Venetoclax Plus Azacitidine as a Bridge Treatment to Allogeneic Stem Cell Transplantation in Unfit Patients with Acute Myeloid Leukemia

Simple Summary The study explores a promising treatment avenue for acute myeloid leukemia (AML), emphasizing the drug combination venetoclax and azacitidine (VEN plus AZA) to improve patient survival and enable stem cell transplantation in those unsuitable for intensive chemotherapy. Comparative analysis with conventional treatment shows comparable survival rates, influenced by genetic factors. In conclusion, for older AML patients unable to endure intensive chemotherapy, the VEN plus AZA regimen emerges as a viable, less taxing option, offering the potential for curative stem cell transplantation.Abstract Background: Allogeneic hematopoietic stem cell transplantation (HSCT) is rarely recommended for unfit patients with newly diagnosed acute myeloid leukemia (AML). Patient survival can improve with venetoclax plus azacitidine (VEN plus AZA). However, the long-term outcome of this treatment strategy is still unsatisfactory. The high response and low treatment toxicity rates of patients receiving VEN plus AZA can provide an opportunity for HSCT among unfit patients. Nevertheless, the outcomes and complications of VEN plus AZA, followed by HSCT, remain unclear. Methods: This single-center retrospective study aimed to compare patients with newly diagnosed AML receiving VEN plus AZA as induction therapy (n = 27) to those receiving the conventional I3A7 regimen as induction therapy (n = 34). Result: The 1-year overall survival, relapse, and non-relapse mortality rates in the two groups were similar. The cytogenetic risks and the hematopoietic cell transplantation-specific comorbidity index are the most significant predictive factors of overall survival. Conclusion: In older patients unfit for intensive chemotherapy, a low-intensity regimen with VEN plus AZA is a suitable bridge therapy. Furthermore, allo-HSCT is feasible and can be a curative option.