How effective is the treatment of MDS patients with venetoclax/venetoclax?

Acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) are very difficult to treat in elderly patients, especially those with comorbidities and high-risk disease factors. Venetoclax, the first Bcl-2 inhibitor, has shown efficacy and safety in combination with azacitidine in the treatment of high-risk myeloid diseases. Myelodysplastic syndrome is a heterogeneous clonal hematological disorder characterized by failure of hematopoiesis, peripheral blood (PB) cytopenias, and an increased risk of leukemic transformation.

To further explore the therapeutic effect of combination therapy with azacitidine and venetoclax in AML and high-risk MDS, we compared overall survival (OS) and progression-free survival (PFS) between patients who received azacitidine alone and those who received the combination therapy. The study results show that azacitidine combined with venetoclax is not only effective but also safe for elderly and frail patients with AML and high-risk MDS. The median overall survival of these patients reached 22.3 months, which was not only higher than the 14.7 months reported in the phase III trial, but also far exceeded the 5.94 months of the historical cohort. Patients treated with the combination showed a longer duration of progression-free survival compared with patients treated with azacytidine alone. Of particular note, patients may experience greater clinical benefit when azacytidine and venetoclax are used as upfront therapy.

In summary, based on the real-world experience of a single center, azacitidine combined with venetoclax shows significant efficacy in the treatment of acute myeloid leukemia and high-riskMDS. At recommended doses, the drug combination is effective and safe in frail, elderly patients with acute myeloid leukemia and high-risk MDS. Especially when azacitidine and venetoclax are used as early treatments, the clinical benefit of patients may be further improved.