Blastic Plasmacytoid Dendritic Cell Neoplasm in the Dual CD123-targeted Therapy Era: A Clinical Decision Review of Treatment Selection, Sequencing, CNS Management, and Transplant Integration.

Authors

Xichang Fei, Haimin Chen, Ying Zhang, Fan Zhou

Published in

Clinical lymphoma, myeloma & leukemia. Sep 09, 2026. Epub Sep 09, 2026.

Abstract

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) has entered a dual CD123-targeted therapy era following the availability of tagraxofusp and the 2026 approval of pivekimab sunirine-pvzy. This development creates a new clinical problem: treatment can no longer be selected solely by distinguishing targeted therapy from conventional chemotherapy; clinicians must choose between mechanistically distinct CD123 platforms while integrating central nervous system (CNS) management, transplantation, comorbidity, organ function, and prior treatment. This narrative review reframes BPDCN around those decisions. It summarizes the minimum diagnostic and staging information needed before therapy; compares tagraxofusp and pivekimab using response, survival, follow-up, and transplant-bridging data; and proposes scenario-based approaches for newly diagnosed, older or frail, transplant-eligible, and relapsed or refractory patients. Tagraxofusp has the longest disease-specific experience and a defined role as remission induction and a bridge to hematopoietic cell transplantation, but capillary leak syndrome requires stringent selection and cycle-1 monitoring. Pivekimab offers a distinct antibody-drug conjugate platform with once-every-3-week dosing and activity in both treatment-naive and relapsed disease, but hepatotoxicity, including hepatic veno-occlusive disease, is a central limitation. Neither agent has established CNS-protective activity. Baseline cerebrospinal fluid assessment and routine prophylactic intrathecal chemotherapy for CSF-negative patients should therefore be distinguished from intensified therapeutic intrathecal treatment for documented CNS involvement. Early donor search, multidimensional response evaluation, and trial enrollment remain integral. The proposed algorithm is evidence-informed rather than guideline-mandated and highlights where prospective studies are most urgently needed.

PMID:
42816252
Bibliographic data and abstract were imported from PubMed on 01 Oct 2026.

Read full publication at:
Please sign in to see all details.

Stats

1-terrible, 9-excellent. How would you rate this publication? Sign in in to submit your rating.

Post a comment

You need to be signed in to post comments. You can sign in here.

Comments

There are no comments yet.