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

Tuğba Ocak; Ayşe Bulur; Burcu Yağız; Belkis Nihan Coskun; Hüseyin Ediz Dalkılıç; Yavuz Pehlivan

Makale detayları

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

Yazarlar:

Tuğba Ocak | Ayşe Bulur | Burcu Yağız | Belkis Nihan Coskun | Hüseyin Ediz Dalkılıç | Yavuz Pehlivan

Yayıncı:

Bursa Uludağ University

Ana Başlıklar:

Romatoloji ve Artrit

Dergi:

Uludağ Üniversitesi Tıp Fakültesi Dergisi Vol.51 Issue 1, pp.87-92

Belge Tipi:

Makale

Yayın Tarihi:

5/2025

Belge Dili:

İngilizce

Document ID (Org No.):

2001349001

pISSN:

1300-414X

Anahtar kelimeler:

Özet:

 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 canakinumabs 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 p0.001, ESR p0.001, CRP p0.001, and SAA levels p0.001. A decrease was observed post-treatment among patients with proteinuria, though not statistically significant p0.140. 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.

 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 canakinumabs 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 p0.001, ESR p0.001, CRP p0.001, and SAA levels p0.001. A decrease was observed post-treatment among patients with proteinuria, though not statistically significant p0.140. 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.

 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 canakinumabs 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 p0.001, ESR p0.001, CRP p0.001, and SAA levels p0.001. A decrease was observed post-treatment among patients with proteinuria, though not statistically significant p0.140. 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.

 Ailesel Akdeniz ateşi AAA, Mediterranean Fever MEFV geninde mutasyonların neden olduğu ve aşırı interlökin-1 beta IL-1β uretimine 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 inhibitöru olan kanakinumab, potansiyel bir tedavi seçeneği olarak ortaya çıkmıştır. Bu çalışma, kolşisine dirençli veya kolşisine intoleransı olan AAA hastalarında kanakinumabın gerçek yaşamdaki etkinliğini ve guvenliğini değerlendirmeyi amaçlamaktadır. Ocak 2013 ve Ekim 2023 tarihleri arasında kanakinumab tedavisine başlayan 18 yaş ustu 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 uzere 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 p0,001, ESR p0,001, CRP p0,001 ve SAA duzeylerini p0,001 anlamlı ölçude azaltmıştır. Proteinurisi olan hastalarda tedavi sonrasında istatistiksel olarak anlamlı olmasa da bir duşuş gözlenmiştir p0,140. Aktif hastalık veya yan etkiler nedeniyle uç hastada tedavi kesilmiştir. Hiçbir ciddi enfeksiyon bildirilmemiştir. Sonuç olarak, kanakinumab kolşisine dirençli veya kolşisine intoleranslı AAA hastalarında umut verici bir tedavi seçeneği olabilir.

Makale detayları

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

Yazarlar:

Tuğba Ocak | Ayşe Bulur | Burcu Yağız | Belkis Nihan Coskun | Hüseyin Ediz Dalkılıç | Yavuz Pehlivan

Yayıncı:

Bursa Uludağ University

Ana Başlıklar:

Romatoloji ve Artrit

Dergi:

Uludağ Üniversitesi Tıp Fakültesi Dergisi Vol.51 Issue 1, pp.87-92

Belge Tipi:

Makale

Yayın Tarihi:

5/2025

Belge Dili:

İngilizce

Document ID (Org No.):

2001349001

pISSN:

1300-414X

Canakinumab

Colchicine intolerant

Colchicine resistant

Familial Mediterranean feverKanakinumab

Kolşsin intolreansı

Kolşissin direnci

Ailevi Akdeniz ateşi

Sayı

Makale detayları

Yazarlar: Tuğba Ocak | Ayşe Bulur | Burcu Yağız | Belkis Nihan Coskun | Hüseyin Ediz Dalkılıç | Yavuz Pehlivan

Yayıncı: Bursa Uludağ University

Ana Başlıklar: Romatoloji ve Artrit

Dergi: Uludağ Üniversitesi Tıp Fakültesi Dergisi Vol.51 Issue 1, pp.87-92

Belge Tipi: Makale

Yayın Tarihi: 5/2025

Belge Dili: İngilizce

Document ID (Org No.): 2001349001

pISSN: 1300-414X

Sayı

Yazarlar:

Tuğba Ocak | Ayşe Bulur | Burcu Yağız | Belkis Nihan Coskun | Hüseyin Ediz Dalkılıç | Yavuz Pehlivan

Yayıncı:

Bursa Uludağ University

Ana Başlıklar:

Romatoloji ve Artrit

Dergi:

Uludağ Üniversitesi Tıp Fakültesi Dergisi Vol.51 Issue 1, pp.87-92

Belge Tipi:

Makale

Yayın Tarihi:

5/2025

Belge Dili:

İngilizce

Document ID (Org No.):

2001349001

pISSN:

1300-414X