Therapy for Patients With Familial Mediterranean Fever When Colchicine Is Not Enough or Is Not Tolerated FREE

Familial Mediterranean fever (FMF) is a rare disease that causes repeated bouts of fever and accumulations of fluid in the body, causing pain in the joints, abdomen, chest, and other sites. There is no known cure for the disease, but many patients have a decrease in the number of attacks with a drug called colchicine. Some patients, however, do not experience enough relief with colchicine or have problematic side effects, making its use difficult or impossible. At present, there is no known therapy for such patients. Familial Mediterranean fever is caused by abnormalities in a gene that controls the action of a chemical involved in inflammation called interleukin-1.

To see whether rilonacept, a drug that can stop the function of interleukin-1, may help patients with FMF who cannot tolerate colchicine or have attacks despite its use.

14 patients with FMF who could not tolerate colchicine or had continued attacks while taking it.

After 1 to 3 months of observation on current therapies to see how many attacks were normally experienced at “baseline,” each patient received rilonacept injections under the skin each week for 3 months as well as a 3-month period of placebo injections; each 3-month treatment was repeated. The order of treatments was randomized, and neither the patients nor the doctors knew which one was being received. During each 3-month period, the number of FMF attacks was monitored. Patients (or their parents, if the patients were children) also assessed the effect of FMF on their quality of life during the treatment periods.

During the treatment periods with rilonacept, patients had fewer attacks of FMF than with placebo, and more patients had a decrease to less than one half of their previous baseline number. Patients also reported better physical aspects of quality of life with rilonacept. Patients had pain with the rilonacept injections more often than with placebo, although all of these reactions were considered mild by the patients.

Because FMF is a rare disease, there were few patients in the study and there may be differences between these patients and others with the disease. In addition, the study was not large enough to assess whether patients with certain gene abnormalities causing FMF are more likely to respond to rilonacept. The study could not assess how long the effect of rilonacept may last or whether there are long-term safety problems. It is also not known whether rilonacept can help prevent other long-term health problems in FMF that colchicine can prevent.

Rilonacept may be a treatment option for patients with FMF who cannot tolerate colchicine or have attacks despite its use.