title: Results of unrelated cord blood transplant in fanconi anemia patients: risk factor analysis for engraftment and survival.
abstract: We retrospectively analyzed results of unrelated cord blood transplantation (UCBT) in 93 Fanconi anemia (FA) patients. Median age at transplantation was 8.6 years (1-45). The units transplanted were HLA-A, -B, or -DRB1 identical in 12 cases, 1 HLA mismatch in 35 cases, and 2 or 3 HLA differences in 45 cases. The median number of nucleated cells (NC) and CD34+ cells infused of recipient weight was 4.9x10(7)/kg and 1.9x10(5)/kg, respectively. Participating centers selected the preparative regimen of their choice, in 57 patients (61%), it included Fludarabine. Graft-versus-host disease (GVHD) prophylaxis consisted mostly of cyclosporine with prednisone. Cumulative incidence (CI) of neutrophil recovery was 60+/-5% at day +60. In multivariate analysis, Fludarabine containing regimen and NC infused>or=4.9x10(7)/kg were associated with higher probability of recovery. CI of grade II-IV acute and of chronic GVHD (aGVHD, cGVHD) was 32%+/-5% and 16%+/-4%, respectively. Overall survival (OS) was 40%+/-5%. In multivariate analysis, factors associated with favorable outcome were use of Fludarabine in the conditioning regimen, number of NC infused>or=4.9x10(7)/kg, and negative cytomegalovirus (CMV) serology in the recipient. In conclusion, factors easily modifiable such as donor selection and a Fludarabine-containing regimen can considerably improve survival in FA patients given a UCBT. These data are the basis for designing prospective protocols.
authors: Hôpital Saint Louis AP/HP, University of Paris VII, IUH, Paris, France. eliane.gluckman@sls.aphp.fr
authors: Eliane
authors: E
authors: Gluckman
authors: Vanderson
authors: V
authors: Rocha
authors: Irina
authors: I
authors: Ionescu
authors: Marc
authors: M
authors: Bierings
authors: Richard E
authors: RE
authors: Harris
authors: John
authors: J
authors: Wagner
authors: Joanne
authors: J
authors: Kurtzberg
authors: Martin A
authors: MA
authors: Champagne
authors: Carmem
authors: C
authors: Bonfim
authors: Marco
authors: M
authors: Bittencourt
authors: Philip
authors: P
authors: Darbyshire
authors: Manuél-Nicolas
authors: MN
authors: Fernandez
authors: Franco
authors: F
authors: Locatelli
authors: Ricardo
authors: R
authors: Pasquini
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D005199:Fanconi Anemia / Q000401:mortality / Q000628:therapy*; D005260:Female; D006085:Graft Survival; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D012307:Risk Factors; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use
chemical_list: D014740:Vidarabine; C024352:fludarabine