Unrelated peripheral blood stem cell transplantation with 'megadoses' of purified CD34+ cells in three children with refractory severe aplastic anemia.
Three children with refractory severe aplastic anemia were transfused with high numbers of unrelated matched (n = 2) or C-locus haploidentical mismatched (n = 1) CD34-selected peripheral blood stem cells in the absence of an HLA-identical family donor. Two leukaphereses of the donors yielded a median number of 10.1 x 10(10) nucleated cells (range 9.7-15.4) with a median number of 9.89 x 10(8) CD34+ cells (range 7.46-26.1) and a median percentage of CD34+cells of 0.98% (range 0.77-1.7). After positive selection by magnetic cell sorting the patients received a median of 14.3 x 10(6) CD34+ cells/kg (range 11.7-24.3) and of 1.3 x 10(4) CD3+ cells/kg (range 0.57-5.8). Median time to ANC >/=0.5 x 10(9)/l was 7 days (range 7-12) and to platelets >/=20 x 10(9)/l 13 days (range 13-27). Chimerism analysis of peripheral blood after transplantation revealed permanent 100% donor hematopoiesis in all patients. The patient with the C-locus haploidentical mismatch presented with acute GVHD (grade III-IV) of the skin, liver and lower gastrointestinal tract (onset day +40) and died despite intensive immunosuppressive treatment on day +238. The two survivors developed lymphopoietic recovery of B and T lymphocytes within 3 months after transplantation. To our knowledge this experience represents the first report of transplantation with unrelated CD34+ enriched peripheral blood stem cell in children with refractory severe aplastic anemia. Bone Marrow Transplantation (2000) 25, 513-517.
Schwinger
W
W
Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Graz, Austria.
Urban
C
C
Lackner
H
H
Kerbl
R
R
Benesch
M
M
Dornbusch
H J
HJ
Sovinz
P
P
Schauenstein
K
K
Schumm
M
M
Handgretinger
R
R
D000293:Adolescent; D000741:Anemia, Aplastic / Q000150:complications / Q000628:therapy*; D015703:Antigens, CD / Q000032:analysis; D018952:Antigens, CD34 / Q000097:blood / Q000627:therapeutic use*; D016516:CD4-CD8 Ratio; D002648:Child; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D007937:Leukapheresis / Q000379:methods; D018655:Lymphocyte Count; D008297:Male; D013234:Stem Cells / Q000276:immunology; D013997:Time Factors; D014019:Tissue Donors; D018183:Transplantation Chimera; D014184:Transplantation, Homologous
D015703:Antigens, CD; D018952:Antigens, CD34