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Review
. 2024 Apr;24(4):e130-e137.
doi: 10.1016/j.clml.2023.12.010. Epub 2023 Dec 20.

Recent Advances in the Biology and CD123-Directed Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm

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Free article
Review

Recent Advances in the Biology and CD123-Directed Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm

Naveen Pemmaraju et al. Clin Lymphoma Myeloma Leuk. 2024 Apr.
Free article

Abstract

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive myeloid malignancy of the dendritic cell lineage that affects patients of all ages, though the incidence appears to be highest in patients over the age of 60 years. Diagnosis is based on the presence of plasmacytoid dendritic cell precursors expressing CD123, the interleukin-3 (IL-3) receptor alpha, and a distinct histologic appearance. Timely diagnosis remains a challenge, due to lack of disease awareness and overlapping biologic and clinical features with other hematologic malignancies. Prognosis is poor with a median overall survival of 8 to 14 months, irrespective of disease presentation pattern. Historically, the principal treatment was remission induction therapy followed by a stem cell transplant (SCT) in eligible patients. However, bridging to SCT is often not achieved with induction chemotherapy regimens. The discovery that CD123 is universally expressed in BPDCN and is considered to have a pathogenetic role in its development paved the way for the successful introduction of tagraxofusp, a recombinant human IL-3 fused to a truncated diphtheria toxin payload, as an initial treatment for BPDCN. Tagraxofusp was approved in 2018 by the United States Food and Drug Administration for the treatment of patients aged 2 years and older with newly diagnosed and relapsed/refractory BPDCN, and by the European Medicines Agency in 2021 for first-line treatment of adults. The advent of tagraxofusp has opened a new era of precision oncology in the treatment of BPDCN. Herein, we present an overview of BPDCN biology, its diagnosis, and treatment options, illustrated by clinical cases.

Keywords: BPDCN; Clinical cases; Myeloid malignancy; Precision oncology; Tagraxofusp.

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Conflict of interest statement

Disclosures Naveen Pemmaraju: 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): Consultancy/Scientific Advisory Board/Speaking; United States Department of Defense, National Institute of Health/National Cancer Institute (NIH/NCI): Research (Grant); Affymetrix: Research Funding; Dan's House of Hope: Membership on an entity's Board of Directors/Management; ASH Committee on Communications, ASCO Cancer.Net Editorial Board: Leadership; Karger Publishers: Licences; HemOnc Times/Oncology Times: Uncompensated. Eric Deconinck: Stemline Therapeutics: Consultancy, honoraria; ImmunoGen: Consultancy, honoraria; Chugai: Research funding; Novartis: Research funding. Priyanka Mehta: Pfizer, AbbVie, Celgene, Daiichi Sankyo, Jazz Pharmaceuticals, Stemline Therapeutics: Advisory board/honoraria. Irwin Walker: Sanofi: Research funding. Marco Herling: AbbVie, BeiGene, Jazz, Janssen, Stemline Therapeutics, Takeda: Consultancy; Mundipharma EDO, Janpix, Novartis, Roche: Funding of preclinical research. Francine Garnache-Ottou: Stemline Therapeutics, LFB: Consultancy. Nadia Gabarin: Declarations of interest: none. Clinton Campbell: Declarations of interest: none. Johannes Duell: Stemline: Lecturing Yakir Moshe: AbbVie: Advisory board, research funding, speaker; Astellas, Medison: Advisory board, speaker; Gilead, Novartis, Stemline: Advisory board Tariq Mughal: Informa Publishing, Oxford University Press: Royalties. Consultant: Stemline Therapeutics Inc, New York, NY, USA. Mohamad Mohty: Jazz Pharmaceuticals: Research funding, honoraria. Emanuele Angelucci: Menarini/Stemline, Novartis: Honoraria; bluebird bio, Glaxo, Gilead, Roche: Board of Directors or advisory committees; Vifor Pharma, BMS, Vertex Inc.: Participation DMC.

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