TY - JOUR
T1 - The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen
T2 - A report from the Italian Pediatric Group
AU - Locatelli, Franco
AU - Zecca, Marco
AU - Pession, Andrea
AU - Morreale, Giuseppe
AU - Longoni, Daniela
AU - Di Bartolomeo, Paolo
AU - Porta, Fulvio
AU - Fagioli, Franca
AU - Nobili, Bruno
AU - Bernardo, Maria Ester
AU - Messina, Chiara
PY - 2007/10
Y1 - 2007/10
N2 - Background and Objectives: Hematopoietic stem cell transplantation (HSCT) still represents the only treatment potentially able to prevent/rescue the development of marrow failure and myeloid malignancies in patients with Fanconi anemia (FA). While in the past HSCT from an HLA-identical sibling was proven to cure many patients, a higher incidence of treatment failure has been reported in recipients of an unrelated donor (UD) or HLA-partially matched related allograft. Design and Methods: We analyzed the outcome of 64 FA patients (age range, 2-20 years) who underwent HSCT between January 1989 and December 2005. Patients were transplanted from either an HLA-identical sibling (n=31), an UD (n=26), or an HLA-partially matched relative (n=7). T-cell depletion of the graft was performed in patients transplanted from an HLA-disparate relative. Results: The 8-year estimate of overall survival (OS) for the whole cohort was 67%; it was 87%, 40% and 69% when the donor was an HLA-identical sibling, an UD and a mismatched relative, respectively (p
AB - Background and Objectives: Hematopoietic stem cell transplantation (HSCT) still represents the only treatment potentially able to prevent/rescue the development of marrow failure and myeloid malignancies in patients with Fanconi anemia (FA). While in the past HSCT from an HLA-identical sibling was proven to cure many patients, a higher incidence of treatment failure has been reported in recipients of an unrelated donor (UD) or HLA-partially matched related allograft. Design and Methods: We analyzed the outcome of 64 FA patients (age range, 2-20 years) who underwent HSCT between January 1989 and December 2005. Patients were transplanted from either an HLA-identical sibling (n=31), an UD (n=26), or an HLA-partially matched relative (n=7). T-cell depletion of the graft was performed in patients transplanted from an HLA-disparate relative. Results: The 8-year estimate of overall survival (OS) for the whole cohort was 67%; it was 87%, 40% and 69% when the donor was an HLA-identical sibling, an UD and a mismatched relative, respectively (p
KW - Fanconi anemia
KW - Fludarabine
KW - Hematopoietic stem cell transplantation
KW - Unrelated donor
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U2 - 10.3324/haematol.11436
DO - 10.3324/haematol.11436
M3 - Article
C2 - 18024375
AN - SCOPUS:36348964259
VL - 92
SP - 1381
EP - 1388
JO - Haematologica
JF - Haematologica
SN - 0390-6078
IS - 10
ER -