title: Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival.
abstract: The potential benefits of unrelated donor marrow transplantation are offset by the immunologic complications of graft-versus-host disease (GVHD) and infection. Therefore, we used cryopreserved umbilical cord blood (UCB) as a strategy to reduce the risks of GVHD and treatment-related mortality (TRM) and improve survival. Data on 102 patients (median age 7.4 years) who received transplants between 1994 and 2001 for the treatment of malignant (n = 65; 68% were high-risk patients) and nonmalignant (n = 37) diseases were evaluated. Log-rank tests and Cox regression analyses were used to determine the effects of various demographic, graft-related, and treatment factors on engraftment, GVHD, TRM, relapse, and survival. As of October 15, 2001, the median follow-up was 2.7 years (range, 0.3-7.2). Incidences of neutrophil and platelet engraftment were 0.88 (CI, 0.81-0.95) and 0.65 (CI, 0.53-0.77), respectively. Notably, incidences of severe acute and chronic GVHD were 0.11 (CI, 0.05-0.17) and 0.10 (CI, 0.04-0.16), respectively. At 1 year after transplantation, proportions of TRM and survival were 0.30 (CI, 0.21-0.39) and 0.58 (CI, 0.48-0.68), respectively. In Cox regression analyses, CD34 cell dose was the one factor consistently identified as significantly associated with rate of engraftment, TRM, and survival. Despite the low incidence of GVHD, the proportion of patients with leukemia relapse at 2 years was 0.17 (CI, 0.00-0.38) and 0.45 (CI, 0.28-0.61) for patients with standard and high-risk disease, respectively. There is a high probability of survival in recipients of UCB grafts that are disparate in no more than 2 human leukocyte antigens (HLAs) when the grafts contain at least 1.7 x 10(5) CD34(+) cells per kilogram of recipient's body weight. Therefore, graft selection should be based principally on CD34 cell dose when multiple UCB units exist with an HLA disparity of 2 or less.
authors.affiliation: Blood and Marrow Transplant Program of the Department of Pediatrics, University of Minnesota Cancer Center and School of Medicine, Minneapolis 55455, USA. wagner002@tc.umn.edu
authors.forename: John E
authors.initials: JE
authors.lastname: Wagner
authors.forename: Juliet N
authors.initials: JN
authors.lastname: Barker
authors.forename: Todd E
authors.initials: TE
authors.lastname: DeFor
authors.forename: K Scott
authors.initials: KS
authors.lastname: Baker
authors.forename: Bruce R
authors.initials: BR
authors.lastname: Blazar
authors.forename: Cindy
authors.initials: C
authors.lastname: Eide
authors.forename: Anne
authors.initials: A
authors.lastname: Goldman
authors.forename: John
authors.initials: J
authors.lastname: Kersey
authors.forename: William
authors.initials: W
authors.lastname: Krivit
authors.forename: Margaret L
authors.initials: ML
authors.lastname: MacMillan
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Orchard
authors.forename: Charles
authors.initials: C
authors.lastname: Peters
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Weisdorf
authors.forename: Norma K C
authors.initials: NK
authors.lastname: Ramsay
authors.forename: Stella M
authors.initials: SM
authors.lastname: Davies
mesh_terms: D000293:Adolescent; D000328:Adult; D018952:Antigens, CD34; D001772:Blood Cell Count; D002648:Child; D002675:Child, Preschool; D005260:Female; D005312:Fetal Blood; D006680:HLA Antigens; D006402:Hematologic Diseases / Q000401:mortality / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006650:Histocompatibility Testing; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D008659:Metabolic Diseases / Q000401:mortality / Q000628:therapy*; D008875:Middle Aged; D012008:Recurrence; D016019:Survival Analysis; D014184:Transplantation, Homologous
chemical_list: D018952:Antigens, CD34; D006680:HLA Antigens