Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes.
Outcomes of 159 young patients with inherited metabolic disorders (IMDs) undergoing transplantation with partially HLA-mismatched unrelated donor umbilical cord blood were studied to investigate the impact of graft and patient characteristics on engraftment, overall survival (OS), and graft-versus-host disease (GVHD). Patients received myeloablative chemotherapy (busulfan, cyclophosphamide, ATG) and cyclosporine-based GVHD prophylaxis. Infused cell doses were high (7.57 x 10(7)/kg) because of the patients' young age (median, 1.5 years) and small size (median, 12 kg). Median follow-up was 4.2 years (range, 1-11 years). The cumulative incidences of neutrophil and platelet engraftment were 87.1% (95% confidence interval [CI], 81.8%-92.4%) and 71.0% (95% CI, 63.7%-78.3%). A total of 97% achieved high (> 90%) donor chimerism. Serum enzyme normalized in 97% of patients with diseases for which testings exist. Grade III/IV acute GVHD occurred in 10.3% (95% CI, 5.4%-15.2%) of patients. Extensive chronic GVHD occurred in 10.8% (95% CI, 5.7%-15.9%) of patients by 1 year. OS at 1 and 5 years was 71.8% (95% CI, 64.7%-78.9%) and 58.2% (95% CI, 49.7%-66.6%) in all patients and 84.5% (95% CI, 77.0%-92.0%) and 75.7% (95% CI, 66.1%-85.3%) in patients with high (80-100) performance score. In multivariate analysis, favorable factors for OS were high pretransplantation performance status, matched donor/recipient ethnicity, and higher infused colony forming units.
Prasad
Vinod K
VK
Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, NC, USA. vinod.prasad@duke.edu .
Mendizabal
Adam
A
Parikh
Suhag H
SH
Szabolcs
Paul
P
Driscoll
Timothy A
TA
Page
Kristin
K
Lakshminarayanan
Sonali
S
Allison
June
J
Wood
Susan
S
Semmel
Deborah
D
Escolar
Maria L
ML
Martin
Paul L
PL
Carter
Shelly
S
Kurtzberg
Joanne
J
D000293:Adolescent; D000328:Adult; D001792:Blood Platelets / Q000166:cytology; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000628:therapy; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D008661:Metabolism, Inborn Errors / Q000401:mortality / Q000628:therapy*; D015999:Multivariate Analysis; D009504:Neutrophils / Q000166:cytology; D015996:Survival Rate; D014019:Tissue Donors; D016896:Treatment Outcome; D014481:United States / Q000453:epidemiology