Conventional and Novel Therapeutic Options in Children with Familial Mediterranean Fever: A Rare Autoinflammatory Disease.
2021
Familial Mediterranean Fever (FMF) is the most common monogenic autoinflammatory disease and is usually diagnosed in childhood, especially in the first decade of life. Pediatric FMF is characterized by a protean clinical expression and a variable therapeutic response, which can make its medical management very challenging. However, even if long-term complications of untreated FMF (e.g. amyloidosis and related organ damage) are less frequent in children compared to adults, they are not uncommon. Colchicine is the mainstay of the therapy in pediatric FMF; however, if children develop colchicine intolerance and/or resistance, biologics, particularly IL-1 antagonists, must be considered. Other conventional or biological therapeutic options do not currently have appropriate evidence-based support, except for some specific clinical presentations (e.g., arthritis). In this review, we discuss the biological basis and the clinical evidence for the current pharmacological treatment options available for pediatric FMF.
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