title: Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study.
abstract: We report on the outcome of children with advanced primary myelodysplastic syndrome (MDS) transplanted from an HLA-matched sibling (MSD) or an unrelated donor (UD) following a preparative regimen with busulfan, cyclophosphamide and melphalan. Ninety-seven patients with refractory anemia with excess blasts (RAEB, n=53), RAEB in transformation (RAEB-T, n=29) and myelodysplasia-related acute myeloid leukemia (MDR-AML, n=15) enrolled in the European Working Group of MDS in Childhood (EWOG-MDS) 98 study and given hematopoietic stem cell transplantation (HSCT) were analyzed. Median age at HSCT was 11.1 years (range 1.4-19.0). Thirty-nine children were transplanted from an MSD, whereas 58 were given the allograft from a UD (n=57) or alternative family donor (n=1). Stem cell source was bone marrow (n=69) or peripheral blood (n=28). With a median follow-up of 3.9 years (range 0.1-10.9), the 5-year probability of overall survival is 63%, while the 5-year cumulative incidence of transplantation-related mortality (TRM) and relapse is 21% each. Age at HSCT greater than 12 years, interval between diagnosis and HSCT longer than 4 months, and occurrence of acute or extensive chronic graft-versus-host disease were associated with increased TRM. The risk of relapse increased with more advanced disease. This study indicates that HSCT following a myeloablative preparative regimen offers a high probability of survival for children with advanced MDS.
authors.affiliation: Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, University of Freiburg, Freiburg, Germany. brigitte.strahm@uniklinik-freiburg.de
authors.forename: B
authors.initials: B
authors.lastname: Strahm
authors.forename: P
authors.initials: P
authors.lastname: Nöllke
authors.forename: M
authors.initials: M
authors.lastname: Zecca
authors.forename: E T
authors.initials: ET
authors.lastname: Korthof
authors.forename: M
authors.initials: M
authors.lastname: Bierings
authors.forename: I
authors.initials: I
authors.lastname: Furlan
authors.forename: P
authors.initials: P
authors.lastname: Sedlacek
authors.forename: A
authors.initials: A
authors.lastname: Chybicka
authors.forename: M
authors.initials: M
authors.lastname: Schmugge
authors.forename: V
authors.initials: V
authors.lastname: Bordon
authors.forename: C
authors.initials: C
authors.lastname: Peters
authors.forename: A
authors.initials: A
authors.lastname: O'Marcaigh
authors.forename: C D
authors.initials: CD
authors.lastname: de Heredia
authors.forename: E
authors.initials: E
authors.lastname: Bergstraesser
authors.forename: B D
authors.initials: BD
authors.lastname: Moerloose
authors.forename: M M
authors.initials: MM
authors.lastname: van den Heuvel-Eibrink
authors.forename: J
authors.initials: J
authors.lastname: Starý
authors.forename: M
authors.initials: M
authors.lastname: Trebo
authors.forename: D
authors.initials: D
authors.lastname: Wojcik
authors.forename: C M
authors.initials: CM
authors.lastname: Niemeyer
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality; D006801:Humans; D007223:Infant; D008297:Male; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000601:surgery*; D012008:Recurrence