{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T23:51:00Z","timestamp":1793317860842,"version":"4.5.0"},"reference-count":35,"publisher":"American Society of Hematology","issue":"5","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2001,3,1]]},"abstract":"<jats:p>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)\u2013compatible 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.<\/jats:p>","DOI":"10.1182\/blood.v97.5.1227","type":"journal-article","created":{"date-parts":[[2002,7,27]],"date-time":"2002-07-27T12:46:47Z","timestamp":1027774007000},"page":"1227-1231","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":455,"title":["Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta"],"prefix":"10.1182","volume":"97","author":[{"given":"Edwin M.","family":"Horwitz","sequence":"first","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Darwin J.","family":"Prockop","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia L.","family":"Gordon","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Winston W. K.","family":"Koo","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorraine A.","family":"Fitzpatrick","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael D.","family":"Neel","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Elizabeth","family":"McCarville","sequence":"additional","affiliation":[{"name":"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."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul J.","family":"Orchard","sequence":"additional","affiliation":[{"name":"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 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