15‐days duration of venetoclax combined with azacitidine in the treatment of relapsed/refractory high‐risk myelodysplastic syndromes: A retrospective single‐center study

Abstract The treatment of patients with refractory and/or relapsed (R/R) high‐risk myelodysplastic syndrome (HR‐MDS) remains a daunting clinical challenge. Venetoclax is a selective BCL‐2 inhibitor, which combined with hypomethylating agents (HMAs), increased responses and prolonged survival in unfit and previously untreated acute myeloid leukemia. We performed a retrospective study of patients with R/R HR‐MDS receiving combination azacytidine (AZA) plus 15‐days duration of venetoclax (VEN‐15d) in order to determine their efficacy and toxicity in this context. We showed that the overall response rate was 57.2% (20/35) and the median over survival was 14 months in R/R MDS. The most common treatment‐emergent adverse events were peripheral blood cytopenias and infectious complications. Our retrospective study showed that the real‐world experience of treating R/R MDS with AZA plus VEN‐15d highlights an encouraging response rate with myelosuppression being the major toxicity. Of note, VEN‐15d with AZA may salvage patients failing to respond optimally to HMAs and reduce the disease‐burden for subsequent allogeneic stem cell transplantation in our analysis. These data of combination AZA plus VEN‐15d in R/R MDS warrant further prospective evaluation in clinical trials.