研究概要
这种多层面的方法为针对这种具有挑战性的血液系统恶性肿瘤实现更有效和更有针对性的治疗干预带来了希望。
中文摘要
母细胞性浆细胞样树突状细胞肿瘤(BPDCN)是一种罕见的侵袭性血液系统恶性肿瘤,起源于浆细胞样树突状细胞前体的恶性转化。该恶性肿瘤进展迅速,复发频繁,总体生存率低,凸显了对有效治疗的迫切需求。然而,由于其罕见性及缺乏标准化方案,BPDCN的诊断和治疗历来具有挑战性。BPDCN被认定为一种独立疾病实体是近年来的事,标准化治疗方案尚未建立。传统上,常规化疗和干细胞移植一直是治疗BPDCN患者的主要方法。免疫表型和分子谱分析的进展已识别出潜在的治疗靶点,推动临床和研究领域向CD123靶向免疫治疗转变。SL-401、IMGN632、CD123嵌合抗原受体(CAR)T细胞和双特异性抗体(BsAb)的持续开发显示出有前景的进展。然而,CD123靶向治疗的治疗效果需要通过创新方法及与其他抗白血病药物的联合治疗来改善。建议探索CD123靶向免疫治疗与阿扎胞苷和维奈克拉的联合方案,以增强抗肿瘤反应并提高BPDCN患者的生存率。总之,这种多层面的方法为针对这一具有挑战性的血液系统恶性肿瘤实现更有效和个体化的治疗干预带来了希望。
展开英文摘要原文
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic cancer originating from the malignant transformation of plasmacytoid dendritic cell precursors. This malignancy progresses rapidly, with frequent relapses and a poor overall survival rate, underscoring the urgent need for effective treatments. However, diagnosing and treating BPDCN have historically been challenging due to its rarity and the lack of standardized approaches. The recognition of BPDCN as a distinct disease entity is recent, and standardized treatment protocols are yet to be established. Traditionally, conventional chemotherapy and stem cell transplantation have been the primary methods for treating BPDCN patients. Advances in immunophenotyping and molecular profiling have identified potential therapeutic targets, leading to a shift toward CD123-targeted immunotherapies in both clinical and research settings. Ongoing developments with SL-401, IMGN632, CD123 chimeric antigen receptor (CAR) T-cells, and bispecific antibodies (BsAb) show promising advancements. However, the therapeutic effectiveness of CD123-targeting treatments needs improvement through innovative approaches and combinations of treatments with other anti-leukemic drugs. The exploration of combinations such as CD123-targeted immunotherapies with azacitidine and venetoclax is suggested to enhance antineoplastic responses and improve survival rates in BPDCN patients. In conclusion, this multifaceted approach offers hope for more effective and tailored therapeutic interventions against this challenging hematologic malignancy.
论文信息
- 作者
- Zanotta S、Galati D、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 sciences2024 Jan 25