Allogeneic bone marrow transplantation: cure for familial Mediterranean fever

Abstract

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. (Blood. 2002;100: 774-777)

How to Manage Familial Mediterranean Fever (FMF) Patients in Daily Practice

“…Therefore, the concept that bone marrow transplantation has no role in the therapeutic approach to patients with FMF is shared by all clinicians dealing with FMF. Nevertheless, an interesting case report underscoring the curative potential of this therapeutic modality therapy has been reported by Milledge et al [ 40 ]. They reported on a 7-year-old girl with congenital dyserythropoietic anemia (CDA) who also had FMF.…”

Familial autoinflammatory diseases: genetics, pathogenesis and treatment

“…In such patients, several adjunctive approaches are under investigation, including subcutaneous interferon-a [125,126 • ,127 • ] and biologic therapies aimed at TNF [128 • ] or interleukin-1b. Allogeneic bone marrow transplantation has recently been proposed as a treatment for refractory FMF [129], based on the predominant expression of MEFV in leukocytes [5]. Although it is possible that this approach could be effective, in nearly all cases other options exist, and the risks outweigh the potential benefits [130].…”

Successful Treatment with Infliximab and Low-dose Methotrexate in a Japanese Patient with Familial Mediterranean Fever

“…Allogeneic bone marrow transplantation has also been shown to be effective in the prevention of FMF attacks (9). Several recent reports have demonstrated that the anti-tumor necrosis factor (TNF)-α monoclonal antibody, infliximab, reduces the frequency of FMF attacks in Spanish and Turkish patients (10,11 …”