Tagraxofusp, Azacitidine, and Venetoclax (TAG-AZA-VEN) Triplet Therapy Shows Efficacy, Tolerability, and Transplant Potential in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Results of a Phase 2 Trial

At the 2025 ASH Annual Meeting, Andrew A. Lane, MD, presented phase 2 findings evaluating the triplet regimen of tagraxofusp, azacitidine, and venetoclax (TAG-AZA-VEN) in patients with newly diagnosed and relapsed/refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN). In this multicenter study of 27 adults, the combination demonstrated high composite complete response rates, including 88% in the frontline cohort and 64% in the relapsed/refractory cohort, with rapid time to response. The safety profile was consistent with prior experience, with manageable capillary leak syndrome observed during cycle 1 and no new safety signals identified. A substantial proportion of patients proceeded to allogeneic stem cell transplant, suggesting the regimen may serve as an effective bridge to curative-intent therapy. Survival outcomes compared favorably with historical data for single-agent tagraxofusp. These results support TAG-AZA-VEN as a promising, CD123-targeted, transplant-enabling treatment strategy for patients with BPDCN.