Updates in Novel Therapies for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN).
BPDCN
Leukemia
Stem cell transplantation
Tagraxofusp
Journal
Current hematologic malignancy reports
ISSN: 1558-822X
Titre abrégé: Curr Hematol Malig Rep
Pays: United States
ID NLM: 101262565
Informations de publication
Date de publication:
12 2019
12 2019
Historique:
pubmed:
20
12
2019
medline:
18
8
2020
entrez:
20
12
2019
Statut:
ppublish
Résumé
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. 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. 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.
Identifiants
pubmed: 31853773
doi: 10.1007/s11899-019-00556-2
pii: 10.1007/s11899-019-00556-2
doi:
Substances chimiques
Biomarkers, Tumor
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
515-522Subventions
Organisme : NCI NIH HHS
ID : P30 CA016672
Pays : United States
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