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  • 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
  • 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 (en)
Title
  • Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study
  • Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study (en)
skos:prefLabel
  • Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study
  • Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study (en)
skos:notation
  • RIV/00064203:_____/11:7013!RIV12-MZ0-00064203
http://linked.open...avai/riv/aktivita
http://linked.open...avai/riv/aktivity
  • I
http://linked.open...iv/cisloPeriodika
  • 3
http://linked.open...vai/riv/dodaniDat
http://linked.open...aciTvurceVysledku
http://linked.open.../riv/druhVysledku
http://linked.open...iv/duvernostUdaju
http://linked.open...titaPredkladatele
http://linked.open...dnocenehoVysledku
  • 201874
http://linked.open...ai/riv/idVysledku
  • RIV/00064203:_____/11:7013
http://linked.open...riv/jazykVysledku
http://linked.open.../riv/klicovaSlova
  • myelodysplastic syndrome; stem cell transplantation; children; acute myeloid-leukemia; juvenile myelomonocytic leukemia; bone-marrow-transplantation; versus-host-disease; childhood; graft; classification; experience; survival; therapy (en)
http://linked.open.../riv/klicoveSlovo
http://linked.open...odStatuVydavatele
  • GB - Spojené království Velké Británie a Severního Irska
http://linked.open...ontrolniKodProRIV
  • [7A129A2A4824]
http://linked.open...i/riv/nazevZdroje
  • Leukemia
http://linked.open...in/vavai/riv/obor
http://linked.open...ichTvurcuVysledku
http://linked.open...cetTvurcuVysledku
http://linked.open...UplatneniVysledku
http://linked.open...v/svazekPeriodika
  • 25
http://linked.open...iv/tvurceVysledku
  • Bergstraesser, E.
  • Locatelli, F.
  • Niemeyer, CM
  • Nollke, P.
  • Sedláček, Petr
  • Starý, Jan
  • Strahm, B.
  • Trebo, M.
  • Wojcik, D.
  • Zecca, M.
  • van den Heuvel-Eibrink, MM
  • Bierings, M.
  • Bordon, V.
  • Chybicka, A.
  • Furlan, I.
  • Korthof, ET
  • Moerloose, BD
  • O\'Marcaigh, A.
  • Peters, C.
  • Schmugge, M.
  • de Heredia, CD
http://linked.open...ain/vavai/riv/wos
  • 000288159500008
issn
  • 0887-6924
number of pages
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