TY - JOUR
T1 - Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study
AU - Strahm, B.
AU - Nöllke, P.
AU - Zecca, M.
AU - Korthof, E. T.
AU - Bierings, M.
AU - Furlan, I.
AU - Sedlacek, P.
AU - Chybicka, A.
AU - Schmugge, M.
AU - Bordon, V.
AU - Peters, C.
AU - O'Marcaigh, A.
AU - De Heredia, C. D.
AU - Bergstraesser, E.
AU - Moerloose, B. D.
AU - Van Den Heuvel-Eibrink, M. M.
AU - Star, J.
AU - Trebo, M.
AU - Wojcik, D.
AU - Niemeyer, C. M.
AU - Locatelli, Franco
PY - 2011
Y1 - 2011
N2 - 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. Leukemia (2011) 25, 455-462; doi:10.1038/leu.2010.297; published online 7 January 2011
AB - 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. Leukemia (2011) 25, 455-462; doi:10.1038/leu.2010.297; published online 7 January 2011
KW - myelodysplastic syndrome
KW - children
KW - stem cell transplantation
KW - myelodysplastic syndrome
KW - children
KW - stem cell transplantation
UR - http://hdl.handle.net/10807/249216
U2 - 10.1038/leu.2010.297
DO - 10.1038/leu.2010.297
M3 - Article
SN - 1476-5551
VL - 25
SP - 455
EP - 462
JO - Leukemia
JF - Leukemia
ER -