Subgroup-specific gene expression profiles and mixed epistasis in chronic lymphocytic leukemia

Haematologica. 2023 Oct 1;108(10):2664-2676. doi: 10.3324/haematol.2022.281869.

Abstract

Understanding the molecular and phenotypic heterogeneity of cancer is a prerequisite for effective treatment. For chronic lymphocytic leukemia (CLL), recurrent genetic driver events have been extensively cataloged, but this does not suffice to explain the disease's diverse course. Here, we performed RNA sequencing on 184 CLL patient samples. Unsupervised analysis revealed two major, orthogonal axes of gene expression variation: the first one represented the mutational status of the immunoglobulin heavy variable (IGHV) genes, and concomitantly, the three-group stratification of CLL by global DNA methylation. The second axis aligned with trisomy 12 status and affected chemokine, MAPK and mTOR signaling. We discovered non-additive effects (epistasis) of IGHV mutation status and trisomy 12 on multiple phenotypes, including the expression of 893 genes. Multiple types of epistasis were observed, including synergy, buffering, suppression and inversion, suggesting that molecular understanding of disease heterogeneity requires studying such genetic events not only individually but in combination. We detected strong differentially expressed gene signatures associated with major gene mutations and copy number aberrations including SF3B1, BRAF and TP53, as well as del(17)(p13), del(13)(q14) and del(11)(q22.3) beyond dosage effect. Our study reveals previously underappreciated gene expression signatures for the major molecular subtypes in CLL and the presence of epistasis between them.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Epistasis, Genetic
  • Humans
  • Leukemia, Lymphocytic, Chronic, B-Cell* / genetics
  • Mutation
  • Prognosis
  • Transcriptome
  • Trisomy

Grants and funding

Funding: The work was supported by the European Union (Horizon 2020 project SOUND under grant agreement number 633974) and the German Federal Ministry of Education and Research (TRANSCAN project GCH-CLL 143 under grant agreement number 01KT1610 and CompLS project MOFA under grant agreement number 031L0171A). TZ was supported by the UZH Clinical Research Priority Program “Next Generation Drug Response Profiling for Personalized Cancer Care”, the Swiss Cancer League (KFS-4439-02-2018), The LOOP Zurich (INTERCEPT) and the Monique-Dornonville-dela-Cour Stiftung. RR received funding from the Swedish Cancer Society, the Swedish Research Council, the Knut and Alice Wallenberg Foundation, and Radiumhemmets Forskningsfonder, Stockholm.