Biological therapies in monogenic autoinflammatory diseases: long-term efficacy and safety

Introduction Autoinflammatory diseases (AIDs) represent a group of monogenic disorders, related to mutations in genes encoding proteins involved in the regulation of inflammatory responses. The aims of therapy in AIDs patients are controlling disease activity, improving patient’s quality of life and preventing long-term complications. Biological agents have recently changed the natural history and the prognosis of AIDs. According to a pathogenetic criterium, they can be classified as: 1 ) Anti-TNF agents: i) Etanercept, a dimeric fusion protein of TNFR2 linked with the Fc region of human IgG1, ii) Adalimumab, a fully human monoclonal antiTNF antibody, iii) Infliximab, a mouse/human chimeric monoclonal anti-TNF antibody [1,2]. 2) Anti-IL-1 agents: i) Anakinra, a competitive IL-1 receptor antagonist, ii) Canakinumab, a fully humanized anti-IL-1 monoclonal antibody, iii) Rilonacept, a fusion protein of IL-1 extracellular domains and Fc region of human IgG [1]. 3) Anti-IL-6 agents: Tocilizumab, a monoclonal antibody binding and inhibiting both soluble and membrane IL-6 receptor [1,2].