Bone Marrow Transplants
for Children with Severe OI

The OI Foundation's Medical Advisory Council reviewed an article published in the March 1999 issue of Nature Medicine concerning bone marrow transplants for children with severe OI, which were performed at the St. Jude's Research Hospital in Memphis. After reviewing and discussing the published article, the Medical Advisory Council provided the following statement:

The article by Horwitz and coworkers, published in Nature Medicine on March 1, 1999, reports initial results using a complex and controversial experimental procedure to treat osteogenesis imperfecta (OI), also called "brittle bone disease."

The authors transplanted bone marrow, taken from siblings, into three children who had suffered multiple bone fractures and deformity from this heritable disease. They describe improvements in bone mineral density, better growth, and fewer fractures, all occurring during a relatively brief period after marrow transplantation.

Although the findings are of interest, the clinical nature of OI and the techniques used to assess the impact of the bone marrow transplantation on the skeleton, some inherently imprecise, do not permit a definitive evaluation of the results at this time. Close follow-up of these three patients over several years will be necessary to explain the early findings, to clarify the risks and benefits of this potentially fatal procedure, and to fully assess its role compared to other existing or emerging treatments for OI. Researchers must also develop and study good animal models for OI to help evaluate bone marrow transplantation for this disorder.

The following physicians and researchers compose the OI Foundation Medical Advisory Council:

Chair: Michael Whyte, M.D., Shriners Hospital for Children, St. Louis, Mo.; Peter Bullough, M.D., Hospital for Special Surgery, New York, N.Y.; Peter Byers, M.D., University of Washington, Department of Pathology, Seattle, Wash.; Lynn Gerber, M.D., National Institutes of Health, Bethesda, Md.; Francis Glorieux, M.D., Ph.D., Shriners Hospital for Children, Montreal, Quebec; Joan Marini, M.D., National Institutes of Health, Bethesda, Md.; Darwin Prockop, M.D., Center for Gene Therapy, Allegheny University, Philadelphia, Pa.; David Rowe, M.D., University of Connecticut Health Center, Farmington, Conn.; Jay Shapiro, M.D., Walter Reed Army Medical Center, Washington, D.C.; David Vernick, Joint Center for Otolarygology, Boston, Mass.

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