Secular trends in outcomes for fanconi anemia patients who receive transplants: Implications for future studies

Philip S. Rosenberg, Blanche P. Alter, Gerard Socié, Eliane Gluckman

Research output: Contribution to journalArticlepeer-review


Transplantation protocols for patients with Fanconi anemia are being modified continuously. However, it is unclear how outcomes have changed over time. We determined historical adverse event rates from long-term follow-up of 117 Fanconi anemia patients in the Hôpital Saint Louis transplant cohort, who received low-dose cyclophosphamide- and irradiation-based conditioning, in combination with other modalities, between 1976 and October 2002. In high-risk patients with mismatched donors, the peritransplantation mortality rate during 0 to 6 months declined significantly over time (P = .003), from 28%/month (95% confidence interval [CI], 9%-87%/month) during 1985 to 1989 to 3.3%/ month (95% CI, 0.8%-13.3%/month) during 2000 to October 2002. The corresponding proportion of patients who developed severe acute graft-versus-host disease also declined significantly over time (P = .003). In low-risk patients with matched sibling donors, the peritransplantation mortality rate was consistently low, 1.4%/month (95% CI, 0.3%-5.3%/month), during 1990 to October 2002. Sample sizes to detect 2-fold reductions from rates and risks observed since the mid-1990s are larger than recently reported case series. To demonstrate further advances in survival, transplant centers may need to coordinate their protocols and engage in multicenter collaborative studies.

Original languageEnglish (US)
Pages (from-to)672-679
Number of pages8
JournalBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Issue number9
StatePublished - Sep 2005
Externally publishedYes


  • Fanconi anemia
  • Numerical data
  • Risk assessment
  • Statistics
  • Stem cell transplant
  • Study design

ASJC Scopus subject areas

  • Transplantation

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