Longer survival and favorable healthcare resource utilization of tagraxofusp versus venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm: results of a real-world analysis.

BACKGROUND: Tagraxofusp is a first-in-class CD123-targeted therapy approved for blastic plasmacytoid dendritic cell neoplasm (BPDCN). Although venetoclax is not approved for BPDCN, limited use has been reported. We evaluated overall survival (OS) and healthcare resource utilization in patients with BPDCN treated with tagraxofusp- or venetoclax-based regimens in a US real-world setting. METHODS: Claims data were analyzed from patients with ≥1 BPDCN diagnosis (January 2016-December 2023) and initiation of tagraxofusp- or venetoclax-based therapy. Index date was defined as first inpatient tagraxofusp dosing or venetoclax-based therapy initiation. Patients were matched 1:1 by line of therapy (LOT). RESULTS: Each cohort had 47 patients. Tagraxofusp-based treatment was associated with longer median OS versus venetoclax-based (35.3 vs 10.5 months), consistent across patients receiving monotherapy, combination, or transplantation. Tagraxofusp-treated patients were more likely to proceed to subsequent LOT versus venetoclax-treated (47% vs 11%). During months 2-6, tagraxofusp reduced median inpatient hospitalization duration (3 vs 7 days) and transfusion rates (3% vs 32%) versus venetoclax. CONCLUSION: In this retrospective claims-based analysis, tagraxofusp was associated with longer survival versus venetoclax, including across treatment contexts. These real-world results are consistent with the established role of tagraxofusp as the first-line standard of care for patients with BPDCN, irrespective of transplant eligibility, and support continued evaluation of tagraxofusp-based regimens as a backbone for future combinations.