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Anakinra treatment in patients with familial Mediterranean fever: a single-centre experience
Serdal Ugurlu1, Bilgesu Ergezen1, Bugra Han Egeli1
1Division of Rheumatology, Department of Internal Medicine, Cerrahpasa Medical Faculty, Istanbul University-Cerrahpasa, Istanbul, Turkey.
Objectives:
Anakinra is proven to be effective in controlled trials in terms of attack frequency and subclinical inflammation in colchicine-resistant patients. The objective of this study was to review the patients followed in our single centre with FMF who received anakinra because of insufficient colchicine response.
Methods:
The study was conducted at a tertiary rheumatology centre experienced in autoinflammatory diseases. The patients were treated for at least 1 month with anakinra. Patients with amyloidosis and pregnancy were not included. Attack frequency, patient global assessment scales of disease severity and acute phase reactants were recorded before and throughout anakinra treatment. Criteria of treatment termination were side effects, disease remission, inadequate response, pregnancy plan and non-compliance.
Results:
One hundred and six patients diagnosed with FMF were treated with anakinra; 45.92% of the patients had a homozygous M694V mutation; 83 of the 98 patients tested for MEFV carried at least one copy of M694V. Attack frequency decreased while on anakinra treatment; in fact, no attacks were observed in 75 patients. Visual analogue scale score decreased from 7.49 (2.03) to 3.08 (2.82) (P = 0.001). Currently, 71 patients are still on anakinra treatment. Treatment of 34 patients was discontinued (32%). Insufficient response and side effects were the most common reasons for treatment discontinuation. All of the side effects observed were reversible and the patients alleviated after treatment cessation. In four patients, leukopenia was observed.
Conclusion:
In patients who were refractory to colchicine, anti-IL-1 agent anakinra was shown to be effective and safe. The effectiveness of anakinra stems from preventing attacks and increasing the quality of life.
Insights
Anakinra effectively reduces Familial Mediterranean Fever (FMF) attacks and inflammation in colchicine-resistant patients. This anti-IL-1 therapy improves quality of life and is generally safe, though some patients discontinue due to side effects or inadequate response.
Area of Science:
- Rheumatology
- Autoinflammatory Diseases
- Genetics
Background:
- Familial Mediterranean Fever (FMF) is a genetic autoinflammatory disease characterized by recurrent inflammatory attacks.
- Colchicine is the standard treatment, but some patients are resistant or intolerant.
- Anakinra, an anti-interleukin-1 agent, has shown promise in managing FMF in clinical trials.
Purpose of the Study:
- To review the outcomes of FMF patients treated with anakinra due to insufficient response to colchicine at a single tertiary center.
- To evaluate the efficacy and safety of anakinra in a real-world clinical setting for colchicine-resistant FMF.
Main Methods:
- A retrospective review of 106 FMF patients treated with anakinra for at least one month.
- Data collected included attack frequency, patient-reported disease severity (Visual Analogue Scale), and acute phase reactants.
- Exclusion criteria included amyloidosis and pregnancy.
Main Results:
- Anakinra treatment led to a significant decrease in attack frequency, with 75 patients experiencing no attacks.
- Patient global assessment scores improved significantly (P < 0.001).
- 32% of patients discontinued treatment due to insufficient response or side effects; all side effects were reversible.
Conclusions:
- Anakinra is an effective and safe therapeutic option for FMF patients refractory to colchicine.
- The drug demonstrates efficacy in preventing attacks and improving patients' quality of life.
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