HLA-haploidentical blood progenitor cell transplantation in osteopetrosis.
作者： Ansgar S. Schulz , Carl Friedrich Classen , Walther A. Mihatsch , Michael Sigl-Kraetzig , Markus Wiesneth
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摘要: Infantile osteopetrosis (OP) carries an extremely poor prognosis unless treated early by hematopoietic stem cell transplantation. We explored the use of purified blood progenitor cells from HLA-haploidentical parents in 7 patients lacking suitable matched donors. Blood were positive selection and additional T-cell depletion using rosette formation. For conditioning, received busulfan, thiotepa, either cyclophosphamide (5 patients) or fludarabine (2 patients). Stable donor engraftment developed 6 patients. Graft-versus-host disease was not observed. Three had no major complications 4 both veno-occlusive respiratory failure. Five survive with complete cure OP at a median years. Patients HLA-matched donors can be successfully transplantation
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