicenter, observational study in 5 hospitals from Castilla y Leon region, Spain. Interventions All data were collected from patients' medical records and included: concomitant diseases, main baseline characteristics, prior treatment and the response obtained, startend date of venetoclax, dose and combination. Main outcome measures rates of CR/CRi (complete remission/complete remission with incomplete hematologic response, overall survival (OS), hematological toxicity, mortality. Results We included 10 patients (8 men/ 2 women), median age 73.5 (41-78) years, and ECOG Conclusions In the R/R-AML setting, the rate of CR/CRi and OS of venetoclax combination treatment is low. However, the patients included had very poor prognosis. These results show a real-world evolution of patients without being included in clinical trials under strict inclusion criteria that diminish the selection of patients. Further research is warranted.