: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic malignancy characterized by the overexpression of CD123 and CD303 surface antigens. These molecular markers play a crucial role in diagnosing diseases and developing targeted therapies. Traditional treatment options for BPDCN have demonstrated limited effectiveness, highlighting the need for new and innovative therapeutic strategies. Recent advances in immunotherapy, particularly therapeutic monoclonal antibodies, bispecific T-cell engagers, and CAR T-cell therapy, have provided promising alternatives. Tagraxofusp, the first FDA-approved CD123-targeted therapy, has significantly improved patient outcomes. Additionally, emerging CD303-targeting strategies offer the potential for further advancements. Despite these breakthroughs, challenges such as treatment resistance and toxicity remain. This review explores the latest developments in BPDCN treatment, emphasizing the potential of CD123 and CD303 as targets for precision medicine interventions. The ongoing evolution of targeted immunotherapies holds promise for improving patient survival and redefining treatment paradigms in hematologic malignancies.
Immunotherapies Targeting CD123 and CD303: A New Frontier in Treating Blastic Plasmacytoid Dendritic Cell Neoplasm / Galati, D.; Zanotta, S.; Florio, F.; Mele, S.; De Filippi, R.; Pinto, A.. - In: INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. - ISSN 1422-0067. - 26:6(2025), p. 2732. [10.3390/ijms26062732]
Immunotherapies Targeting CD123 and CD303: A New Frontier in Treating Blastic Plasmacytoid Dendritic Cell Neoplasm
De Filippi R.Writing – Review & Editing
;
2025
Abstract
: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic malignancy characterized by the overexpression of CD123 and CD303 surface antigens. These molecular markers play a crucial role in diagnosing diseases and developing targeted therapies. Traditional treatment options for BPDCN have demonstrated limited effectiveness, highlighting the need for new and innovative therapeutic strategies. Recent advances in immunotherapy, particularly therapeutic monoclonal antibodies, bispecific T-cell engagers, and CAR T-cell therapy, have provided promising alternatives. Tagraxofusp, the first FDA-approved CD123-targeted therapy, has significantly improved patient outcomes. Additionally, emerging CD303-targeting strategies offer the potential for further advancements. Despite these breakthroughs, challenges such as treatment resistance and toxicity remain. This review explores the latest developments in BPDCN treatment, emphasizing the potential of CD123 and CD303 as targets for precision medicine interventions. The ongoing evolution of targeted immunotherapies holds promise for improving patient survival and redefining treatment paradigms in hematologic malignancies.| File | Dimensione | Formato | |
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