Koya Junji, Kogure Yasunori, Kataoka Keisuke
Division of Hematology, Department of Medicine, Keio University School of Medicine.
Division of Molecular Oncology, National Cancer Center Research Institute.
Rinsho Ketsueki. 2024;65(9):1019-1024. doi: 10.11406/rinketsu.65.1019.
Adult T-cell leukemia/lymphoma (ATLL) is an aggressive peripheral T-cell malignancy caused by human T-cell leukemia virus type-1 (HTLV-1) infection. Genetic alterations are thought to contribute to the pathogenesis of ATLL alongside HTLV-1 products such as Tax and HBZ. Several large-scale genetic analyses have delineated the entire landscape of somatic alterations in ATLL, which is characterized by frequent alterations in T-cell receptor/NF-κB pathways and immune-related molecules. Notably, up to one-fourth of ATLL patients harbor structural variations disrupting the 3'-UTR of the PD-L1 gene, which facilitate escape of tumor cells from anti-tumor immunity. Among these alterations, PRKCB and IRF4 mutations, PD-L1 amplification, and CDKN2A deletion are associated with poor prognosis in ATLL. More recently, several single-cell transcriptome and immune repertoire analyses have revealed phenotypic features of premalignant cells and tumor heterogeneity as well as virus- and tumor-related changes of the non-malignant hematopoietic pool in ATLL. Here we summarize the current understanding of the molecular pathogenesis of ATLL, focusing on recent progress made by genetic, epigenetic, and single-cell analyses. These findings not only provide a deeper understanding of the molecular pathobiology of ATLL, but also have significant implications for diagnostic and therapeutic strategies.
成人T细胞白血病/淋巴瘤(ATLL)是一种由人类T细胞白血病病毒1型(HTLV-1)感染引起的侵袭性外周T细胞恶性肿瘤。人们认为,除了Tax和HBZ等HTLV-1产物外,基因改变也有助于ATLL的发病机制。几项大规模基因分析描绘了ATLL体细胞改变的全貌,其特征是T细胞受体/NF-κB途径和免疫相关分子频繁改变。值得注意的是,高达四分之一的ATLL患者存在破坏PD-L1基因3'-UTR的结构变异,这有助于肿瘤细胞逃避免疫抗肿瘤作用。在这些改变中,PRKCB和IRF4突变、PD-L1扩增以及CDKN2A缺失与ATLL的不良预后相关。最近,几项单细胞转录组和免疫组库分析揭示了癌前细胞的表型特征和肿瘤异质性,以及ATLL中非恶性造血池的病毒和肿瘤相关变化。在此,我们总结了目前对ATLL分子发病机制的认识,重点关注基因、表观遗传和单细胞分析取得的最新进展。这些发现不仅能让我们更深入地了解ATLL的分子病理生物学,也对诊断和治疗策略具有重要意义。