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Updates in Novel Therapies for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

  • Acute Myeloid Leukemias (H Erba, Section Editor)
  • Published:
Current Hematologic Malignancy Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, clinically aggressive hematologic malignancy that has heterogeneous presentation and can involve the skin, lymph nodes, and bone marrow. Recent advancements in our patho-biologic understanding of the disease have led to the development of new targeted therapies for BPDCN. In this review, we aimed to describe some of the novel treatments that are being put forward for the management of BPDCN.

Recent Findings

Tagraxofusp is the first CD123-targeted therapy approved as the first ever targeted treatment of BPDCN in patients aged 2 years and older. This agent was approved based on a pivotal clinical trial that showed that it was associated with high rates of clinical responses in both treatment-naïve and treatment-experienced patients. The most serious adverse event was occurrence of the capillary leak syndrome. Other targeted therapies are actively being investigated in clinical trials. These include other CD123-targeted approaches, as well as active investigation in targets beyond CD123, such as the BCL-2 inhibitor, venetoclax.

Summary

BPDCN is a rare hematologic clonal disorder with historically poor outcomes. Newer targeted therapies have been recently introduced, with promising results and novel toxicities that are important to recognize and understand. Stem cell transplantation after achievement of complete remission remains the mainstay of therapy among younger/fit, eligible patients, regardless of treatment modality used.

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Funding

This research is supported in part by the M. D. Anderson Cancer Center Support Grant P30 CA016672.

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Authors and Affiliations

  1. Department of Internal Medicine, The University of Texas School of Health Sciences at Houston, Houston, TX, USA

    Minas P. Economides

  2. Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC, USA

    David Rizzieri

  3. Department of Leukemia, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030, USA

    Naveen Pemmaraju

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  1. Minas P. Economides
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Correspondence to Naveen Pemmaraju.

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Conflict of Interest

Minas P. Economides declares no conflict of interest.

David Rizzieri declares the following: advisory and consultative work with Immunogen, Mustang Bioscience, Stemline; honoraria from Stemline.

Naveen Pemmaraju declares the following: consulting/honorarium from Celgene, Stemline, Incyte Corporation, Novartis, MustangBio, Roche Diagnostics, and LFB. Research funding/clinical trials support from Stemline, Novartis, Abbvie, Samus, Cellectis, Plexxikon, Daiichi-Sankyo, Affymetrix, and SagerStrong Foundation.

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Economides, M.P., Rizzieri, D. & Pemmaraju, N. Updates in Novel Therapies for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). Curr Hematol Malig Rep 14, 515–522 (2019). https://doi.org/10.1007/s11899-019-00556-2

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