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Venetoclax Regimens Assessed in Patients with AML

Am J Hematol; ePub 2017 Dec 23; DiNardo, et al

Certain low-intensity chemotherapy regimens appear to be reasonable options even in patients with relapsed/refractory acute myeloid leukemia (AML) and other myeloid disorders, according to a study involving 43 individuals. Participants’ median age was 68 years. Most had AML, and the remainder had either myelodysplastic syndrome or blastic plasmacytoid dendritic cell neoplasm. They were treated with hypomethylating agents (HMAs) or low-dose cytarabine, in combination with venetoclax in the salvage setting. Among the results:

  • 72% received HMAs and 19% low-dose cytarabine with venetoclax.
  • 9 patients (21%) achieved an objective response, including 2 complete responses, 3 CRi, and 4 morphologic leukemia-free state.
  • Patients survived a median of 3 months.
  • Estimated 6-month survival was 24%.
  • ~One-fourth of patients with intermediate-risk cytogenetics responded.
  • 27% of those with IDH1/2-mutant responded, as did half RUNX1-mutated patients.
  • Of the 3 responding patients with adverse cytogenetics, all had concurrent RUNX1 mutations.


DiNardo C, Rausch C, Benton C, et al. Clinical experience with the BCL2-inhibitor venetoclax in combination therapy for relapsed and refractory acute myeloid leukemia and related myeloid malignancies. [Published online ahead of print December 23, 2017]. Am J Hematol. doi:10.1002/ajh.25000.