Allogeneic bone marrow stem cell transplantation following CD34+ immunomagnetic enrichment in patients with inherited metabolic storage diseases.
T-cell depletion is an essential step in reducing the risk of graft-versus-host disease (GVHD) in patients with inherited metabolic storage diseases (IMSD) undergoing hematopoietic stem cell transplantation. This goal can be achieved either by selective removal of T cells or by positive selection of CD34+ cells. Large-scale preparations of purified CD34+ cells from bone marrow products have not been extensively described. We report our results with bone marrow CD34+ cell enrichment using the CliniMACS system in eight children with IMSD. The median recovery of positively selected CD34+ cells was 46.2% with a purity of 97.5%, and a residual T cell content of 0.04 x 10(6). A median of 5.5 x 10(6)/kg of CD34+ cells was infused. All patients engrafted at a median time of 12 days and none of the patients developed GVHD. This method is technically feasible and can be successfully used to transplant children with IMSD.
Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, Monza, Italy.
G
G
Gaipa
M
M
Dassi
P
P
Perseghin
N
N
Venturi
P
P
Corti
S
S
Bonanomi
A
A
Balduzzi
D
D
Longoni
C
C
Uderzo
A
A
Biondi
G
G
Masera
R
R
Parini
B
B
Bertagnolio
G
G
Uziel
C
C
Peters
A
A
Rovelli
D018952:Antigens, CD34 / Q000276:immunology*; D001402:B-Lymphocytes / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D018189:Immunomagnetic Separation; D007223:Infant; D007965:Leukodystrophy, Globoid Cell / Q000276:immunology / Q000628:therapy*; D008212:Lymphocyte Depletion / Q000379:methods*; D008297:Male; D008059:Mucopolysaccharidosis I / Q000276:immunology / Q000628:therapy*; D013601:T-Lymphocytes / Q000276:immunology; D014184:Transplantation, Homologous / Q000276:immunology; D016896:Treatment Outcome
D018952:Antigens, CD34