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.
Gaipa
G
G
Clinica Pediatrica, Università di Milano-Bicocca, Ospedale San Gerardo, Monza, Italy.
Dassi
M
M
Perseghin
P
P
Venturi
N
N
Corti
P
P
Bonanomi
S
S
Balduzzi
A
A
Longoni
D
D
Uderzo
C
C
Biondi
A
A
Masera
G
G
Parini
R
R
Bertagnolio
B
B
Uziel
G
G
Peters
C
C
Rovelli
A
A
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