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Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a clinically aggressive hematologic malignancy derived from precursors of dendritic cells and involves most frequently the skin, bone marrow and lymph nodes. Diagnosis depends upon identification of specific tumor markers including CD4, CD56 an...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6759709/ https://www.ncbi.nlm.nih.gov/pubmed/31579499 http://dx.doi.org/10.1177/2040620719874733 |
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author | Economides, Minas P. Konopleva, Marina Pemmaraju, Naveen |
author_facet | Economides, Minas P. Konopleva, Marina Pemmaraju, Naveen |
author_sort | Economides, Minas P. |
collection | PubMed |
description | Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a clinically aggressive hematologic malignancy derived from precursors of dendritic cells and involves most frequently the skin, bone marrow and lymph nodes. Diagnosis depends upon identification of specific tumor markers including CD4, CD56 and CD123. Historically, the median survival has been less than 2 years in most reported series. While for many years, conventional chemotherapy followed by stem cell transplantation was the standard of care, recently tagraxofusp, a cytotoxin directed against CD123, received United States Food and Drug Administration approval specifically for patients with BPDCN. In this review, we will discuss the markers used for diagnosis of BPDCN and focus on the new targeted treatments available. Specifically in BPDCN, tagraxofusp was highly effective with a safety profile found to be acceptable overall, with the noted occurrence of capillary leak syndrome. Future directions in therapy approaches for patients with BPDCN will include the development of other CD123-targeted agents, agents targeting beyond CD123 and investigation of rational combination approaches of CD123-directed therapy with other therapies. |
format | Online Article Text |
id | pubmed-6759709 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | SAGE Publications |
record_format | MEDLINE/PubMed |
spelling | pubmed-67597092019-10-02 Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm Economides, Minas P. Konopleva, Marina Pemmaraju, Naveen Ther Adv Hematol Review Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a clinically aggressive hematologic malignancy derived from precursors of dendritic cells and involves most frequently the skin, bone marrow and lymph nodes. Diagnosis depends upon identification of specific tumor markers including CD4, CD56 and CD123. Historically, the median survival has been less than 2 years in most reported series. While for many years, conventional chemotherapy followed by stem cell transplantation was the standard of care, recently tagraxofusp, a cytotoxin directed against CD123, received United States Food and Drug Administration approval specifically for patients with BPDCN. In this review, we will discuss the markers used for diagnosis of BPDCN and focus on the new targeted treatments available. Specifically in BPDCN, tagraxofusp was highly effective with a safety profile found to be acceptable overall, with the noted occurrence of capillary leak syndrome. Future directions in therapy approaches for patients with BPDCN will include the development of other CD123-targeted agents, agents targeting beyond CD123 and investigation of rational combination approaches of CD123-directed therapy with other therapies. SAGE Publications 2019-09-23 /pmc/articles/PMC6759709/ /pubmed/31579499 http://dx.doi.org/10.1177/2040620719874733 Text en © The Author(s), 2019 http://www.creativecommons.org/licenses/by-nc/4.0/ This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (http://www.creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage). |
spellingShingle | Review Economides, Minas P. Konopleva, Marina Pemmaraju, Naveen Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm |
title | Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm |
title_full | Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm |
title_fullStr | Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm |
title_full_unstemmed | Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm |
title_short | Recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm |
title_sort | recent developments in the treatment of blastic plasmacytoid dendritic cell neoplasm |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6759709/ https://www.ncbi.nlm.nih.gov/pubmed/31579499 http://dx.doi.org/10.1177/2040620719874733 |
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