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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) has to be considered an orphan tumoral disease. BPDCN is a good model concerning the structuring and the organization of a concerted medical program on a nation-based, transnational, or international level. In 2019 in France the diagnosis process for BPDCN was clearly established. Two prospective clinical trials are ongoing. Because of the difficulties in diagnostic procedures and the rarity of the disease it is important that European countries collaborate to build a real European network to ensure the best and equitable medical care to all BPDCN patients., Competing Interests: Disclosure E. Deconinck belongs to the EBMT and SFH, is a member of a subcommittee of the Agence française de la Biomédecine, and participates in boards for Stemline therapeutics and ImunoGen., (Copyright © 2020 Elsevier Inc. All rights reserved.)

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, aggressive hematological malignancy, derived from plasmacytoid dendritic cells. It mainly occurs in older adults, but has been reported across all age groups. Most patients present with skin lesions with or without marrow involvement and leukemic dissemination. Treatment with high-risk acute lymphoblastic leukemia therapy regimens with central nervous system prophylaxis is recommended in pediatric patients. Stem cell transplant in children is recommended for relapsed/refractory disease or high-risk disease at presentation. New targeted therapies including the recently FDA-approved anti-CD123 cytotoxin show great promise in improving the response rate., Competing Interests: Disclosure The authors have nothing to disclose., (Copyright © 2020 Elsevier Inc. All rights reserved.)

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., Competing Interests: Disclosure D. Hammond has nothing to disclose. N. 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. This research is supported in part by the MD Anderson Cancer Center support grant P30 CA016672., (Copyright © 2020 Elsevier Inc. All rights reserved.)

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an orphan hematologic malignancy with poor outcomes. Tagraxofusp (SL-401) was the first drug approved specifically for patients with BPDCN, in 2018. Additional therapeutic strategies are still needed to improve survival and minimize treatment-related toxicity. This article outlines novel targeted approaches that are in preclinical or clinical development for BPDCN. Although there is no known targetable genetic abnormality that defines BPDCN, data from functional testing of primary tumors, gene expression analyses, and adaptation of targeted drug approaches from other cancers to BPDCNs harboring specific mutations have nominated several promising strategies., Competing Interests: Disclosure A.A. Lane has received research support from AbbVie and Stemline Therapeutics and consulting fees from N-of-One/Qiagen., (Copyright © 2020 Elsevier Inc. All rights reserved.)