Hematopoietic Cell Transplantation for Juvenile Myelomonocytic Leukemia

Charlotte M. Niemeyer, Franco Locatelli

Risultato della ricerca: Contributo in libroChapter

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.
Lingua originaleEnglish
Titolo della pubblicazione ospiteThomas' Hematopoietic Cell Transplantation: Fifth Edition
Pagine554-565
Numero di pagine12
Volume1-2
DOI
Stato di pubblicazionePubblicato - 2016

Keywords

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

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