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

B. 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. Star, M. Trebo, D. Wojcik, C. M. NiemeyerF. Locatelli

Research output: Contribution to journalArticlepeer-review


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, n53), RAEB in transformation (RAEB-T, n29) and myelodysplasia-related acute myeloid leukemia (MDR-AML, n15) 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 (n57) or alternative family donor (n1). Stem cell source was bone marrow (n69) or peripheral blood (n28). 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.

Original languageEnglish
Pages (from-to)455-462
Number of pages8
Issue number3
Publication statusPublished - Mar 2011


  • children
  • myelodysplastic syndrome
  • stem cell transplantation

ASJC Scopus subject areas

  • Hematology
  • Cancer Research
  • Anesthesiology and Pain Medicine


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