TY - JOUR
T1 - Results of unrelated cord blood transplant in fanconi anemia patients: risk factor analysis for engraftment and survival
AU - Gluckman, Eliane
AU - Rocha, Vanderson
AU - Ionescu, Irina
AU - Bierings, Marc
AU - Harris, Richard E.
AU - Wagner, John
AU - Kurtzberg, Joanne
AU - Champagne, Martin A.
AU - Bonfim, Carmem
AU - Bittencourt, Marco
AU - Darbyshire, Philip
AU - Fernandez, Manuél-Nicolas
AU - Locatelli, Franco
AU - Pasquini, Ricardo
PY - 2007
Y1 - 2007
N2 - 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 4.9 X 107 /kg and 1.9 X 105/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 >= 4.9 x 107 /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 >= 4.9 x 10(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. (c) 2007 American Society for Blood and Marrow Transplantation
AB - 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 4.9 X 107 /kg and 1.9 X 105/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 >= 4.9 x 107 /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 >= 4.9 x 10(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. (c) 2007 American Society for Blood and Marrow Transplantation
KW - Fanconi anemia
KW - fludarabine
KW - unrelated cord blood transplantation
KW - Fanconi anemia
KW - fludarabine
KW - unrelated cord blood transplantation
UR - http://hdl.handle.net/10807/257391
U2 - 10.1016/j.bbmt.2007.05.015
DO - 10.1016/j.bbmt.2007.05.015
M3 - Article
SN - 1083-8791
VL - 13
SP - 1073
EP - 1082
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
ER -