HLA-haploidentical blood progenitor cell transplantation in osteopetrosis.
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.
Schulz
Ansgar S
AS
Department of Pediatrics, University of Ulm, Ulm, Germany. ansgar.schulz@gmx.de
Classen
Carl Friedrich
CF
Mihatsch
Walther A
WA
Sigl-Kraetzig
Michael
M
Wiesneth
Markus
M
Debatin
Klaus-Michael
KM
Friedrich
Wilhelm
W
Müller
Susanna M
SM
D001773:Blood Cells / Q000637:transplantation; D018572:Disease-Free Survival; D005192:Family Health; D005260:Female; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008212:Lymphocyte Depletion; D008297:Male; D010022:Osteopetrosis / Q000150:complications / Q000151:congenital / Q000628:therapy*; D019172:Transplantation Conditioning / Q000009:adverse effects; D016896:Treatment Outcome