title: Stem cell transplantation for aplastic anemia and myelodysplastic syndrome.
abstract: Stem cell transplantation (SCT) from a histocompatible sibling is treatment of choice for severe aplastic anemia. Survival rates have been reported to be as high as 90% for children. Immunosuppressive therapy (IST) is employed in patients who are not candidates for SCT due to donor unavailability. The addition of cyclosporin A to antilymphocyte globulin has improved the response rate to 70-80%, and survival at 5 years among responders is about 90%. In all, 30% of patients treated by IST suffer from relapse, but long-term prognosis does not appear to be affected by this complication. Juvenile myelomonocytic leukemia (JMML) shares both myelodysplastic and myeloproliferative features. Survival (10-year) of patients with JMML without SCT is only 6%. Children with JMML should be transplanted early in the course of their disease. Conditioning regimen composed of three alkylating agents, busulfan, cyclophosphamide and melphalan has been favored by the EWOG-MDS and EBMT-Pediatric WP in the second half of the 1990s. SCT using this conditioning regimen is capable of curing approximately 50% of patients with JMML. More than 70% of patients with refractory cytopenia and more than 50% of children with advanced MDS are cured of by the early performed allogeneic SCT.
authors.affiliation: Department of Pediatric Hematology and Oncology, University Hospital Motol, Prague, Czech Republic. jan.stary@lfmotol.cuni.cz
authors.forename: J
authors.initials: J
authors.lastname: Starý
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: C M
authors.initials: CM
authors.lastname: Niemeyer
mesh_terms: D000741:Anemia, Aplastic / Q000628:therapy*; D002648:Child; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D006801:Humans; D007951:Leukemia, Myeloid / Q000628:therapy*; D008297:Male; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D033581:Stem Cell Transplantation; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D014184:Transplantation, Homologous