| 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. |
| Forthcoming: |
No |
| Link eMedia: |
Download |
| 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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