title: Secular trends in outcomes for Fanconi anemia patients who receive transplants: implications for future studies.
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.
authors.affiliation: Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Rockville, MD 20852, USA. rosenbep@mail.nih.gov
authors.forename: Philip S
authors.initials: PS
authors.lastname: Rosenberg
authors.forename: Blanche P
authors.initials: BP
authors.lastname: Alter
authors.forename: Gerard
authors.initials: G
authors.lastname: Socié
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
mesh_terms: D016026:Bone Marrow Transplantation / Q000401:mortality / Q000706:statistics & numerical data / Q000639:trends; D005199:Fanconi Anemia / Q000401:mortality* / Q000628:therapy; D005260:Female; D006086:Graft vs Host Disease / Q000401:mortality* / Q000628:therapy; D006650:Histocompatibility Testing; D006801:Humans; D019520:Living Donors; D008297:Male; D015337:Multicenter Studies as Topic / Q000639:trends; D018307:Neoplasms, Squamous Cell / Q000401:mortality / Q000556:secondary; D012189:Retrospective Studies; D012307:Risk Factors; D019737:Transplants / Q000639:trends