Abstract |
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.
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Authors | Philip S Rosenberg, Blanche P Alter, Gerard Socié, Eliane Gluckman |
Journal | Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
(Biol Blood Marrow Transplant)
Vol. 11
Issue 9
Pg. 672-9
(Sep 2005)
ISSN: 1083-8791 [Print] United States |
PMID | 16125637
(Publication Type: Journal Article)
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Topics |
- Bone Marrow Transplantation
(mortality, statistics & numerical data, trends)
- Fanconi Anemia
(mortality, therapy)
- Female
- Graft vs Host Disease
(mortality, therapy)
- Histocompatibility Testing
- Humans
- Living Donors
- Male
- Multicenter Studies as Topic
(trends)
- Neoplasms, Squamous Cell
(mortality, secondary)
- Retrospective Studies
- Risk Factors
- Transplants
(trends)
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