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• W2000025145 abstract "Children and adolescents with homozygous beta-thalassemia can be cured by transplantation of normal stem cells after eradication of the thalassemic hematopoietic system. In an attempt to achieve durable engraftment and to minimize regimen-related toxicity (RRT), we have initiated a fludarabine-based pilot protocol not containing cyclophosphamide. Between 1999 and 2004, five children with beta-thalassemia major were enrolled. Median age at transplantation was 11.5 years (range 4–14 years). Three patients received conditioning with fludarabine (30 mg/m2/day × 6), oral busulfan (3.5 mg/kg/day × 4), and ATG rabbit Fresenius (10 mg/kg/day × 4). Two children received intravenous busulfan instead of oral busulfan at a dose of 2 × 1.4 mg/kg/day × 4 days. All children were transplanted with a fresh bone marrow graft from an HLA-identical sibling. Mean cell doses given were 3.7 × 108 nucleated cells/kg BW (range 2.4–6.2 × 108/kg). Overall, 5/5 patients achieved donor engraftment and are alive and well. No GVHD exceeding grade I was observed, and 2/5 children maintained donor chimerism at 100%. One patient maintains mixed hematopoietic donor chimerism being between 94 and 97% nearly 5 years after transplant." @default.
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• W2000025145 date "2005-07-04" @default.
• W2000025145 modified "2023-10-18" @default.
• W2000025145 title "Complete substitution of cyclophosphamide by fludarabine and ATG in a busulfan-based preparative regimen for children and adolescents with β-thalassemia" @default.
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• W2000025145 doi "https://doi.org/10.1038/sj.bmt.1705082" @default.
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