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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.

AbstractBACKGROUND 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<0.01). The outcome of recipients of grafts from an UD improved over time, the probability of survival being 10% and 72% for patients transplanted before and after 1998, respectively (p<0.05). The OS probability of children who did or did not receive fludarabine in preparation for the allograft was 86% and 59%, respectively (p<0.05). INTERPRETATION AND CONCLUSIONS: These data, useful for counselling, provide support to the concept that a relevant proportion of FA patients undergoing HSCT can now be successfully cured, even in the absence of an HLA-identical sibling, especially if the conditioning regimen includes fludarabine.
AuthorsFranco Locatelli, Marco Zecca, Andrea Pession, Giuseppe Morreale, Daniela Longoni, Paolo Di Bartolomeo, Fulvio Porta, Franca Fagioli, Bruno Nobili, Maria Ester Bernardo, Chiara Messina, Italian pediatric group (Affiliation: Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo e Università di Pavia, Pavia, Italy. f.locatelli at smatteo.pv.it)
JournalHaematologica (Haematologica) Vol. 92 Issue 10 Pg. 1381-8 (Oct 2007) ISSN: 1592-8721 Italy
PMID18024375 (Publication Type: Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
Chemical References
  • fludarabine
  • Vidarabine
Topics
  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Fanconi Anemia (pathology, surgery)
  • Female
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Italy
  • Male
  • Probability
  • Survival Rate
  • Transplantation Conditioning
  • Treatment Outcome
  • Vidarabine (analogs & derivatives, pharmacology)