title: 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.
abstract: 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.
authors.affiliation: Bone Marrow Transplant Unit, Hôpital Saint Louis, Paris, France.
authors.forename: Philippe
authors.initials: P
authors.lastname: Guardiola
authors.forename: Peter
authors.initials: P
authors.lastname: Kurre
authors.forename: Amalia
authors.initials: A
authors.lastname: Vlad
authors.forename: Jean-Michel
authors.initials: JM
authors.lastname: Cayuela
authors.forename: Hélène
authors.initials: H
authors.lastname: Espérou
authors.forename: Agnès
authors.initials: A
authors.lastname: Devergie
authors.forename: Patricia
authors.initials: P
authors.lastname: Ribaud
authors.forename: Gérard
authors.initials: G
authors.lastname: Socié
authors.forename: Patrice
authors.initials: P
authors.lastname: Richard
authors.forename: Richard
authors.initials: R
authors.lastname: Traineau
authors.forename: Rainer
authors.initials: R
authors.lastname: Storb
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
mesh_terms: D000208:Acute Disease; D000328:Adult; D002648:Child; D005199:Fanconi Anemia / Q000601:surgery*; D017809:Fatal Outcome; D005260:Female; D020544:Graft vs Leukemia Effect; D006801:Humans; D007951:Leukemia, Myeloid / Q000601:surgery*; D009190:Myelodysplastic Syndromes / Q000601:surgery*; D012086:Reoperation; D033581:Stem Cell Transplantation; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous