156A clicnical trial of gene transfer to bone marrow CD34+ cells from ADA-deficient SCID subjects
Year: 2003
Type: article
Abstract: Poster Presentations -Session II and the Nashville Veterans Affairs Hospital from 1994 to 2002.Central pathologic review was performed on biopsies from 12 patients.All but two patients presented with advanced stage at diagnosis.Median age at transplant for the autologous and allogeneic groups was 53.5 and 49.5 years, respectively.A total of 17 autologous, 1 syngeneic, and 6 alIogeneic transplants were performed.Five patients underwent autologous transplantation in first complete remission.Results: In the autologous group, median overall survival was 4.75 years (95% C.I. 3.7 to 5.8y) and median survival from transplant was 2.5 years (95% C.I. 1.3 to 3.7y).The Kaplan-Meier estimate of 5-year overall survival was 41% (95% C.I. 10% to 72%). in the allogeneic group, 4 patients have died.Post-transplant survival ranged from 13 days to 3.4 years.Chemosensitivity, defined as the maintenance of at least a partial response after the last chemotherapy regimen until the date of autologous transplant, was associated with improved overall survival by log-rank analysis (p=.0005).For these 13 patients the estimated 5-year overall survival was 56% (95% C.I. 17% to 95%).
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W1978463025