Abstract
An 8 year old boy affected by a rare form of X-linked Chronic Granulomatous Disease (CGD) and present cytochrome b underwent successful bene marrow transplantation on August 1989 in Pescara 13.4 ×10 nucleated cells/Kg) from his 6 year old histocompatible sister (HLA-identical and HLC negative) after a preconditioning regimen of Busaifan (3.25 mg/kg/day for 4 days) followed by Cyclophosphamide (50 mg/kg/day for 4 days). The patient was protected by LAF isolator and by our decontamination protocol. Cyclosporme was administered for 12 months and oral Acyclovir for 270 days, The clinical course was uncomplicated and the boy was engrafted promptly with PMN counts normalizing or day +28 and the complete reversal of neutrophil function defect. Cytogenetic showed complete engraftment of donor origin at 4 months after BMt, Eighteen months after BMT the boy still continues to be free of infections. In fact NBT was already positive at the nirteth month for Zymosan (92%) and for PHA (91%), The 0% generation hith PMA was 8.88 nmol/min/10− cells and with fMLA was 13.01 nmol/min/106 cells. Cytochrome b spectra showed peals at 428, 530, 556 nm (7.81 pmol/106 cells) and all his neutrophils stained for cytochrome b on the cell surface by using monoclonal antibodies 7D5 (98%). Earlier atrespts of BMT for CGD by bone marrow from related or unrelated donors have failed either because of slow loss of the graft and gradual deterioration in neutrophil function or because of lethal infections and/or GvH disease. We conclude that as well as in CGD %/b-, previously described b/ us (Bone Narrow Transplant 4, 695, 1989;, a complete neutrophil engraftment can be achieved also in this rare form CGD X/b+.
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Manzionna, M., Bartolomeo, P., Girolamo, B. et al. 102 BONE MARROW TRANSPLANIATION FOR CHRONIC GRANULDMATDUS DISEASE X/b+. Pediatr Res 30, 645 (1991). https://doi.org/10.1203/00006450-199112000-00132
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DOI: https://doi.org/10.1203/00006450-199112000-00132