Real-world Use of Canakinumab in Familial Mediterranean Fever and Other Autoinflammatory Disorders: A Medical Records Review Single-center Study From Turkey.

Background/objective: Canakinumab (CAN), a monoclonal antibody targeting interleukin-1β, has demonstrated efficacy in various autoinflammatory diseases (AIDs), particularly in inadequate response to colchicine in familial Mediterranean fever (FMF). This study aimed to evaluate the indications, efficacy, and safety of CAN based on real-life experience from a tertiary rheumatology clinic.

Methods: This single-center study included 54 patients treated with CAN between May 2020 and September 2024. Patients were grouped as MEFV-positive FMF (n=42), MEFV-negative FMF (n=7), non-FMF autoinflammatory diseases (n=2), and adult-onset Still's disease (AOSD; n=3). Demographic and clinical data, treatment indications, response patterns, laboratory parameters, and adverse events were analyzed.

Results: CAN was initiated mainly due to adverse effects (40.5%) or inadequate response (42.8%) to anakinra and colchicine. The median duration of CAN therapy was 22 months. Among MEFV-positive FMF patients, 81% achieved a complete response and 19% partial response. CAN significantly reduced attack frequency and duration, and improved inflammatory markers (CRP, ESR, WBC, and neutrophil count). Proteinuria decreased in a statistically significant but clinically modest manner following CAN treatment. Only 1 patient experienced reversible cytopenia. Dose intervals were successfully prolonged in 54.8% of MEFV-positive patients without loss of efficacy.

Conclusions: Canakinumab is an effective and well-tolerated IL-1β inhibitor in FMF and other AIDs, particularly in patients who are inadequately responsive or intolerant to colchicine and anakinra. Real-world experience supports its sustained efficacy and the feasibility of dose interval extension in selected cases.