Autoinflammatory diseases (AIDs) are a heterogeneous disorders caused by overproduction of interleukin (IL)-1. Appropriate treatment is crucial, also considering that AIDs may persist into adulthood with negative consequences on patients’ quality of life. IL-1 blockade results in a sustained reduction of disease severity in most AIDs. A growing experience with the human IL-1 receptor antagonist anakinra (ANA) and the monoclonal anti IL-1 antibody canakinumab (CANA) has been engendered, highlighting their efficacy upon protean clinical manifestations of AIDs. Safety and tolerability have been confirmed by several clinical trials and observational studies on both large and small cohorts of AID patients. The same treatment has been proposed in refractory Kawasaki disease, an acute inflammatory vasculitis occurring in children before 5 years which has been postulated to be autoinflammatory for its phenotypical and immunological similarity with systemic juvenile idiopathic arthritis. Nevertheless, minor concerns about IL-1 antagonists have been raised regarding their employment in children, and the development of novel pharmacological formulations is aimed at minimizing side effects that may affect adherence to treatment. The present review summarizes all current findings on the efficacy, safety and tolerability of ANA and CANA for treatment of AIDs and Kawasaki vasculitis with specific focusing on pediatric setting.
Bettiol, A., Lopalco, G., Emmi, G., Cantarini, L., Urban, M., Vitale, A., Denora, N., Lopalco, A., Cutrignelli, A., Lopedota, A., Venerito, V., Fornaro, M., Vannacci, A., Rigante, D., Cimaz, R., Iannone, F., Unveiling the efficacy, safety, and tolerability of anti-interleukin-1 treatment in monogenic and multifactorial autoinflammatory diseases, <<INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES>>, 2019; 2019 (20(8)): 1-10. [doi:10.3390/ijms20081898] [http://hdl.handle.net/10807/135288]
Unveiling the efficacy, safety, and tolerability of anti-interleukin-1 treatment in monogenic and multifactorial autoinflammatory diseases
Rigante, Donato;
2019
Abstract
Autoinflammatory diseases (AIDs) are a heterogeneous disorders caused by overproduction of interleukin (IL)-1. Appropriate treatment is crucial, also considering that AIDs may persist into adulthood with negative consequences on patients’ quality of life. IL-1 blockade results in a sustained reduction of disease severity in most AIDs. A growing experience with the human IL-1 receptor antagonist anakinra (ANA) and the monoclonal anti IL-1 antibody canakinumab (CANA) has been engendered, highlighting their efficacy upon protean clinical manifestations of AIDs. Safety and tolerability have been confirmed by several clinical trials and observational studies on both large and small cohorts of AID patients. The same treatment has been proposed in refractory Kawasaki disease, an acute inflammatory vasculitis occurring in children before 5 years which has been postulated to be autoinflammatory for its phenotypical and immunological similarity with systemic juvenile idiopathic arthritis. Nevertheless, minor concerns about IL-1 antagonists have been raised regarding their employment in children, and the development of novel pharmacological formulations is aimed at minimizing side effects that may affect adherence to treatment. The present review summarizes all current findings on the efficacy, safety and tolerability of ANA and CANA for treatment of AIDs and Kawasaki vasculitis with specific focusing on pediatric setting.
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