Makishima Hideki
Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University.
Rinsho Ketsueki. 2019;60(6):600-609. doi: 10.11406/rinketsu.60.600.
Myelodysplastic syndromes (MDS) and myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are heterogeneous myeloid neoplasms that frequently evolve into secondary acute myeloid leukemia (sAML). Recent progress in next-generation sequencing technologies has allowed us to discover frequent mutations throughout the coding regions of MDS, MDS/MPN, and sAML, subsequently providing information on more than 60 driver genes in these diseases. As shown by many study groups recently, such driver mutations are acquired in a gene-specific fashion. DDX41 and SAMD9/SAMD9L mutations are observed in germline cells long before MDS presentation. In blood samples from healthy elderly individuals, somatic DNMT3A, TET2, and ASXL1 mutations are detected as age-related clonal hematopoiesis and supposed to be a risk factor for hematological neoplasms. Recent reports on MDS have shown that mutations in genes such as NRAS and FLT3, designated as Type I genes, were significantly associated with leukemic evolution. Another type (Type II) of genes, including RUNX1 and GATA2, has been shown to be related to the progression from low-risk to high-risk MDS. These driver mutations are significantly concomitant during disease progression. Overall, various types of driver mutations are sequentially acquired in MDS, accounting for the heterogeneity of these disorders.
骨髓增生异常综合征(MDS)和骨髓增生异常/骨髓增殖性肿瘤(MDS/MPN)是异质性髓系肿瘤,常进展为继发性急性髓系白血病(sAML)。新一代测序技术的最新进展使我们能够在MDS、MDS/MPN和sAML的编码区域发现频繁的突变,随后提供了有关这些疾病中60多个驱动基因的信息。正如许多研究小组最近所表明的,此类驱动突变是以基因特异性方式获得的。在MDS出现之前很久就在生殖细胞中观察到DDX41和SAMD9/SAMD9L突变。在健康老年人的血液样本中,体细胞DNMT3A、TET2和ASXL1突变被检测为与年龄相关的克隆性造血,被认为是血液系统肿瘤的危险因素。最近关于MDS的报告表明,NRAS和FLT3等基因(称为I型基因)的突变与白血病进展显著相关。另一类(II型)基因,包括RUNX1和GATA2,已被证明与MDS从低危向高危进展有关。这些驱动突变在疾病进展过程中显著伴随出现。总体而言,MDS中依次获得各种类型的驱动突变,这解释了这些疾病的异质性。