Hematopoietic Cell Transplantation for Juvenile Myelomonocytic Leukemia

Charlotte M. Niemeyer, Franco Locatelli

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

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
Pages554-565
Number of pages12
Volume1-2
ISBN (Electronic)9781118416426
ISBN (Print)9781118416006
DOIs
Publication statusPublished - Jan 1 2016

    Fingerprint

Keywords

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

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Niemeyer, C. M., & Locatelli, F. (2016). Hematopoietic Cell Transplantation for Juvenile Myelomonocytic Leukemia. In Thomas' Hematopoietic Cell Transplantation: Fifth Edition (Vol. 1-2, pp. 554-565). Wiley Blackwell. https://doi.org/10.1002/9781118416426.ch49