Venetoclax and hypomethylating agents versus tagraxofusp in older patients with blastic plasmacytoid dendritic cell neoplasm.

ABSTRACT: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a highly aggressive and rare hematologic malignancy characterized by poor response to multiagent chemotherapy and dismal survival outcomes of 8-16 months. Tagraxofusp, the first-in-class CD123-directed antineoplastic agent, has emerged as a highly effective therapy and is the only FDA approved drug for BPDCN. Nonetheless, significant treatment-related toxicities with tagraxofusp such as hepatotoxicity and capillary leak syndrome are unfortunately not uncommon and can be prohibitive for older or unfit patients. The success of venetoclax (VEN) with a hypomethylating agent (HMA) has recently been described in the literature however, clinical outcomes are limited to case reports and small case series. To confirm these findings, we perform

SUBMITTER: Lee BJ 

PROVIDER: S-EPMC12031789 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature