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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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Blood, 1 August 2002, Vol. 100, No. 3, pp. 774-777
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Allogeneic bone marrow transplantation: cure for familial
Mediterranean fever
John Milledge,
Peter J. Shaw,
Albert Mansour,
Sarah Williamson,
Bruce Bennetts,
Tony Roscioli,
Julie Curtin, and
John Christodoulou
From the Oncology Department, Department of
Paediatrics, Western Sydney Genetics Program, Department of
Haematology, Royal Alexandra Hospital for Children, Sydney, Australia;
and Department of Paediatrics and Child Health, University of Sydney,
Sydney, Australia.
We describe data on a 7-year-old girl with congenital
dyserythropoietic anemia (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, at the age of 4 years, she underwent
allogeneic, sibling bone marrow transplantation (BMT). 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, 2 years after the transplantation, 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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