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Blood, 15 November 2008, Vol. 112, No. 10, pp. 4318-4327. Prepublished online as a Blood First Edition Paper on August 21, 2008; DOI 10.1182/blood-2007-06-098020.
TRANSPLANTATION Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies1 Duke University Medical Center, Durham, NC; 2 EMMES Corporation, Rockville, MD; 3 University of Minnesota, Minneapolis; 4 University of California, San Francisco; 5 Texas Transplant Institute, San Antonio; 6 Children's Hospital of Los Angeles, CA; 7 Dana-Farber Cancer Institute, Boston, MA; 8 Mattel Children's Hospital, Los Angeles, CA; 9 National Heart, Lung, and Blood Institute, Bethesda, MD; and 10 Memorial Sloan-Kettering Cancer Center, New York, NY Outcomes of unrelated donor cord blood transplantation in 191 hematologic malignancy children (median age, 7.7 years; median weight, 25.9 kg) enrolled between 1999 and 2003 were studied (median follow-up, 27.4 months) in a prospective phase 2 multicenter trial. Human leukocyte antigen (HLA) matching at enrollment was 6/6 (n = 17), 5/6 (n = 58), 4/6 (n = 111), or 3/6 (n = 5) by low-resolution HLA-A, -B, and high-resolution (HR) DRB1. Retrospectively, 179 pairs were HLA typed by HR. The median precryopreservation total nucleated cell (TNC) dose was 5.1 x 107 TNC/kg (range, 1.5-23.7) with 3.9 x 107 TNC/kg (range, 0.8-22.8) infused. The median time to engraftment (absolute neutrophil count > 500/mm3 and platelets 50 000/µL) was 27 and 174 days. The cumulative incidence of neutrophil engraftment by day 42 was 79.9% (95% confidence interval [CI], 75.1%-85.2%); acute grades III/IV GVHD by day 100 was 19.5% (95% CI, 13.9%-25.5%); and chronic GVHD at 2 years was 20.8% (95% CI, 14.8%-27.7%). HR matching decreased the probability of severe acute GVHD. The cumulative incidence of relapse at 2 years was 19.9% (95% CI, 14.8%-25.7%). The probabilities of 6-month and 2-year survivals were 67.4% and 49.5%. Unrelated donor cord blood transplantation from partially HLA-mismatched units can cure many children with leukemias. The study was registered at www.clinicaltrials.gov as #NCT00000603 [ClinicalTrials.gov] .
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