Abstract
A 5 month-old Amish male infant with CGD underwent BMT from his 5 year-old, histocompatible brother after a preconditioning regimen of busulfan 8 mg/kg and cyclophosphamide 200 mg/kg. At the time of BMT, he was free of infection and remained so through the course of his transplant. He engrafted promptly with complete reversal of the neutrophil function defect (Table), and no sign of graft vs. host disease. This was followed by loss of the erythroid graft and gradual deterioration in neutrophil function (Table). Now, 16 months after BMT, he is free of infection, growing normally with essentially no evidence for neutrophil engraftment.
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Kamani, N., August, C., Douglas, S. et al. BONE MARROW TRANSPLANTATION (BMT) IN CHRONIC GRANULOMATOUS DISEASE (CGD). Pediatr Res 18 (Suppl 4), 242 (1984). https://doi.org/10.1203/00006450-198404001-00892
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DOI: https://doi.org/10.1203/00006450-198404001-00892
