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Two infants with malignant osteopetrosis were treated by allogeneic bone-marrow transplantation after marrow ablation with busulphan and cyclophosphamide. Engraftment without graft-vs-host disease occurred in both cases. The first child established stable chimerism and is clinically well 30 months after receiving a histocompatible sibling bone-marrow graft: haematological, radiological, and biochemical features of osteopetrosis have completely resolved. The second child was non-identical with the marrow donor (his older sister) at a single HLA-B locus and received cyclosporin A as prophylaxis for graft-vs-host disease. 11 months after transplantation haematological abnormalities have resolved and radiological features have improved. The findings indicate that allogeneic bone-marrow transplantation should be considered the treatment of choice for infants with severe osteopetrosis who have histocompatible siblings.
C A Sieff, and
J M Chessells, and
R J Levinsky, and
J Pritchard, and
D W Rogers, and
A Casey, and
K Muller, and
C M Hall
November 1995,
Journal of pediatric hematology/oncology,
C A Sieff, and
J M Chessells, and
R J Levinsky, and
J Pritchard, and
D W Rogers, and
A Casey, and
K Muller, and
C M Hall
March 1980,
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C A Sieff, and
J M Chessells, and
R J Levinsky, and
J Pritchard, and
D W Rogers, and
A Casey, and
K Muller, and
C M Hall
January 2022,
Kathmandu University medical journal (KUMJ),
C A Sieff, and
J M Chessells, and
R J Levinsky, and
J Pritchard, and
D W Rogers, and
A Casey, and
K Muller, and
C M Hall
December 2001,
Pediatric radiology,
C A Sieff, and
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R J Levinsky, and
J Pritchard, and
D W Rogers, and
A Casey, and
K Muller, and
C M Hall
November 1997,
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C A Sieff, and
J M Chessells, and
R J Levinsky, and
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A Casey, and
K Muller, and
C M Hall
January 1999,
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C A Sieff, and
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J Pritchard, and
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A Casey, and
K Muller, and
C M Hall
April 1988,
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C A Sieff, and
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R J Levinsky, and
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C A Sieff, and
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January 2004,
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C A Sieff, and
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A Casey, and
K Muller, and
C M Hall
March 1990,
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