Galati Domenico, Zanotta Serena, Florio Fabrizia, Mele Sara, De Filippi Rosaria, Pinto Antonio
Hematology-Oncology and Stem-Cell Transplantation Unit, Department of Onco-Hematology and Innovative Diagnostics, Istituto Nazionale Tumori-IRCCS-Fondazione G. Pascale, 80131 Napoli, Italy.
Department of Clinical Medicine and Surgery, Università degli Studi di Napoli Federico II, 80131 Napoli, Italy.
Int J Mol Sci. 2025 Mar 18;26(6):2732. doi: 10.3390/ijms26062732.
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.
母细胞性浆细胞样树突状细胞肿瘤（BPDCN）是一种罕见且侵袭性强的血液系统恶性肿瘤，其特征为CD123和CD303表面抗原的过表达。这些分子标志物在疾病诊断和开发靶向治疗中起着关键作用。BPDCN的传统治疗方案效果有限，这凸显了对新型创新治疗策略的需求。免疫疗法的最新进展，特别是治疗性单克隆抗体、双特异性T细胞衔接器和嵌合抗原受体T细胞（CAR T细胞）疗法，提供了有前景的替代方案。塔格昔单抗（Tagraxofusp）是首个获美国食品药品监督管理局（FDA）批准的靶向CD123的疗法，显著改善了患者预后。此外，新兴的靶向CD303策略具有进一步取得进展的潜力。尽管有这些突破，但诸如治疗耐药性和毒性等挑战仍然存在。本综述探讨了BPDCN治疗的最新进展，强调了CD123和CD303作为精准医学干预靶点的潜力。靶向免疫疗法的不断发展有望提高患者生存率并重新定义血液系统恶性肿瘤的治疗模式。
