Summary:
A 13-year-old Caucasian female with a systemic connective tissue disease (overlap syndrome with pulmonary vasculitis) underwent nonmyeloablative allogeneic BMT after failure of prolonged combination immunosuppressives to induce remission. The procedure also included cotransplantation of donor bone chips as a source of stromal cells. The unique protocol allowed good engraftment of hematopoietic (>95%) and bone core stromal cells (>60%). The patient was clinically improved, stable, and off all immunosuppressive medications 36 months post-transplant. To our knowledge, this is the first pediatric nonmyeloablative BMT with cotransplantation of stromal cells solely for treatment of an autoimmune disease.
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Acknowledgements
We express our sincere appreciation to Dr Allison Green, the primary care pediatrician, and all specialty physicians, including Drs Martin Klemperer, Gail Cawkwell, and Magdalen Gondor for their excellent care and collaboration. We thank Drs Noorbibi Day and Joe M Jones for critical review and Ms Tazim Verjee for help in the preparation of this manuscript.
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Jones, O., Good, R. & Cahill, R. Nonmyeloablative allogeneic bone marrow transplantation for treatment of childhood overlap syndrome and small vessel vasculitis. Bone Marrow Transplant 33, 1061–1063 (2004). https://doi.org/10.1038/sj.bmt.1704482
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DOI: https://doi.org/10.1038/sj.bmt.1704482
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