title: Juvenile myelomonocytic leukemia.
abstract: Juvenile myelomonocytic leukemia is an aggressive neoplasia of early childhood. Only allogeneic stem cell transplantation (SCT) offers a long-term cure. In the absence of an HLA-matched family donor, early SCT from an unrelated donor will be the treatment of choice for most children. With clear evidence of a graft-versus-leukemia effect and a high post-transplant relapse rate, outcome of SCT will depend, in part, on the management of immunosuppression during the procedure. The impact of pretransplant cytoreductive treatment, such as intensive chemotherapy, splenectomy, or 13-cis retinoic acid, is unclear. Hypersensitivity for granulocyte-macrophage colony-stimulating factor and pathologic activation of the Ras/MAPK pathway play an important role in the pathophysiology of juvenile myelomonocytic leukemia and will provide the opportunity for several novel therapy approaches.
authors.affiliation: Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Mathildenstrasse 1, 79106 Freiburg, Germany. niemeyer@kikli.ukl.uni-freiburg.de
authors.forename: Charlotte Marie
authors.initials: CM
authors.lastname: Niemeyer
authors.forename: Christian
authors.initials: C
authors.lastname: Kratz
mesh_terms: D002675:Child, Preschool; D003131:Combined Modality Therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D015479:Leukemia, Myelomonocytic, Acute / Q000473:pathology / Q000628:therapy; D015477:Leukemia, Myelomonocytic, Chronic / Q000473:pathology / Q000628:therapy