Novel Therapies for Blastic Plasmacytoid Dendritic Cell Neoplasm

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an orphan hematologic malignancy with poor outcomes. Tagraxofusp (SL-401) was the first drug approved specifically for patients with BPDCN, in 2018. Additional therapeutic strategies are still needed to improve survival and minimize treatment-related toxicity. This article outlines novel targeted approaches that are in preclinical or clinical development for BPDCN. Although there is no known targetable genetic abnormality that defines BPDCN, data from functional testing of primary tumors, gene expression analyses, and adaptation of targeted drug approaches from other cancers to BPDCNs harboring specific mutations have nominated several promising strategies.