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Published on: August 29, 2012
Chronic granulomatous disease 100% corrected by displacement bone marrow transplantation from a volunteer unrelated
J R Hobbs1, M Monteil, D R McCluskey
1Department of Immunology, Charing Cross and Westminster Medical School, Westminster Hospital, London, United Kingdom.
Insights
Bone marrow transplants can successfully treat chronic granulomatous disease (CGD) in children. Complete displacement of abnormal marrow with donor cells ensures a normal childhood, highlighting the importance of early intervention.
Area of Science:
- Immunology
- Pediatric Hematology
- Genetics
Background:
- Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by a lack of functional cytochrome B-245 in phagocytes.
- CGD leads to recurrent severe infections and significant morbidity, often with fatal outcomes in childhood.
Observation:
- A boy with CGD, whose sibling died from the disease, presented with non-functional phagocytes resistant to interferon gamma stimulation.
- He received an elective unrelated donor bone marrow transplant at 8 months of age.
Findings:
- The transplant resulted in 100% replacement of the patient's CGD granulocytes with normal donor cells.
- The patient has experienced a normal childhood for three years post-transplant, indicating successful engraftment and immune reconstitution.
Implications:
- This case underscores the efficacy of bone marrow transplantation for CGD, emphasizing complete marrow ablation with busulphan for stable engraftment.
- Early elective transplantation before irreversible organ damage is crucial for optimal outcomes.
- Further research is needed to establish criteria for early identification of high-risk CGD patients requiring intensive management.
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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