The genomic landscape of pediatric myelodysplastic syndromes

Nat Commun. 2017 Nov 16;8(1):1557. doi: 10.1038/s41467-017-01590-5.

Abstract

Myelodysplastic syndromes (MDS) are uncommon in children and have a poor prognosis. In contrast to adult MDS, little is known about the genomic landscape of pediatric MDS. Here, we describe the somatic and germline changes of pediatric MDS using whole exome sequencing, targeted amplicon sequencing, and/or RNA-sequencing of 46 pediatric primary MDS patients. Our data show that, in contrast to adult MDS, Ras/MAPK pathway mutations are common in pediatric MDS (45% of primary cohort), while mutations in RNA splicing genes are rare (2% of primary cohort). Surprisingly, germline variants in SAMD9 or SAMD9L were present in 17% of primary MDS patients, and these variants were routinely lost in the tumor cells by chromosomal deletions (e.g., monosomy 7) or copy number neutral loss of heterozygosity (CN-LOH). Our data confirm that adult and pediatric MDS are separate diseases with disparate mechanisms, and that SAMD9/SAMD9L mutations represent a new class of MDS predisposition.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Animals
  • Cell Line
  • Child
  • Cohort Studies
  • Genetic Predisposition to Disease / genetics*
  • Genomics / methods*
  • HEK293 Cells
  • Humans
  • Intracellular Signaling Peptides and Proteins
  • Loss of Heterozygosity
  • Mice
  • Mutation*
  • Myelodysplastic Syndromes / genetics*
  • Myelodysplastic Syndromes / pathology
  • Proteins / genetics
  • Survival Analysis
  • Tumor Suppressor Proteins / genetics

Substances

  • Intracellular Signaling Peptides and Proteins
  • Proteins
  • SAMD9 protein, human
  • SAMD9L protein, human
  • Tumor Suppressor Proteins