Is venetoclax/venetoclax effective in treating mds?

Treatment of acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) is difficult in older patients with comorbidities and high-risk disease factors. Venetoclax is the first Bcl-2 inhibitor that has been shown to be effective and safe in combination with azacitidine for the treatment of high-risk myeloid diseases. Myelodysplastic syndromes are another heterogeneous group of clonal hematological disorders characterized by ineffective hematopoiesis, peripheral blood (PB) cytopenias, and an increased risk of leukemic transformation.

To study the efficacy of azacitidine combined with venetoclax in AML and high-risk MDS, OS and PFS were compared between patients who received azacitidine monotherapy and those who received the combination. Azacitidine combined with venetoclax is effective and safe in elderly and frail AML and high-risk MDS patients, with a median overall survival of 22.3 months, which is higher than that reported in phase III trials (14.7 months) and higher than the historical cohort (5.94 months). Progression-free survival was better in patients treated with the drug combination compared with patients treated with azacytidine alone; clinical benefit may be increased when azacytidine and venetoclax are used as upfront therapy. But in the study, 45% of cases had grade 3 or higher thrombocytopenia, 42% had neutropenia, and 42% had febrile neutropenia.

In summary, this single-center, real-world experience with azacitidine plus venetoclax in the treatment of acute myeloid leukemia and high-risk MDS demonstrates that the drug combination is effective and safe at recommended doses in elderly and frail patients with acute myeloid leukemia and high-risk MDS, and that clinical benefit may be increased when azacitidine and venetoclax are used as upfront therapy.