Tagraxofusp for Blastic Plasmacytoid Dendritic Cell Neoplasm

Hematol Oncol Clin North Am. 2020 Jun;34(3):565-574. doi: 10.1016/j.hoc.2020.01.005. Epub 2020 Mar 18.

Abstract

Tagraxofusp, a CD123-targeted immunotoxin, is the first FDA-approved treatment for patients 2 years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN). It has been shown to be safe and effective in treatment-naïve and previously treated adult patients, with high rates of successful bridging to hematopoietic stem cell transplantation. The pediatric experience is more limited but demonstrates safety. Given the risk of potentially fatal capillary leak syndrome with tagraxofusp, judicious patient selection is recommended. Combination therapy with hypomethylating agents and/or BCL-2 inhibitors are rational next lines of investigation, especially in patients ineligible to receive high-dose chemotherapy.

Keywords: BPDCN; CD123; Diphtheria toxin; Interleukin-3; Tagraxofusp.

Publication types

  • Meta-Analysis
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Age Factors
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Clinical Trials as Topic
  • Combined Modality Therapy
  • Dendritic Cells / drug effects*
  • Dendritic Cells / metabolism
  • Dendritic Cells / pathology
  • Disease Management
  • Humans
  • Myeloproliferative Disorders / diagnosis
  • Myeloproliferative Disorders / drug therapy*
  • Myeloproliferative Disorders / etiology
  • Recombinant Fusion Proteins / pharmacology
  • Recombinant Fusion Proteins / therapeutic use*
  • Treatment Outcome

Substances

  • Antineoplastic Agents
  • Recombinant Fusion Proteins
  • tagraxofusp