The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: A report from the Italian Pediatric Group

Franco Locatelli, Marco Zecca, Andrea Pession, Giuseppe Morreale, Daniela Longoni, Paolo Di Bartolomeo, Fulvio Porta, Franca Fagioli, Bruno Nobili, Maria Ester Bernardo, Chiara Messina

Research output: Contribution to journalArticlepeer-review

Abstract

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

Original languageEnglish
Pages (from-to)1381-1388
Number of pages8
JournalHaematologica
Volume92
Issue number10
DOIs
Publication statusPublished - Oct 2007

Keywords

  • Fanconi anemia
  • Fludarabine
  • Hematopoietic stem cell transplantation
  • Unrelated donor

ASJC Scopus subject areas

  • Hematology

Fingerprint Dive into the research topics of 'The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: A report from the Italian Pediatric Group'. Together they form a unique fingerprint.

Cite this