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Blood, 1 October 2008, Vol. 112, No. 7, pp. 2979-2989. Prepublished online as a Blood First Edition Paper on June 27, 2008; DOI 10.1182/blood-2008-03-140830. This Article Full Text (PDF) All Versions of this Article: blood-2008-03-140830v1 112/7/2979    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Prasad, V. K. Articles by Kurtzberg, J. Search for Related Content PubMed PubMed Citation Articles by Prasad, V. K. Articles by Kurtzberg, J. Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted March 18, 2008 Accepted June 17, 2008 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 transplant outcomes Vinod K. Prasad*, Adam Mendizabal, Suhag H Parikh, Paul Szabolcs, Timothy A Driscoll, Kristin Page, Sonali Lakshminarayanan, June Allison, Susan Wood, Deborah Semmel, Maria L Escolar, Paul L Martin, Shelly Carter, and Joanne Kurtzberg Division of Blood and Marrow Transplant, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States Biostatistics, EMMES Corporation, Rockville, MD, United States Program for Neurodevelopmental Function in Rare Disorders, Ctr for study of Development and Learning, University of North Carolina, Chapel Hill, NC, United States * Corresponding author; email: vinod.prasad{at}duke.edu. Unrelated donor umbilical cord blood transplantation (UCBT) has yielded encouraging results in leukemia patients lacking suitable adult donors. We present results of 159 young patients with inherited metabolic disorders (IMD) transplanted with partially HLA-mismatched unrelated donor UCB grafts and 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. Cell doses infused were high (7.57x107/kg) because of patient's young age (median 1.5years) and small size (median 12kg). Median follow-up was 4.2 years (range 1-11). The cumulative incidences of neutrophil and platelet engraftment were 87.1% (95%CI 81.8%-92.4%) and 71.0% (95%CI 63.7%-78.3%). 97% achieved high donor-chimerism (>90%). Serum enzyme normalized in 97% patients with diseases for which testing exist. Grade III/IV acute GvHD occurred in 10.3% (95%CI 5.4%-15.2%). Extensive chronic GvHD occurred in 10.8% (95%CI: 5.7%-15.9%) by 1 year. OS at 1 and 5 years were 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 (Lansky 80-100) performance score. In multivariate analysis, favorable factors for OS were high pre-transplant performance status, matched donor/recipient ethnicity, and higher infused colony forming units. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: L. Ye, J. C. Chang, C. Lin, X. Sun, J. Yu, and Y. W. Kan Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases PNAS, June 16, 2009; 106(24): 9826 - 9830. [Abstract] [Full Text] [PDF]

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