Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study.

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.
AuthorsB Strahm, P Nöllke, M Zecca, E T Korthof, M Bierings, I Furlan, P Sedlacek, A Chybicka, M Schmugge, V Bordon, C Peters, A O'Marcaigh, C D de Heredia, E Bergstraesser, B D Moerloose, M M van den Heuvel-Eibrink, J Starý, M Trebo, D Wojcik, C M Niemeyer, F Locatelli,
JournalLeukemia (Leukemia) Vol. 25 Issue 3 Pg. 455-62 (Mar 2011) ISSN: 1476-5551 [Electronic] England
PMID21212791 (Publication Type: Journal Article)
  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Female
  • Graft vs Host Disease (mortality)
  • Hematopoietic Stem Cell Transplantation (mortality)
  • Humans
  • Infant
  • Male
  • Myelodysplastic Syndromes (mortality, surgery)
  • Recurrence

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