title: Matched sibling donor stem cell transplantation for Fanconi anemia patients with T-cell somatic mosaicism.
abstract: SCT from HLA-identical sibling donors is generally associated with an excellent survival in FA patients if performed prior to the development of MDS or leukemia. However, the optimal conditioning regimen has not been defined. We report here our experience with 15 Japanese FA patients who underwent HLA-matched sibling donor SCT. The aim of this study is to compare radiation-based conditioning to Flu-based conditioning for FA patients in a Japanese population where the T-cell somatic mosaicism is higher than in the Caucasian population. Eight patients (a-group) received a radiation-based conditioning (500-600 cGy of thoracoabdominal/TBI) with CY dose modification (20-120 mg/kg), and ATG; two patients exhibited rejection. Seven patients (b-group) received CY (40 mg/kg), 150-180 mg/m(2) of Flu, and ATG. Durable engraftment was demonstrated in all patients. In FA patients, Flu-based conditioning may allow stable engraftment in matched sibling donor transplantation without radiation, even in patients with T-cell somatic mosaicism.
authors.affiliation: Department of Cell Transplantation, Tokai University Hospital, Kanagawa, Japan. miharu@is.icc.u-tokai.ac.jp
authors.forename: Miharu
authors.initials: M
authors.lastname: Yabe
authors.forename: Takashi
authors.initials: T
authors.lastname: Shimizu
authors.forename: Tsuyoshi
authors.initials: T
authors.lastname: Morimoto
authors.forename: Takashi
authors.initials: T
authors.lastname: Koike
authors.forename: Hiromitsu
authors.initials: H
authors.lastname: Takakura
authors.forename: Hideo
authors.initials: H
authors.lastname: Tsukamoto
authors.forename: Kazuo
authors.initials: K
authors.lastname: Muroi
authors.forename: Koichi
authors.initials: K
authors.lastname: Oshima
authors.forename: Keiko
authors.initials: K
authors.lastname: Asami
authors.forename: Minoru
authors.initials: M
authors.lastname: Takata
authors.forename: Takayuki
authors.initials: T
authors.lastname: Yamashita
authors.forename: Shunichi
authors.initials: S
authors.lastname: Kato
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
mesh_terms: D000293:Adolescent; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D046528:Chimerism; D036101:Cord Blood Stem Cell Transplantation; D003520:Cyclophosphamide / Q000627:therapeutic use; D005199:Fanconi Anemia / Q000235:genetics / Q000601:surgery*; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000517:prevention & control; D006086:Graft vs Host Disease / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008297:Male; D009030:Mosaicism; D033581:Stem Cell Transplantation; D013601:T-Lymphocytes; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine