Division of Hematology, Mayo Clinic, 200 First St SW, Rochester, MN, 55905, USA.
Curr Hematol Malig Rep. 2018 Aug;13(4):265-274. doi: 10.1007/s11899-018-0455-9.
This article provides an overview of the current knowledge regarding the biology and treatment of T cell acute lymphoblastic leukemia (T-ALL) and highlights the most recent findings in this field over the past 5 years.
Remarkable progress has been made in the genomic landscape of T-ALL over the past few years. The discovery of activating mutations of NOTCH1 and FBXW7 in a majority of patients has been a seminal observation, with several early phase clinical trials currently exploring these as potential therapeutic targets. Characterization of early T cell precursor ALL, incorporation of minimal residual disease assessment into therapeutic protocols, and use of pediatric-intensive regimens along with judicious use of allogeneic HCT have significantly improved risk stratification and treatment outcomes. Improved risk stratification and the use of novel targeted therapies based on recent genomic discoveries are expected to change the therapeutic landscape of T-ALL and hopefully improve the outcomes of this historically poor prognosis disease.
本文概述了目前关于 T 细胞急性淋巴细胞白血病（T-ALL）的生物学和治疗的知识，并重点介绍了过去 5 年来该领域的最新发现。
在过去的几年中，T-ALL 的基因组图谱取得了显著的进展。大多数患者中 NOTCH1 和 FBXW7 的激活突变的发现是一个开创性的观察结果，目前有几项早期临床试验正在探索这些作为潜在的治疗靶点。对早期 T 细胞前体 ALL 的特征描述、将微小残留病评估纳入治疗方案，以及使用儿科强化方案和异体造血干细胞移植的合理应用，极大地改善了风险分层和治疗结果。基于最近的基因组发现，改善风险分层和使用新型靶向治疗方法有望改变 T-ALL 的治疗格局，并有望改善这种历史上预后不良疾病的结局。
