title: Transplantation-related mortality, graft failure, and survival after reduced-toxicity conditioning and allogeneic hematopoietic stem cell transplantation in 100 consecutive pediatric recipients.
abstract: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) with myeloablative conditioning is associated with a 10%-40% risk of day +100 transplantation-related mortality (TRM). We evaluated the feasibility and safety of reduced-toxicity conditioning and allo-HSCT in 100 consecutive children and adolescent recipients (mean age, 9.2 ± 6.8 years). The mean duration of follow-up was 1278 ± 1042 days. Fifty patients had malignant disease. The median time to neutrophil recovery was 18 days, and the median time to platelet recovery was 43 days. Median donor chimerism in engrafted patients was 98% on day +100 and 98% on day +365. The cumulative incidence of acute graft-versus-host disease (GVHD) was 20% (95% confidence interval [CI], 12.1%-27.9%), and that of chronic GVHD was 13.5% (95% CI, 6.6%-20.4%). TRM was 3% (95% CI, 0%-6.4%) by day +100 and 13.6% (95% CI, 6.7%-20.5%) for the entire study period. The incidence of primary graft failure (PGF) was 16% overall, 31.4% after umbilical cord blood transplantation (UCBT), and 0% after allo-HSCT with matched unrelated or matched sibling donors (P < .0001). The incidence of PGF in UCBT recipients was 46.7% (14 of 30) in chemotherapy-naive recipients, versus 9.5% (2 of 21) in non-chemotherapy-naive recipients (P = .019). Five-year event-free survival was 59.5% ± 5%, and 5-year overall survival was 72.9% ± 5%. Only PGF and poor-risk disease status were significantly associated with decreased overall survival (P = .03). Reduced-toxicity conditioning allo-HSCT in pediatric recipients is associated with low TRM; however, chemotherapy-naive UCBT recipients have a significantly higher incidence of PGF.
authors.affiliation: Department of Pediatrics, Columbia University, New York, NY 10032, USA. ps2087@columbia.edu
authors.forename: Prakash
authors.initials: P
authors.lastname: Satwani
authors.forename: Zhezhen
authors.initials: Z
authors.lastname: Jin
authors.forename: Deirdre
authors.initials: D
authors.lastname: Duffy
authors.forename: Erin
authors.initials: E
authors.lastname: Morris
authors.forename: Monica
authors.initials: M
authors.lastname: Bhatia
authors.forename: James H
authors.initials: JH
authors.lastname: Garvin
authors.forename: Diane
authors.initials: D
authors.lastname: George
authors.forename: Mary Brigid
authors.initials: MB
authors.lastname: Bradley
authors.forename: Lauren
authors.initials: L
authors.lastname: Harrison
authors.forename: Kristen
authors.initials: K
authors.lastname: Petrillo
authors.forename: Joseph
authors.initials: J
authors.lastname: Schwartz
authors.forename: Sandra
authors.initials: S
authors.lastname: Foley
authors.forename: Ria
authors.initials: R
authors.lastname: Hawks
authors.forename: Lee Ann
authors.initials: LA
authors.lastname: Baxter-Lowe
authors.forename: Mitchell S
authors.initials: MS
authors.lastname: Cairo
mesh_terms: D000293:Adolescent; D001792:Blood Platelets / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000517:prevention & control*; D006086:Graft vs Host Disease / Q000276:immunology* / Q000401:mortality / Q000473:pathology; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D008297:Male; D019653:Myeloablative Agonists / Q000627:therapeutic use; D009504:Neutrophils / Q000276:immunology; D018183:Transplantation Chimera / Q000276:immunology; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous
chemical_list: D019653:Myeloablative Agonists