Real-World Experience with Canakinumab in Familial Mediterranean Fever: A Single-Center Study

Abstract

Familial Mediterranean fever (FMF) is an autoinflammatory disorder caused by mutations in the Mediterranean fever gene (MEFV), leading to excessive interleukin-1 beta (IL-1β) production. While colchicine is the primary treatment for FMF, a subset of patients exhibits resistance or intolerance, necessitating alternative therapeutic strategies. Canakinumab, a selective IL-1β inhibitor, has emerged as a potential treatment option. This study aims to evaluate canakinumab's real-world efficacy and safety in colchicine-resistant or colchicine-intolerant FMF patients. A retrospective, single-center study was conducted on FMF patients aged over 18 who initiated canakinumab treatment between January 2013 and October 2023. A total of 34 patients experiencing colchicine resistance or intolerance criteria were analyzed. Clinical and laboratory parameters, including Pras scores, erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), and serum amyloid A (SAA), were assessed before and after canakinumab treatment. Statistical analyses were performed using the Wilcoxon test and paired sample t-test. Canakinumab treatment significantly reduced Pras scores (p<0.001), ESR (p<0.001), CRP (p<0.001), and SAA levels (p<0.001). A decrease was observed post-treatment among patients with proteinuria, though not statistically significant (p=0.140). Treatment was discontinued in three patients due to active disease or adverse effects. No serious infections were reported. In conclusion, canakinumab could be a promising treatment option in colchicine-resistant or colchicine-intolerant FMF patients.

Keywords

• Canakinumab
• Colchicine intolerant
• Colchicine resistant
• Familial Mediterranean fever

Ailevi Akdeniz Ateşinde Kanakinumab ile Gerçek Yaşam Deneyimi: Tek Merkezli Bir Çalışma

Abstract

Ailesel Akdeniz ateşi (AAA), Mediterranean Fever (MEFV) geninde mutasyonların neden olduğu ve aşırı interlökin-1 beta (IL-1β) üretimine yol açan otoinflamatuar bir hastalıktır. Kolşisin AAA için ana tedavi olsa da, hastaların bir alt grubu direnç veya intolerans göstererek alternatif tedavi stratejilerine ihtiyaç duymaktadır. Selektif bir IL-1β inhibitörü olan kanakinumab, potansiyel bir tedavi seçeneği olarak ortaya çıkmıştır. Bu çalışma, kolşisine dirençli veya kolşisine intoleransı olan AAA hastalarında kanakinumabın gerçek yaşamdaki etkinliğini ve güvenliğini değerlendirmeyi amaçlamaktadır. Ocak 2013 ve Ekim 2023 tarihleri arasında kanakinumab tedavisine başlayan 18 yaş üstü AAA hastalarında retrospektif, tek merkezli bir çalışmadır. Kolşisin direnci veya intoleransı kriterlerini karşılayan toplam 34 hasta analiz edildi. Pras skorları, eritrosit sedimantasyon hızı (ESR), C-reaktif protein (CRP) ve serum amiloid A (SAA) dahil olmak üzere klinik ve laboratuvar parametreleri kanakinumab tedavisinden önce ve sonra değerlendirilmiştir. İstatistiksel analizler Wilcoxon testi ve eşleştirilmiş örneklem t-testi kullanılarak gerçekleştirilmiştir. Kanakinumab tedavisi Pras skorlarını (p<0,001), ESR (p<0,001), CRP (p<0,001) ve SAA düzeylerini (p<0,001) anlamlı ölçüde azaltmıştır. Proteinürisi olan hastalarda tedavi sonrasında istatistiksel olarak anlamlı olmasa da bir düşüş gözlenmiştir (p=0,140). Aktif hastalık veya yan etkiler nedeniyle üç hastada tedavi kesilmiştir. Hiçbir ciddi enfeksiyon bildirilmemiştir. Sonuç olarak, kanakinumab kolşisine dirençli veya kolşisine intoleranslı AAA hastalarında umut verici bir tedavi seçeneği olabilir.

Keywords

• Kanakinumab
• Kolşsin intolreansı
• Kolşissin direnci
• Ailevi Akdeniz ateşi

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