Treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN): focus on the use of tagraxofusp and clinical considerations

Abstract

BPDCN is an aggressive myeloid malignancy with a poor prognosis. It derives from the precursors of plasmacytoid dendritic cells and is characterized by CD123 overexpression, which is seen in all patients with BPDCN. The CD123-directed therapy tagraxofusp is the only approved treatment for BPDCN; it was approved in the US as monotherapy for the treatment of patients aged ≥2 years with treatment-naive or relapsed/refractory BPDCN. Herein, we review the available data supporting the utility of tagraxofusp in treating patients with BPDCN. In addition, we present best practices and real-world insights from clinicians in academic and community settings in the US on how they use tagraxofusp to treat BPDCN. Several case studies illustrate the efficacy of tagraxofusp and discuss its safety profile, as well as the prevention, mitigation, and management of anticipated adverse events.

Keywords:

• Blastic plasmacytoid dendritic cell neoplasm
• tagraxofusp
• SL-401
• Elzonris
• CD-123 directed therapy

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Acknowledgments

Editorial and medical writing assistance was provided by Joanne Franklin, PhD, CMPP, from Aptitude Health, The Hague, the Netherlands, and funding of this was supported by Stemline Therapeutics Inc., NY, USA. The authors are fully responsible for all content and editorial decisions for this manuscript.

Disclosure statement

Naveen Pemmaraju: Consultancy fees: Pacylex Pharmaceuticals, Astellas Pharma US, ImmunoGen, Inc, Bristol-Myers Squibb Co., Cimeio Therapeutics AG, EUSA Pharma, Menarini Group, Blueprint Medicines, CTI BioPharma, ClearView Healthcare Partners, Novartis Pharmaceutical, Neopharm, Celgene Corporation, AbbVie Pharmaceuticals, Pharma Essentia, Curio Science, DAVA Oncology, Imedex, Intellisphere, CancerNet, Harborside Press, Aptitude Health, Medscape, Magdalen Medical Publishing, OncLive, CareDx, Patient Power, Physcican Education Resource (PER). Participation on Data Safety Monitoring Board or Advisory Board: Pacylex Pharmaceuticals, Astellas Pharma US, ImmunoGen, Inc, Bristol-Myers Squibb Co., Cimeio Therapeutics AG, EUSA Pharma, Menarini Group, Blueprint Medicines, CTI BioPharma, ClearView Healthcare Partners, Novartis Pharmaceutical, Neopharm, Celgene Corporation, AbbVie Pharmaceuticals, Pharma Essentia, Curio Science, DAVA Oncology, Imedex, Intellisphere, CancerNet, Harborside Press, Aptitude Health, Medscape, Magdalen Medical Publishing, OncLive, CareDx,, Patient Power, Physician Education Resource (PER). Leadership role: ASH Committee on Communications, ASCO Cancer. Net Editorial Board, Dan’s House of Hope, Karger Publishers. Yazan F. Madanat: Consultancy fees: Blueprint Medicinces, GERON, OncLive. Honoraria: Blueprint Medicinces, GERON, OncLive, Sierra Oncology, Stemline Therapeutics, Morphosys, Taiho Oncology, Novartis, Rigel Pharmaceuticals. Support for meetings/travel: Blueprint Medicines, Morphosys. David Rizzieri: Consultancy fees: Amgen, Celltrion, Kite, Arog Pharmaceuticals, Pharmacyclics, Pfizer, Novartis, Sanofi-Aventis, Jazz, AbbVie, Seattle Genetics, Incyte, Gilead. Honoraria: Celgene, Teva, Amgen, Celltrion/Teva, Kite, Arog Pharmaceuticals, Pharmacyclics, Pfizer, Novartis, Sanofi-Aventis, Jazz, AbbVie, Seattle Genetics, Incyte, Gilead. Salman Fazal: Honoraria: Amgen, Incyte Corporation, Jazz Pharmaceuticals, Gilead, GlaxoSmithKline, Novartis, Bristol-Myers Squibb, Sanofi Genzyme, Taiho Pharmaceuticals, Servier Pharmaceuticals, Janssen Pharmaceuticals, CTI Biopharm, Pharmaessentia, Blueprint pharmaceuticals, Takeda Pharmaceuticals, Karyopharm. Raajit Rampal: Consultancy fees: Constellation, Incyte, Celgene, Promedior, CTI, Jazz Pharmaceuticals, Blueprint, Stemline, Galecto, Pharmessentia, Abbvie. Research funding: Incyte, Stemline, and Constellation. Gabriel Mannis: Consultancy fees: AbbVie, Agios, Astellas, BMS/Celgene, Genentech, Macrogenics, Servier, Stemline. Research funding: Astex, Celgene/BMS, Forty Seven/Gilead, Glycomimetics, ImmuneOnc, Immunogen, Jazz, Syndax. Eunice S. Wang: Consultancy fees: AbbVie, Astellas, BMS, Daiichi Sankyo, Genentech, Gilead, GlaxoSmithKline, Janssen, Jazz, Kite Pharmaceuticals, Kura Oncology, Novartis, Pfizer, Stemline Therapeutics, Takeda. Honoraria: Stemline Therapeutics, Kura, Pfizer, DAVA Oncology. Data Safety or Advisory Board participation: AbbVie, Gilead. James Foran: Consultancy fees: Revolution Medicine. Honoraria: Novartis, Servier and Pfizer. Research funding: AbbVie, Boehringer Ingelheim, Actinium Pharmaceuticals, Aprea Therapeutics, Aptose Biosciences, H3 Biomedicine, Kura Oncology, Tolero Pharmaceuticals, Trillium Therapeutics, Xencor and Takeda/Millennium. Andrew A. Lane: Consultancy fees: Cimeio Therapeutics, IDRx, Jnana Therapeutics, N-of-One, ProteinQure, Qiagen. Research funding: AbbVie, Stemline Therapeutics.

Correction Statement

This article was originally published with errors, which have now been corrected in the online version. Please see Correction (http://dx.doi.org/10.1080/10428194.2024.2327797)

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.