Secular trends in outcomes for Fanconi anemia patients who receive transplants: implications for future studies.
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.
Rosenberg
Philip S
PS
Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Rockville, MD 20852, USA. rosenbep@mail.nih.gov
Alter
Blanche P
BP
Socié
Gerard
G
Gluckman
Eliane
E
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