Abstract
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 × 1010nucleated cells (range 9.7–15.4) with a median number of 9.89 × 108CD34+ 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 × 106 CD34+ cells/kg (range 11.7–24.3) and of 1.3 × 104 CD3+ cells/kg (range 0.57–5.8). Median time to ANC ⩾0.5 × 109/l was 7 days (range 7–12) and to platelets ⩾20 × 109/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.
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Schwinger, W., Urban, C., Lackner, H. et al. Unrelated peripheral blood stem cell transplantation with ‘megadoses’ of purified CD34+ cells in three children with refractory severe aplastic anemia. Bone Marrow Transplant 25, 513–517 (2000). https://doi.org/10.1038/sj.bmt.1702202
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DOI: https://doi.org/10.1038/sj.bmt.1702202
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