Value of allogeneic versus autologous stem cell transplantation and chemotherapy in patients with myelodysplastic syndromes and secondary acute myeloid leukemia. Final results of a prospective randomized European Intergroup Trial

Theo M. de Witte*, Anne Hagemeijer, Stefan Suciu, Amin Belhabri, Michel Delforge, Guido Kobbe, Dominik Selleslag, Harry C. Schouten, Augustin Ferrant, Harald Biersack, Sergio Amadori, Petra Muus, Joop H. Jansen, Eva Hellstrom-Lindberg, Tibor Kovacsovics, Pierre Wijermans, Gert Jan Ossenkoppele, Alois Gratwohl, Jean-Pierre Marie, Roel Willemze

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

54 Citations (Web of Science)


Background Allogeneic stem cell transplantation is usually considered the only curative treatment option for patients with advanced or transformed myelodysplastic syndromes in complete remission, but post-remission chemotherapy and autologous stem cell transplantation are potential alternatives, especially in patients over 45 years old. Design and Methods We evaluated, after intensive anti-leukemic remission-induction chemotherapy, the impact of the availability of an HLA-identical sibling donor on an intention-to treat basis. Additionally, all patients without a sibling donor in complete remission after the first consolidation course were randomized to either autologous peripheral blood stem cell transplantation or a second consolidation course consisting of high-dose cytarabine. Results The 4-year survival of the 341 evaluable patients was 28%. After achieving complete remission, the 4-year survival rates of patients under 55 years old with or without a donor were 54% and 41%, respectively, with an adjusted hazard ratio of 0.81 (95% confidence interval [95% CI], 0.49-1.35) for survival and of 0.67 (95% CI, 0.42-1.06) for disease-free survival. In patients with intermediate/high risk cytogenetic abnormalities the hazard ratio in multivariate analysis was 0.58 (99% CI, 0.22-1.50) (P=0.14) for survival and 0.46(99% CI, 0.22-1.50) for disease-free survival (P=0.03). In contrast, in patients with low risk cytogenetic characteristics the hazard ratio for survival was 1.17 (99% CI, 0.40-3.42) and that for disease-free survival was 1.02 (99% CI, 0.40-2.56). The 4-year survival of the 65 patients randomized to autologous peripheral blood stem cell transplantation or a second consolidation course of high-dose cytarabine was 37% and 27%, respectively. The hazard ratio in multivariate analysis was 1.22 (95% CI, 0.652.27) for survival and 1.02 (95% CI, 0.56-1.85) for disease-free survival. Conclusions Patients with a donor and candidates for allogeneic stem cell transplantation in first complete remission may have a better disease-free survival than those without a donor in case of myelodysplastic syndromes with intermediate/high-risk cytogenetics. Autologous peripheral blood stem cell transplantation does not provide longer survival than intensive chemotherapy.
Original languageEnglish
Pages (from-to)1754-1761
JournalHaematologica-the Hematology Journal
Issue number10
Publication statusPublished - Oct 2010


  • myelodysplastic syndromes
  • secondary acute myeloid leukemia
  • cytogenetic characteristics
  • allogeneic stem cell transplantation
  • autologous stem cell transplantation
  • intensive chemotherapy

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