Hematopoietic effects of tagraxofusp in treatment-naive patients with blastic plasmacytoid dendritic cell neoplasm.

ABSTRACT:

Background

Blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic neoplasm that expresses CD123, is often observed in the bone marrow (BM), as well as skin, blood, and viscera. Tagraxofusp, a first-in-class CD123-targeted therapy, is the only drug approved to treat BPDCN.

Methods

The authors conducted a post hoc analysis of the pivotal phase 2 trial to evaluate hematopoietic and BM effects of first-line tagraxofusp treatment (12 µg/kg per day on days 1-5 of a 21-day cycle) over time. This trial was registered at www.

Clinicaltrials

gov (ClinicalTrials.gov identifier NCT02113982).

Results

Of 66 treatment-naive patients in this analysis, 32 had baseline BM disease, and 34 had no BM disease. Over the course of tagraxofusp monotherapy treat

SUBMITTER: Konopleva M 

PROVIDER: S-EPMC12766059 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature