title: Juvenile myelomonocytic leukemia.
abstract: Juvenile myelomonocytic leukemia (JMML), a rare myeloid malignancy that occurs in young children, is considered a clonal disease originating in pluripotent stem cells of the hematopoietic system. The pathogenesis of JMML involves disruption of signal transduction through the RAS pathway, with resultant selective hypersensitivity of JMML cells to granulocyte-macrophage colony-stimulating factor. Progress has been made in understanding aspects of the molecular basis of JMML. How these molecular mechanisms may lead to targeted therapeutics and improved outcomes remains to be elucidated. Allogeneic hematopoietic stem cell transplant is the only curative option for children with JMML, and it is fraught with frequent relapse and significant toxicity.
authors.affiliation: Division of Pediatric Hematology/Oncology and Stem Cell Transplantation, Columbia University Medical Center Morgan Stanley Children's Hospital of New York-Presbyterian, 3959 Broadway, CHN-1002, New York, NY 10032, USA. Electronic address: ps2087@columbia.edu.
authors.forename: Prakash
authors.initials: P
authors.lastname: Satwani
authors.affiliation: Division of Pediatric Hematology/Oncology and Stem Cell Transplantation, Columbia University Medical Center Morgan Stanley Children's Hospital of New York-Presbyterian, 3959 Broadway, CHN-1002, New York, NY 10032, USA.
authors.forename: Justine
authors.initials: J
authors.lastname: Kahn
authors.affiliation: Division of Pediatric Allergy, Immunology, and Bone Marrow Transplant, Benioff Children's Hospital, University of California San Francisco, 505 Parnassus Ave., M-659, San Francisco, CA, 94143-1278, USA.
authors.forename: Christopher C
authors.initials: CC
authors.lastname: Dvorak
mesh_terms: D002648:Child; D002675:Child, Preschool; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D054429:Leukemia, Myelomonocytic, Juvenile / Q000150:complications* / Q000628:therapy; D014184:Transplantation, Homologous
keywords: Allogeneic hematopoietic cell transplantation; Children; Leukemia; PTPN11mutation; RAS pathways