Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study
| Autor: | Strahm, B., Nollke, P., Zecca, M., Korthof, E.T., Bierings, M., Furlan, I., Sedlacek, P., Chybicka, A., Schmugge, M., Bordon, V., Peters, C., O'Marcaigh, A., Heredia, C.D. de, Bergstraesser, E., Moerloose, B.D., Heuvel-Eibrink, M.M. van den, Stary, J., Trebo, M., Wojcik, D., Niemeyer, C.M., Locatelli, F., EWOG-MDS Study Grp |
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| Přispěvatelé: | University of Zurich, Strahm, B, Pediatrics |
| Jazyk: | angličtina |
| Rok vydání: | 2011 |
| Předmět: |
Oncology
Melphalan Adult Male Cancer Research medicine.medical_specialty Cyclophosphamide Adolescent medicine.medical_treatment 2720 Hematology Graft vs Host Disease 610 Medicine & health children myelodysplastic syndrome stem cell transplantation Hematopoietic stem cell transplantation Disease-Free Survival Recurrence hemic and lymphatic diseases Internal medicine medicine Humans 1306 Cancer Research Cumulative incidence Child Preparative Regimen Juvenile myelomonocytic leukemia business.industry Hematopoietic Stem Cell Transplantation Infant Hematology medicine.disease Surgery Leukemia surgical procedures operative 10036 Medical Clinic Child Preschool Myelodysplastic Syndromes 2730 Oncology Female business Busulfan medicine.drug |
| Zdroj: | Leukemia, 25(3), 455-462 Leukemia, 25, 455-462. Nature Publishing Group |
| ISSN: | 0887-6924 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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