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

doi:10.7326/0003-4819-157-8-201210160-00001

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

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What is the problem and what is known about it so far?

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.

Why did the researchers do this particular study?

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.

Who was studied?

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

How was the study done?

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.

What did the researchers find?

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.

What were the limitations of the study?

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.

What are the implications of the study?

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

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Summaries for Patients |16 October 2012

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

What is the problem and what is known about it so far?

Why did the researchers do this particular study?

Who was studied?

How was the study done?

What did the researchers find?

What were the limitations of the study?

What are the implications of the study?

This feature is available only to Registered Users

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Summaries for Patients |16 October 2012

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

What is the problem and what is known about it so far?

Why did the researchers do this particular study?

Who was studied?

How was the study done?

What did the researchers find?

What were the limitations of the study?

What are the implications of the study?

This feature is available only to Registered Users

0 Comments

See Also

Related Articles

Journal Club

Related Topics

PubMed Articles

Content

Information For

Services

Awards and Cover

Other Resources

Follow Annals On

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