title: Excellent outcome of allogeneic bone marrow transplantation for Fanconi anemia using fludarabine-based reduced-intensity conditioning regimen.
abstract: Fanconi anemia (FA) is a disorder characterized by developmental anomalies, bone marrow failure and a predisposition to malignancy. It has recently been shown that hematopoietic stem cell transplantation using fludarabine (FLU)-based reduced-intensity conditioning is an efficient and quite safe therapeutic modality. We retrospectively analyzed the outcome of bone marrow transplantation (BMT) in eight patients with FA performed in two institutes between 2001 and 2011. There were seven females and one male with a median age at diagnosis = 4.5 years (range 2-12 years). The constitutional characteristics associated with FA, such as developmental anomalies, short stature and skin pigmentation, were absent in three of the patients. One patient showed myelodysplastic features at the time of BMT. All patients received BMT using FLU, cyclophosphamide (CY) and rabbit anti-thymocyte globulin (ATG) either from a related donor (n = 4) or an unrelated donor (n = 4). Acute graft-versus-host disease (GVHD) of grade I developed in one patient, while chronic GVHD was not observed in any patient. All patients are alive and achieved hematopoietic recovery at a median follow-up of 72 months (range 4-117 months). BMT using FLU/low-dose CY/ATG -based regimens regardless to the donor is a beneficial therapeutic approach for FA patients.
authors.affiliation: Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, Aichi 466-8550, Japan. ashimada@med.nagoya-u.ac.jp
authors.forename: Akira
authors.initials: A
authors.lastname: Shimada
authors.forename: Yoshiyuki
authors.initials: Y
authors.lastname: Takahashi
authors.forename: Hideki
authors.initials: H
authors.lastname: Muramatsu
authors.forename: Asahito
authors.initials: A
authors.lastname: Hama
authors.forename: Olfat
authors.initials: O
authors.lastname: Ismael
authors.forename: Atsushi
authors.initials: A
authors.lastname: Narita
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Sakaguchi
authors.forename: Sayoko
authors.initials: S
authors.lastname: Doisaki
authors.forename: Nobuhiro
authors.initials: N
authors.lastname: Nishio
authors.forename: Makito
authors.initials: M
authors.lastname: Tanaka
authors.forename: Nao
authors.initials: N
authors.lastname: Yoshida
authors.forename: Kimikazu
authors.initials: K
authors.lastname: Matsumoto
authors.forename: Koji
authors.initials: K
authors.lastname: Kato
authors.forename: Nobuhiro
authors.initials: N
authors.lastname: Watanabe
authors.forename: Seiji
authors.initials: S
authors.lastname: Kojima
mesh_terms: D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D005199:Fanconi Anemia / Q000188:drug therapy / Q000401:mortality / Q000601:surgery*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D008297:Male; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome