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Table 2 Studies and case reports that discussed the use of Canakinumab in FMF

From: Update on the management of colchicine resistant Familial Mediterranean Fever (FMF)

Study Description Follow-up
Mitroulis et al. [68] A 25-year-old woman with FMF, homozygous for the MEFV gene mutation, and with longstanding articular involvement affecting her hips and left knee. She had been under treatment with colchicine since the age of 5. At the age of 19, she experienced long-lasting arthritis of her right hip and was treated with anakinra which was discontinued due to severe injection site reactions. She was switched to treatment with etanercept and low dose prednisone which induced a destructive arthritis of the right hip and chronic arthritis of her left knee. Methotrexate was added to the existing treatment. Long-term remission was not achieved. The patient only responded to canakinumab.
Hacihamdioglu et al. [69] A 14-year-old girl homozygote for the M694 V mutation in the MEFV gene became colchicine resistant after 4 years of starting treatment. The patient improved clinically and biochemically after canakinumab initiation until day 70 when she had a relapse. A second dose was administered again with full response.
Brik et al. [70] 7 participants received 3 subcutaneous injections of canakinumab. The primary outcome measure was the proportion of participants with ≥50% reduction in the frequency of FMF attacks during the treatment period versus the pretreatment period. Secondary outcome measures included acute-phase reactant levels, health-related quality of life, physician’s global assessment of FMF control, time to attack following the last canakinumab injection, and safety and tolerability of canakinumab. 6 participants met the primary outcome measure with a ≥ 50% reduction (range 76–100%) in the rate of FMF attacks. 18 of 34 attacks (53%) were rated as severe or very severe during the pretreatment phase, compared with 0 of 8 during the treatment phase. There were no serious adverse events.
Alpa et al. [71] A 30-year old woman with FMF on colchicine for 6 years developed recurrent attacks. Increased colchicine dose was ineffective. The effects of canakinumab were monitored over the following 6-month period. A significant improvement in the clinical symptoms was observed after the first administration. During treatment with canakinumab, the patient reported only three minor episodes of FMF.
Gul et al. [72] Patients who had an attack despite maximal dose of colchicine in the preceding 3 months were eligible for the study. At the first attack, patients started to receive three canakinumab 150 mg subcutaneous injections at 4-week intervals, and were then followed for an additional 2 months. 13 patients were enrolled in the run-in period and 9 advanced to the treatment period. All 9 patients achieved a 50% or more reduction in attack frequency. C-reactive protein and serum amyloid A protein levels remained low throughout the treatment period.
Only 5 patients had an attack during the 2-month follow-up.
Sozeri et al. [73] A 14-year-old male with colchicine-resistant FMF and amyloidosis homozygous for the M694 V mutation in MEFV gene. Because of his poor response to colchicine, severe growth retardation, and severe proteinuria due to amyloidosis, canakinumab was started. After 26 months of follow-up, his complaints, inflammatory parameters, and proteinuria were decreased. No side effects were noted.
Ozkan et al. [74] This study was performed with 4 patients with colchicine-resistant FMF. None of the patients had proteinuria or amyloidosis. First, anakinra was given to all the patients in addition to colchicine and showed a rapid and dramatic effect on attacks of FMF and inflammatory findings. There were no side effects, but daily anakinra injection therapy was changed to monthly injection of canakinumab because of easy implementation.
Yaziltas et al. [75] This study retrospectively evaluated 11 pediatric colchicine-resistant FMF patients who were treated with canakinumab. Three of the patients had amyloidosis and two had uveitis. In all, three patients had FMF-associated amyloidosis, nephrotic syndrome, and CKD, of which 2 had clinical improvement with canakinumab and 1 had partial response. Among the patients, only 1 case died1 year after canakinumab cessation due to sepsis. 9 of our patients continued to receive canakinumab treatment.
Jesenak et al. [76] A 36-year-old man from central Europe presented with a very severe clinical FMF and intolerance to colchicine. The clinical effect of the application of anakinra was insufficient and accompanied with side effects and low tolerability. Switching to canakinumab induced a rapid remission of the disease activity and inflammatory markers.
De Benedetti et al. [77] 63 colchicine-resistant FMF patients were tested for the effectiveness of canakinumab. Primary outcome was described as the resolution of the index flare by day 15 and no new episodes over 16 weeks of treatment. The primary outcome was achieved by 61% of patients in the treatment group compared to 6% in the placebo arm. No opportunistic infections, tuberculosis, or death was observed but 3 serious infections were reported in two patients.
Trabulus et al. [78] A total of 9 kidney transplant recipients who developed AA amyloidosis due to FMF were enrolled. All of the patients had rapid or gradual disappearance of FMF attacks. 1 patient developed a reaction to injection while another showed symptoms of Cytomegalovirus pneumonia.