Gerstung Moritz, Pellagatti Andrea, Malcovati Luca, Giagounidis Aristoteles, Porta Matteo G Della, Jädersten Martin, Dolatshad Hamid, Verma Amit, Cross Nicholas C P, Vyas Paresh, Killick Sally, Hellström-Lindberg Eva, Cazzola Mario, Papaemmanuil Elli, Campbell Peter J, Boultwood Jacqueline
Wellcome Trust Sanger Institute, Hinxton CB10 1SA, UK.
LLR Molecular Haematology Unit, NDCLS, RDM, University of Oxford, Oxford OX3 9DU, UK.
Nat Commun. 2015 Jan 9;6:5901. doi: 10.1038/ncomms6901.
Cancer is a genetic disease, but two patients rarely have identical genotypes. Similarly, patients differ in their clinicopathological parameters, but how genotypic and phenotypic heterogeneity are interconnected is not well understood. Here we build statistical models to disentangle the effect of 12 recurrently mutated genes and 4 cytogenetic alterations on gene expression, diagnostic clinical variables and outcome in 124 patients with myelodysplastic syndromes. Overall, one or more genetic lesions correlate with expression levels of ~20% of all genes, explaining 20-65% of observed expression variability. Differential expression patterns vary between mutations and reflect the underlying biology, such as aberrant polycomb repression for ASXL1 and EZH2 mutations or perturbed gene dosage for copy-number changes. In predicting survival, genomic, transcriptomic and diagnostic clinical variables all have utility, with the largest contribution from the transcriptome. Similar observations are made on the TCGA acute myeloid leukaemia cohort, confirming the general trends reported here.
癌症是一种基因疾病,但两名患者很少具有相同的基因型。同样,患者在临床病理参数上也存在差异,但基因型和表型异质性是如何相互关联的,目前还不太清楚。在这里,我们建立了统计模型,以厘清12个反复突变的基因和4种细胞遗传学改变对124例骨髓增生异常综合征患者的基因表达、诊断临床变量和预后的影响。总体而言,一个或多个基因损伤与所有基因中约20%的表达水平相关,解释了观察到的表达变异性的20%-65%。不同的表达模式因突变而异,并反映了潜在的生物学特性,例如ASXL1和EZH2突变导致的异常多梳抑制,或拷贝数变化导致的基因剂量扰动。在预测生存方面,基因组、转录组和诊断临床变量都具有实用价值,其中转录组的贡献最大。在TCGA急性髓系白血病队列中也有类似的观察结果,证实了本文报道的总体趋势。