Bôle-Richard Elodie, Pemmaraju Naveen, Caël Blandine, Daguindau Etienne, Lane Andrew A
INSERM, EFS BFC, UMR1098, RIGHT, University of Bourgogne Franche-Comté, Interactions Greffon-Hôte-Tumeur/Ingénierie Cellulaire et Génique, F-25000 Besancon, France.
Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Cancers (Basel). 2022 May 3;14(9):2287. doi: 10.3390/cancers14092287.
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive leukemia derived from plasmacytoid dendritic cells (pDCs). It is associated with a remarkably poor prognosis and unmet need for better therapies. Recently, the first-in-class CD123-targeting therapy, tagraxofusp, was approved for treatment of BPDCN. Other CD123-targeting strategies are in development, including bispecific antibodies and combination approaches with tagraxofusp and other novel agents. In other blood cancers, adoptive T-cell therapy using chimeric antigen receptor (CAR)-modified T cells represents a promising new avenue in immunotherapy, showing durable remissions in some relapsed hematologic malignancies. Here, we report on novel and innovative therapies in development to target surface molecules in BPDCN currently in clinical trials or in preclinical stages. We also discuss new cell surface targets that may have implications for future BPDCN treatment.
母细胞样浆细胞样树突状细胞肿瘤（BPDCN）是一种源自浆细胞样树突状细胞（pDCs）的罕见且侵袭性白血病。它与极差的预后相关，并且对更好治疗方法的需求尚未得到满足。最近，首款靶向CD123的疗法——他格拉索夫（tagraxofusp）被批准用于治疗BPDCN。其他靶向CD123的策略正在研发中，包括双特异性抗体以及他格拉索夫与其他新型药物的联合应用方法。在其他血液癌症中，使用嵌合抗原受体（CAR）修饰的T细胞进行过继性T细胞疗法是免疫治疗中一条有前景的新途径，在一些复发的血液系统恶性肿瘤中显示出持久缓解。在此，我们报告目前处于临床试验或临床前阶段的针对BPDCN中表面分子的新型和创新疗法。我们还讨论了可能对未来BPDCN治疗有影响的新细胞表面靶点。
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