Effective graft-versus-leukaemia effect after allogeneic stem cell transplantation using reduced-intensity preparative regimens in Fanconi anaemia patients with myelodysplastic syndrome or acute myeloid leukaemia

Philippe Guardiola, Peter Kurre, Amalia Vlad, Jean Michel Cayuela, Hélène Espérou, Agnès Devergie, Patricia Ribaud, Gérard Socié, Patrice Richard, Richard Traineau, Rainer Storb, Eliane Gluckman

Research output: Contribution to journalArticle

11 Scopus citations


Allogeneic transplantation is the only curative treatment for Fanconi anaemia (FA) patients who develop myeloid malignancies. Dose-intensive preparative regimens, to decrease disease recurrence, lead to unacceptable transplant-related toxicity in FA. We report the outcome of three FA patients with such malignancies who underwent transplantation with reduced-intensity preparative regimens. This approach was well tolerated, even as second transplantations, and resulted in complete leukaemic remissions. However, the graft-versus-leukaemia effect was associated with fatal graft-versus-host disease. Even after transplantation, myeloid malignancies remain associated with a poor outcome in FA. and this argues in favour of early intervention when suitable donors are available.

Original languageEnglish (US)
Pages (from-to)806-809
Number of pages4
JournalBritish Journal of Haematology
Issue number5
Publication statusPublished - Sep 2003
Externally publishedYes



  • Acute leukaemia
  • Allogeneic stem cell transplantation
  • Fanconi anaemia
  • Graft-versus-leukaemia effect
  • Myelodysplasia

ASJC Scopus subject areas

  • Hematology

Cite this