Hematopoietic Cell Transplantation for Juvenile Myelomonocytic Leukemia

Charlotte M. Niemeyer, Franco Locatelli

Research output: Chapter in Book/Report/Conference proceedingChapter


Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative disorder of early childhood; it is characterized by hepatosplenomegaly and organ infiltration due to excessive proliferation of cells of the monocytic and granulocytic lineages. Approximately 85% of JMML patients harbor in their leukemia cells either somatic or germline mutations in the genes PTPN-11, NRAS, KRAS, NF1, or CBL. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the therapy of choice for the majority of affected children. Available data indicate that transplantation from an HLA-identical sibling or a matched unrelated volunteer can cure more than 50% of patients. A similar proportion of children with JMML, especially when transplanted from donors with limited HLA disparity, can be cured with umbilical cord blood transplantation, which can represent a suitable option for children with JMML lacking either a related or an unrelated HSCT donor.

Original languageEnglish
Title of host publicationThomas' Hematopoietic Cell Transplantation: Fifth Edition
PublisherWiley Blackwell
Number of pages12
ISBN (Electronic)9781118416426
ISBN (Print)9781118416006
Publication statusPublished - Jan 1 2016


  • CBL
  • Juvenile myelomonocytic leukemia
  • Leukemia recurrence
  • Neurofibromatosis type 1
  • PTPN-11
  • RAS pathway
  • Second allograft
  • Splenectomy

ASJC Scopus subject areas

  • Medicine(all)


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