Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA; Harvard Medical School, Boston, MA; Broad Institute of MIT and Harvard, Cambridge, MA; Department of Immuno-Oncology, Nuffield Department of Medicine, University of Oxford, Oxford, UK.
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA; Harvard Medical School, Boston, MA; Broad Institute of MIT and Harvard, Cambridge, MA.
Semin Hematol. 2024 Apr;61(2):73-82. doi: 10.1053/j.seminhematol.2024.01.009. Epub 2024 Jan 23.
Clonal expansion of B-cells, from the early stages of monoclonal B-cell lymphocytosis through to chronic lymphocytic leukemia (CLL), and then in some cases to Richter's syndrome (RS) provides a comprehensive model of cancer evolution, notable for the marked morphological transformation and distinct clinical phenotypes. High-throughput sequencing of large cohorts of patients and single-cell studies have generated a molecular map of CLL and more recently, of RS, yielding fundamental insights into these diseases and of clonal evolution. A selection of CLL driver genes have been functionally interrogated to yield novel insights into the biology of CLL. Such findings have the potential to impact patient care through risk stratification, treatment selection and drug discovery. However, this molecular map remains incomplete, with extant questions concerning the origin of the B-cell clone, the role of the TME, inter- and intra-compartmental heterogeneity and of therapeutic resistance mechanisms. Through the application of multi-modal single-cell technologies across tissues, disease states and clinical contexts, these questions can now be addressed with the answers holding great promise of generating translatable knowledge to improve patient care.
B 细胞的克隆扩增,从单克隆 B 细胞淋巴细胞增多症的早期阶段到慢性淋巴细胞白血病(CLL),然后在某些情况下发展为里希特综合征(RS),为癌症进化提供了一个全面的模型,其特征是明显的形态转化和不同的临床表型。对大量患者队列进行高通量测序和单细胞研究已经生成了 CLL 的分子图谱,最近还生成了 RS 的分子图谱,为这些疾病和克隆进化提供了基本的见解。对一些 CLL 驱动基因进行了功能分析,为 CLL 的生物学提供了新的见解。这些发现有可能通过风险分层、治疗选择和药物发现来影响患者的护理。然而,这个分子图谱仍然不完整,存在关于 B 细胞克隆起源、TME 作用、细胞间和细胞内异质性以及治疗耐药机制的问题。通过在组织、疾病状态和临床环境中应用多模态单细胞技术,现在可以解决这些问题,答案有望产生可转化的知识,以改善患者的护理。