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Blood, 1 May 2002, Vol. 99, No. 9, pp. 3458-3460

BRIEF REPORT

HLA-haploidentical blood progenitor cell transplantation in osteopetrosis

Ansgar S. Schulz, Carl Friedrich Classen, Walther A. Mihatsch, Michael Sigl-Kraetzig, Markus Wiesneth, Klaus-Michael Debatin, Wilhelm Friedrich, and Susanna M. Müller

From the Departments of Pediatrics and Transfusion Medicine, University of Ulm, Ulm, Germany.

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 progenitor cells were purified by positive selection and by additional T-cell depletion using rosette formation. For conditioning, patients received busulfan, thiotepa, and either cyclophosphamide (5 patients) or fludarabine (2 patients). Stable donor engraftment developed in 6 of 7 patients. Graft-versus-host disease was not observed. Three of the 7 patients had no major complications and 4 of 7 had both veno-occlusive disease and respiratory failure. Five of 7 patients survive with complete cure of OP at a median of 4 years. Patients with OP lacking HLA-matched donors can be successfully treated by transplantation of purified blood progenitor cells from HLA-haploidentical donors.

© 2002 by The American Society of Hematology.
 

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