Abstract
A boy whose chronic granulomatous disease (CGD) manifested in infancy, and whose elder brother had died at 7 years of age, had phagocytes with complete lack of functional cytochrome B-245 and which could not be induced by interferon gamma to achieve adequate staphylococcal killing. He underwent an elective displacement bone marrow transplant from a volunteer unrelated donor at the age of 8 months. This has achieved 100% replacement of the CGD granulocytes by those of the normal volunteer and the boy has since had a normal childhood for 3 years. Six previous transplants for CGD are briefly reviewed and illustrate that the host abnormal marrow must be completely displaced using an adequate dose of busulphan to ensure 100% stable engraftment of the donor's marrow and that this is best done under elective conditions before septic foci and irreversible organ damage have occurred. Criteria need to be developed to identify early those patients likely to have severe morbidity.
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Abbreviations
- BMT:
-
bone marrow transplant
- CGD:
-
chronic granulomatous disease
- GVHD:
-
graft-versus-host disease
- NBT:
-
nitroblue tetrazolium
- rHuIFNg:
-
recombinant human interferon-gamma
- VUD:
-
volunteer unrelated donor
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Hobbs, J.R., Monteil, M., McCluskey, D.R. et al. Chronic granulomatous disease 100% corrected by displacement bone marrow transplantation from a volunteer unrelated donor. Eur J Pediatr 151, 806–810 (1992). https://doi.org/10.1007/BF01957929
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DOI: https://doi.org/10.1007/BF01957929