Abstract
Anti-interleukin 1 agents are used successfully in colchicine-resistant or intolerant Familial Mediterranean Fever (FMF) patients. Sixty-five patients with FMF who received canakinumab treatment for at least 6 months due to colchicine resistance or intolerance between 2016 and 2020 in our department were retrospectively analyzed. Canakinumab treatment was given subcutaneously every 4 weeks. After completing monthly canakinumab therapy over 12 months, in patients with complete remission, the dosing interval was extended to every 1.5 months for 6 months, then every 2 months for 6 months, and finally every 3 months for a year. In patients without disease activation, canakinumab treatment was discontinued at the end of 3 years and followed up with colchicine treatment. Patients who had a flare switched to the previous dosing interval. In patients with renal amyloidosis, monthly canakinumab treatment was continued without extending the dose intervals. The mean duration of canakinumab use in our patients was 31.4 ± 10.57 months (6–52 months). The mean age at onset of symptoms was 4.65 ± 3.84 (range, 1–18) years, and the mean age at diagnosis was 5.59 ± 3.9 (range, 4–19) years. Complete remission was achieved in 57 (87.6%) and partial remission in seven (10.7%) patients. One patient was unresponsive to treatment. Canakinumab treatment was discontinued in three patients with complete remission and one patient with drug resistance. Erythrocyte sedimentation rate (ESR) (51.85 ± 15.7 vs. 27.80 ± 13.73 mm/h) and C-reactive protein (CRP) [26 (3-73) vs. 5 (1–48) mg/L] values were compared before and after canakinumab treatment in attack-free periods, a significant decrease was found after canakinumab treatment (p < 0.001, p < 0.001, respectively). Bodyweight Z-scores (respectively −0.80 ± 0.86 vs. −0.49 ± 0.92) were compared, similarly, a statistically significant increase after canakinumab treatment (p < 0.001), but no significant increase in height Z scores (−1.00 ± 0.88 vs. −0.96 ± 0.94) (p = 0.445) was detected. Four patients had FMF-related renal amyloidosis. The decrease in proteinuria with canakinumab treatment was not statistically significant (p = 0.068). Cervical lymphadenitis developed in one and local reactions in two patients. No severe adverse effects requiring discontinuation of canakinumab treatment were observed. Our study showed that canakinumab treatment was highly effective, well-tolerated in pediatric FMF patients, and controlled extension of the canakinumab dose interval was safe.
Highlights
Familial Mediterranean fever (FMF) is the most common autoinflammatory disease with autosomal recessive inheritance, characterized by recurrent attacks of fever and serositis
Of the 65 patients, complete remission was achieved in 57 (87.6%) and partial remission in seven (10.7%); the desired response was not achieved in only one patient
Complete remission was achieved in 57 patients, and partial remission was achieved in seven patients; the desired response was not achieved in only one patient
Summary
Familial Mediterranean fever (FMF) is the most common autoinflammatory disease with autosomal recessive inheritance, characterized by recurrent attacks of fever and serositis. The disease often affects ethnic groups of the Eastern Mediterranean region, and the frequency of FMF carriers among Sephardic and Ashkenazi Jews, Arabs, Italians, Armenians, and Turks is around 1/3–1/5 [1, 2]. FMF occurs due to mutations of the Mediterranean fever (MEFV) gene located on the short arm of chromosome 16. This gene encodes a protein made up of 781 amino acids called pyrin ( known as marenostrin). Gain of function mutations in the MEFV gene cause increased activation of the pyrin/inflammasome complex [3]
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