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Myelodysplasias

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

Leukemia volume 25, pages 455–462 (2011)Cite this article

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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.

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Authors and Affiliations

  1. Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, University of Freiburg, Freiburg, Germany

    B Strahm, P Nöllke, I Furlan & C M Niemeyer

  2. Department of Pediatrics, Fondazione, IRCCS Policlinico, San Matteo, Italy

    M Zecca

  3. Department of Pediatrics, Division of Stem Cell Transplantation, Leiden University Medical Center, Leiden, The Netherlands

    E T Korthof

  4. Department of Hematology, University Hospital for Children, Utrecht, The Netherlands

    M Bierings

  5. Department of Pediatric Hematology and Oncology, University Hospital Motol, Prague, Czech Republic

    P Sedlacek & J Starý

  6. Department of Pediatric Bone Marrow Transplantation, Oncology and Hematology, Medical University, Wroclaw, Poland

    A Chybicka & D Wojcik

  7. Department of Hematology and Oncology, University Children's Hospital, Zurich, Switzerland

    M Schmugge & E Bergstraesser

  8. Department of Pediatric Hematology and Oncology, Ghent University Hospital, Ghent, Belgium

    V Bordon & B D Moerloose

  9. Department of Pediatrics, St Anna Children's Hospital, Vienna, Austria

    C Peters & M Trebo

  10. Department of Oncology and Hematology, Our Lady's Hospital for Sick Children, Dublin, Ireland

    A O'Marcaigh

  11. Department of Pediatric Oncology and Hematology, Hospital Vall d'Hebron, Barcelona, Spain

    C D de Heredia

  12. Erasmus Medical Center, Rotterdam, The Netherlands

    M M van den Heuvel-Eibrink

  13. Dutch Childhood Oncology Group, The Hague, The Netherlands

    M M van den Heuvel-Eibrink

  14. Department of Pediatric Hematology and Oncology, IRCCS Ospedale Bambino Gesù, University of Pavia, Rome, Italy

    F Locatelli

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Strahm, B., Nöllke, P., Zecca, M. et al. Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study. Leukemia 25, 455–462 (2011). https://doi.org/10.1038/leu.2010.297

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