Serial investigation of PTPN11 mutation in nonhematopoietic tissues in a patient with juvenile myelomonocytic leukemia who was treated with unrelated cord blood transplantation

Int J Hematol. 2015 Dec;102(6):719-22. doi: 10.1007/s12185-015-1877-y. Epub 2015 Oct 6.

Abstract

After allogeneic stem-cell transplantation, nonhematopoietic tissues contain donor-derived cells; however, whether cells from malignant hematological disease can also be found in nonhematopoietic tissues is unclear. This report describes a juvenile myelomonocytic leukemia (JMML) case with a typical PTPN11 mutation (p.E76K) at different allele frequencies in the bone marrow mononuclear cells, buccal smear cells, and fingernails at diagnosis, which was suggestive of PTPN11 somatic mosaicism; however, the PTPN11 mutation in the buccal smear cells and fingernails was lost after unrelated cord blood transplantation. These results suggest that JMML-derived cells may migrate into and reside in nonhematopoietic tissues and furthermore that these cells can be eradicated by cord blood transplantation.

Keywords: Cord blood transplantation; Juvenile myelomonocytic leukemia; Nonhematological tissue; PTPN11; Somatic mosaicism.

Publication types

  • Case Reports

MeSH terms

  • Bone Marrow Cells
  • Cord Blood Stem Cell Transplantation*
  • Gene Frequency
  • Humans
  • Infant
  • Leukemia, Myelomonocytic, Juvenile / genetics*
  • Leukemia, Myelomonocytic, Juvenile / pathology
  • Leukemia, Myelomonocytic, Juvenile / surgery*
  • Male
  • Mosaicism
  • Mouth Mucosa / cytology
  • Mutation*
  • Nails
  • Protein Tyrosine Phosphatase, Non-Receptor Type 1 / genetics*

Substances

  • PTPN1 protein, human
  • Protein Tyrosine Phosphatase, Non-Receptor Type 1