Vol.:(0123456789) 1 3 Rheumatology International https://doi.org/10.1007/s00296-018-3993-5 OBSERVATIONAL RESEARCH Canakinumab treatment in children with familial Mediterranean fever: report from a single center Fatma Yazılıtaş 1  · Özlem Aydoğ 2  · Sare Gülfem Özlü 3  · Evrim Kargın Çakıcı 1  · Tülin Güngör 4  · Fehime Kara Eroğlu 4  · Gökçe Gür 4  · Mehmet Bülbül 4 Received: 20 November 2017 / Accepted: 5 February 2018 © Springer-Verlag GmbH Germany, part of Springer Nature 2018 Abstract Familial Mediterranean fever (FMF), the most common hereditary autoinflammatory disorder is characterized by recurrent episodes of fever, serositis, arthritis. The major long-term result is amyloidosis. Colchicine remains the principle of the treatment; it not only prevents the acute attacks but also prevents the long-term complications such as amyloidosis; 5–10% of the patients are unresponsive to treatment. Recently new therapeutic options as anti-interleukin 1 agents are successfully used for the patients who do not respond to colchicine treatment. In this study, we retrospectively evaluated 11 pediatric colchicine-resistant FMF patients who were treated with canakinumab. Three of the patients had amyloidosis and two had uveitis. Based on our results, we suggest that canakinumab may be a safe and effective therapy in patients who are resistant to colchicine and even in the patients with amyloidosis. We also suggest that canakinumab might be a safe option for the patients with uveitis. Keywords Amyloidosis · Canakinumab · Childhood · Familial Mediterranean fever · Uveitis Introduction Familial Mediterranean fever (FMF) is the most common monogenic systemic autoinflammatory disease [1]. FMF is caused by point mutations in the Mediterranean Fever (MEFV) gene located on the short arm of chromosome 16 [2]. The mutated gene encodes the protein pyrin (also known as marenostrin), which is expressed in neutrophils, eosino- phils, monocytes, dendritic cells, and synovial fibroblasts [3–5]. Pyrin mediates regulation of caspase-1 activation and NLRP3 inflammasome, an intracellular complex required for conversion of precursor IL-1β (pro IL-1β) into mature IL-1β [6–9]. IL-1β is a cytokine released by cells of the immune system that have potent inflammatory and immu- nomodulatory effects, and is the key mediator of the immune response. IL-1β plays a role in T-cell activation, chemot- axis of polymorphonuclear leukocytes and monocytes, release of proteases from tissue macrophages, stimulation Rheumatology INTERNATIONAL * Fatma Yazılıtaş fmeryemesra@yahoo.com Özlem Aydoğ ozlem_erdogan65@yahoo.com Sare Gülfem Özlü saredr@gmail.com Evrim Kargın Çakıcı evrimkargin@gmail.com Tülin Güngör tulingungor84@gmail.com Fehime Kara Eroğlu fehimekara@yahoo.com Gökçe Gür dr.gokcecan@gmail.com Mehmet Bülbül mbbjkank@yahoo.com.tr 1 Pediatric Nephrology Department, Ankara Dr. Sami Ulus Maternity and Children Hospital, Babur Caddesi No. 44, Altındağ, 06080 Ankara, Turkey 2 Pediatric Nephrology and Rheumatology Department, Ondokuz Mayıs University, Samsun, Turkey 3 Pediatric Nephrology Department, Medical School, Yildirim Beyazit University, Yenimahalle, Ankara, Turkey 4 Pediatric Nephrology and Rheumatology Department, Ankara Dr. Sami Ulus Maternity and Children Hospital, Babur Caddesi No. 44, Altındağ, Ankara, Turkey