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靶向 CD123 和 CD303 的免疫治疗:治疗母细胞性浆细胞样树突状细胞肿瘤的新前沿

英文原题:Immunotherapies Targeting CD123 and CD303: A New Frontier in Treating Blastic Plasmacytoid Dendritic Cell Neoplasm.

PubMed 2025/03/18(内容时间) Int J Mol Sci Q1 · IF 5.6(JCR 2025)

研究概要

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的血液系统恶性肿瘤,其特征是 CD123 和 CD303 表面抗原的过表达。

中文摘要

母细胞性浆细胞样树突细胞肿瘤(BPDCN)是一种罕见且侵袭性的血液系统恶性肿瘤,特征为 CD123 和 CD303 表面抗原过表达。这些分子标志物对于疾病诊断和靶向治疗开发至关重要。传统 BPDCN 治疗方案疗效有限,凸显开发新型创新治疗策略的必要性。免疫治疗的近期进展,尤其是治疗性单克隆抗体、双特异性 T 细胞衔接器和 CAR-T 细胞疗法,提供了有前景的替代选择。首个获 FDA 批准的 CD123 靶向疗法 tagraxofusp 显著改善患者结局。此外,新兴的 CD303 靶向策略也有望进一步推动治疗进展。尽管取得这些突破,治疗耐药和毒性等挑战仍然存在。本综述介绍 BPDCN 治疗最新进展,强调 CD123 和 CD303 作为精准医学干预靶点的潜力。靶向免疫疗法持续演进,有望改善患者生存并重新定义血液系统恶性肿瘤治疗范式。

展开英文摘要原文

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.

论文信息

作者
Galati D、Zanotta S、Florio F、Mele S、De Filippi R、Pinto A
单位
Hematology-Oncology and Stem-Cell Transplantation Unit, Department of Onco-Hematology and Innovative Diagnostics, Istituto Nazionale Tumori-IRCCS-Fondazione G. Pascale, 80131 Napoli, Italy.Italy
文献类型
综述
期刊
International journal of molecular sciences2025 Mar 18
原文标识
PubMed 40141368 · DOI 10.3390/ijms26062732