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Prepublished online as a Blood First Edition Paper on June 14, 2002; DOI 10.1182/blood-2002-02-0651.

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Submitted February 28, 2002
Accepted March 19, 2002

Allogeneic bone marrow transplant: cure for Familial Mediterranean Fever

John Milledge, Peter J Shaw*, Albert Mansour, Sarah Williamson, Bruce Bennetts, Tony Roscioli, Julie Curtin, and John Christodoulou

Department of Oncology, Children's Hospital at Westmead, Sydney, NSW, Australia
Department of Oncology, Children's Hospital at Westmead, Sydney, NSW, Australia; Paediatrics and Child Health, University of Sydney, Sydney, NSW, Australia
Department of Paediatrics, Children's Hospital at Westmead, Sydney, NSW, Australia
Western Sydney Genetics Program, Children's Hospital at Westmead, Sydney, NSW, Australia
Department of Haematology, Children's Hospital at Westmead, Sydney, NSW, Australia
Western Sydney Genetics Program, Children's Hospital at Westmead, Sydney, NSW, Australia; Paediatrics and Child Health, University of Sydney, Sydney, NSW, Australia

* Corresponding author; email: peters{at}chw.edu.au.

We describe a seven year-old girl with congenital dyserythropoietic anaemia (CDA), who also had Familial Mediterranean Fever (FMF). Repeated transfusions required since the age of 6 months to treat her CDA led to iron overload and a persistently high ferritin level. Her relapsing FMF made effective iron chelation therapy very difficult. Consequently, she underwent allogeneic, sibling bone marrow transplant (BMT) at the age of 4 years. During conditioning for her BMT, symptoms of FMF, including splenomegaly, arthritis and recurrent abdominal pain, began to resolve and she was gradually weaned off colchicine. Now, two years post-transplant, she remains free from FMF symptomatology and is off all immunosuppressants. This case demonstrates that symptoms of FMF can be alleviated by the therapy used during allogeneic BMT. In this patient it is likely that the missing factor in FMF is now being provided by granulocytes derived from the stem cells within transplanted bone marrow.


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