HLA-haploidentical blood progenitor cell transplantation in osteopetrosis

Creators: Schulz, Ansgar S. and Classen, Carl F. and Mihatsch, Walter A. and Sigel-Kraetzig, Michael and Wiesneth, Markus and Debatin, Klaus-Michael and Friedrich, Wilhelm and Müller, Susanna M.
Title: HLA-haploidentical blood progenitor cell transplantation in osteopetrosis
Item Type: Article or issue of a publication series
Journal or Series Title: Blood : the journal of hematology
Page Range: pp. 3458-3460
Date: May 2002
Divisions: Gesundheitsmanagement
Abstract: 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.
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Citation:

Schulz, Ansgar S. and Classen, Carl F. and Mihatsch, Walter A. and Sigel-Kraetzig, Michael and Wiesneth, Markus and Debatin, Klaus-Michael and Friedrich, Wilhelm and Müller, Susanna M. (2002) HLA-haploidentical blood progenitor cell transplantation in osteopetrosis. Blood : the journal of hematology, 99 (9). pp. 3458-3460. ISSN 0006-4971

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