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This Article Full Text Full Text (PDF) Alert me when this article is cited Alert me if a correction is posted Citation Map 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 Horwitz, E. M. Articles by Brenner, M. K. Search for Related Content PubMed PubMed Citation Articles by Horwitz, E. M. Articles by Brenner, M. K. Related Collections Transplantation Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  Blood, 1 March 2001, Vol. 97, No. 5, pp. 1227-1231 CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta Edwin M. Horwitz, Darwin J. Prockop, Patricia L. Gordon, Winston W. K. Koo, Lorraine A. Fitzpatrick, Michael D. Neel, M. Elizabeth McCarville, Paul J. Orchard, Reed E. Pyeritz, and Malcolm K. Brenner From the Cell and Gene Therapy Program, St Jude Children's Research Hospital, Memphis, TN; the Center for Gene Therapy, MCP-Hahnemann Medical School, Philadelphia, PA; the Department of Pediatrics and Obstetrics/Gynecology, Wayne State University, Detroit, MI; the Department of Internal Medicine, Mayo Clinic, Rochester, NY; the Department of Pediatrics, University of Minnesota, Minneapolis, MN; and the Department of Human Genetics, MCP Hahnemann, Pittsburgh, PA. Preclinical models have shown that transplantation of marrow mesenchymal cells has the potential to correct inherited disorders of bone, cartilage, and muscle. The report describes clinical responses of the first children to undergo allogeneic bone marrow transplantation (BMT) for severe osteogenesis imperfecta (OI), a genetic disorder characterized by defective type I collagen, osteopenia, bone fragility, severe bony deformities, and growth retardation. Five children with severe OI were enrolled in a study of BMT from human leukocyte antigen (HLA)-compatible sibling donors. Linear growth, bone mineralization, and fracture rate were taken as measures of treatment response. The 3 children with documented donor osteoblast engraftment had a median 7.5-cm increase in body length (range, 6.5-8.0 cm) 6 months after transplantation compared with 1.25 cm (range, 1.0-1.5 cm) for age-matched control patients. These patients gained 21.0 to 65.3 g total body bone mineral content by 3 months after treatment or 45% to 77% of their baseline values. With extended follow-up, the patients' growth rates either slowed or reached a plateau phase. Bone mineral content continued to increase at a rate similar to that for weight-matched healthy children, even as growth rates declined. These results suggest that BMT from HLA-compatible donors may benefit children with severe OI. Further studies are needed to determine the full potential of this strategy. © 2001 by The American Society of Hematology.   CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: S. Ohba, F. Yano, and U.-i. Chung Tissue Engineering of Bone and Cartilage IBMS BoneKEy, November 1, 2009; 6(11): 405 - 419. [Abstract] [Full Text] [PDF] A. H. Undale, J. J. Westendorf, M. J. Yaszemski, and S. Khosla Mesenchymal Stem Cells for Bone Repair and Metabolic Bone Diseases Mayo Clin. Proc., October 1, 2009; 84(10): 893 - 902. [Abstract] [Full Text] [PDF] C. Panaroni, R. Gioia, A. Lupi, R. Besio, S. A. Goldstein, J. Kreider, S. Leikin, J. C. Vera, E. L. Mertz, E. Perilli, et al. 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