TY - JOUR
T1 - 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
AU - Guardiola, Philippe
AU - Kurre, Peter
AU - Vlad, Amalia
AU - Cayuela, Jean Michel
AU - Espérou, Hélène
AU - Devergie, Agnès
AU - Ribaud, Patricia
AU - Socié, Gérard
AU - Richard, Patrice
AU - Traineau, Richard
AU - Storb, Rainer
AU - Gluckman, Eliane
PY - 2003/9
Y1 - 2003/9
N2 - 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.
AB - 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.
KW - Acute leukaemia
KW - Allogeneic stem cell transplantation
KW - Fanconi anaemia
KW - Graft-versus-leukaemia effect
KW - Myelodysplasia
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U2 - 10.1046/j.1365-2141.2003.04503.x
DO - 10.1046/j.1365-2141.2003.04503.x
M3 - Article
C2 - 12930393
AN - SCOPUS:0042913496
SN - 0007-1048
VL - 122
SP - 806
EP - 809
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 5
ER -