Tracing the development of acute myeloid leukemia in CBL syndrome

Blood. 2014 Mar 20;123(12):1883-6. doi: 10.1182/blood-2013-10-533844. Epub 2014 Feb 3.

Abstract

We describe the development of acute myeloid leukemia (AML) in an adult with CBL syndrome caused by a heterozygous de novo germline mutation in CBL codon D390. In the AML bone marrow, the mutated CBL allele was homozygous after copy number-neutral loss-of-heterozygosity and amplified through a chromosomal gain; moreover, an inv(16)(p13q22) and, as assessed by whole-exome sequencing, 12 gene mutations (eg, in CAND1, NID2, PTPRT, DOCK6) were additionally acquired. During complete remission of the AML, in the presence of normal blood counts, the hematopoiesis stably maintained the homozygous CBL mutation, which is reminiscent of the situation in children with CBL syndrome and transient juvenile myelomonocytic leukemia. No additional mutations were identified by whole-exome sequencing in granulocytes during complete remission. The study highlights the development of AML in an adult with CBL syndrome and, more generally, in genetically aberrant but clinically inconspicuous hematopoiesis.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Chromosome Aberrations
  • Chromosomes, Human, Pair 11 / genetics
  • Gene Amplification
  • Germ-Line Mutation
  • Hematologic Diseases / complications
  • Hematologic Diseases / genetics
  • Hematopoiesis / genetics
  • Homozygote
  • Humans
  • Leukemia, Myeloid, Acute / etiology
  • Leukemia, Myeloid, Acute / genetics*
  • Loss of Heterozygosity
  • Male
  • Proto-Oncogene Proteins c-cbl / genetics*
  • Spherocytosis, Hereditary / complications
  • Spherocytosis, Hereditary / genetics
  • Syndrome

Substances

  • Proto-Oncogene Proteins c-cbl
  • CBL protein, human