Abstract
Autoinflammatory diseases (AIDs) are heterogeneous disorders characterized by dysregulation in the inflammasome, a large intracellular multiprotein platform, leading to overproduction of interleukin-1(IL-1)β that plays a predominant pathogenic role in such diseases. Appropriate treatment is crucial, also considering that AIDs may persist into adulthood with negative consequences on patients’ quality of life. IL-1β 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-1β antibody, Canakinumab (CANA), has also 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 current findings on the efficacy, safety, and tolerability of ANA and CANA for treatment of AIDs and Kawasaki vasculitis with a specific focus on the pediatric setting.
Highlights
Autoinflammatory diseases (AIDs) are a heterogeneous group of monogenic and multifactorial diseases characterized by recurrent or chronic inflammation caused by dysregulation of the innate immune system [1,2]
An autoinflammatory origin has been suggested for Kawasaki disease (KD), an acute self-limited systemic vasculitis usually occurring in children before 5 years and involving medium-sized vessels, especially coronary arteries, which represents the first cause of childhood-acquired heart disease in developed countries [10]
We aimed at providing current findings on the efficacy, safety, and tolerability of Anakinra (ANA) and Canakinumab (CANA) for treatment of AIDs and Kawasaki vasculitis with a specific focus on the pediatric setting
Summary
Autoinflammatory diseases (AIDs) are a heterogeneous group of monogenic and multifactorial diseases characterized by recurrent or chronic inflammation caused by dysregulation of the innate immune system [1,2]. Most of these disorders have an early onset, ranging from the first h to the first decade of life. The main subgroup of AIDs includes different hereditary periodic fever syndromes, such as cryopyrin-associated periodic syndrome (CAPS), tumor necrosis factor receptor-associated periodic fever syndrome (TRAPS), hyperimmunoglobulin D syndrome/mevalonate kinase deficiency (HIDS/MKD), and familial Mediterranean fever (FMF) [2] These conditions follow an autosomal dominant (CAPS and TRAPS) or recessive (HIDS/MKD and FMF) hereditary pattern and share a common clinical background marked by recurrent fever attacks and inflammation involving different sites, such as skin, serosal membranes, joints, gastrointestinal tract, or central nervous system [4,5]. We aimed at providing current findings on the efficacy, safety, and tolerability of Anakinra (ANA) and Canakinumab (CANA) for treatment of AIDs and Kawasaki vasculitis with a specific focus on the pediatric setting
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