Juvenile myelomonocytic leukemia displays mutations in components of the RAS pathway and the PRC2 network

Nat Genet. 2015 Nov;47(11):1334-40. doi: 10.1038/ng.3420. Epub 2015 Oct 12.

Abstract

Juvenile myelomonocytic leukemia (JMML) is a rare and severe myelodysplastic and myeloproliferative neoplasm of early childhood initiated by germline or somatic RAS-activating mutations. Genetic profiling and whole-exome sequencing of a large JMML cohort (118 and 30 cases, respectively) uncovered additional genetic abnormalities in 56 cases (47%). Somatic events were rare (0.38 events/Mb/case) and restricted to sporadic (49/78; 63%) or neurofibromatosis type 1 (NF1)-associated (8/8; 100%) JMML cases. Multiple concomitant genetic hits targeting the RAS pathway were identified in 13 of 78 cases (17%), disproving the concept of mutually exclusive RAS pathway mutations and defining new pathways activated in JMML involving phosphoinositide 3-kinase (PI3K) and the mTORC2 complex through RAC2 mutation. Furthermore, this study highlights PRC2 loss (26/78; 33% of sporadic JMML cases) that switches the methylation/acetylation status of lysine 27 of histone H3 in JMML cases with altered RAS and PRC2 pathways. Finally, the association between JMML outcome and mutational profile suggests a dose-dependent effect for RAS pathway activation, distinguishing very aggressive JMML rapidly progressing to acute myeloid leukemia.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylation
  • Acute Disease
  • Child
  • Child, Preschool
  • DNA Copy Number Variations
  • Disease Progression
  • Female
  • Gene Regulatory Networks / genetics*
  • Histones / metabolism
  • Humans
  • Infant
  • Leukemia, Myeloid / genetics
  • Leukemia, Myeloid / metabolism
  • Leukemia, Myelomonocytic, Juvenile / genetics*
  • Leukemia, Myelomonocytic, Juvenile / metabolism
  • Male
  • Methylation
  • Microscopy, Confocal
  • Mutation*
  • Phosphatidylinositol 3-Kinases / genetics
  • Phosphatidylinositol 3-Kinases / metabolism
  • Polycomb Repressive Complex 2 / genetics*
  • Polycomb Repressive Complex 2 / metabolism
  • Protein Subunits / genetics
  • Protein Subunits / metabolism
  • Sequence Analysis, DNA / methods
  • Signal Transduction / genetics*
  • Survival Analysis
  • Transcriptome
  • ras Proteins / genetics*
  • ras Proteins / metabolism

Substances

  • Histones
  • Protein Subunits
  • Polycomb Repressive Complex 2
  • Phosphatidylinositol 3-Kinases
  • ras Proteins