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急性淋巴细胞白血病的新型生物标志物和治疗策略:最新进展。

New biomarkers and therapeutic strategies in acute lymphoblastic leukemias: Recent advances.

机构信息

Department of Medical Sciences, University of Ferrara, Ferrara, Italy.

Department of Morphology, Surgery and Experimental Medicine, University of Ferrara, Ferrara, Italy.

出版信息

Hematol Oncol. 2020 Feb;38(1):22-33. doi: 10.1002/hon.2678. Epub 2019 Oct 21.

Abstract

Acute lymphoblastic leukemia (ALL) represents a heterogeneous group of hematologic malignancies, and it is normally characterized by an aberrant proliferation of immature lymphoid cells. Moreover, dysregulation of multiple signaling pathways that normally regulate cellular transcription, growth, translation, and proliferation is frequently encountered in this malignancy. ALL is the most frequent tumor in childhood, and adult ALL patients still correlate with poor survival. This review focuses on modern therapies in ALL that move beyond standard chemotherapy, with a particular emphasis on immunotherapeutic approaches as new treatment strategies. Bi-specific T-cell Engagers (BiTE) antibodies, the chimeric antigen receptor (CAR)-T cells, or CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats [CRISPR]-associated nuclease 9) represent other new innovative approaches for this disease. Target and tailored therapy could make the difference in previously untreatable cases, i.e., precision and personalized medicine. Clinical trials will help to select the most efficient novel therapies in ALL management and to integrate them with existing treatments to achieve durable cures.

摘要

急性淋巴细胞白血病(ALL)是一组异质性血液恶性肿瘤,其特征通常是幼稚淋巴细胞的异常增殖。此外,在这种恶性肿瘤中,通常调节细胞转录、生长、翻译和增殖的多个信号通路经常失调。ALL 是儿童中最常见的肿瘤,成人 ALL 患者的生存率仍然较差。本综述重点介绍了超越标准化疗的 ALL 现代治疗方法,特别强调了免疫治疗方法作为新的治疗策略。双特异性 T 细胞衔接器(BiTE)抗体、嵌合抗原受体(CAR)-T 细胞或 CRISPR-Cas9(成簇规律间隔短回文重复 [CRISPR]-相关核酸酶 9)代表了该疾病的其他新的创新方法。针对特定靶点的靶向治疗可能会改变以前无法治疗的病例,即精准和个性化医疗。临床试验将有助于选择 ALL 管理中最有效的新型疗法,并将其与现有治疗方法相结合,以实现持久治愈。

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