Conventional and novel therapeutic options in children with familial Mediterranean fever: A rare autoinflammatory disease
Poddighe D. Romano M. Garcia-Bournissen F. Demirkaya E.
June 2022John Wiley and Sons Inc
British Journal of Clinical Pharmacology
2022#88Issue 62484 - 2499 pp.
Familial Mediterranean fever (FMF) is the most common monogenic autoinflammatory disease and is usually diagnosed in childhood, especially in the first decade of life. Paediatric 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 paediatric FMF; however, if children develop colchicine intolerance and/or resistance, biologics, particularly interleukin-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 paediatric FMF.
anakinra , canakinumab , children , colchicine , EMA , familial Mediterranean fever , FDA , interleukin-1 antagonists , rilonacept
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Department of Medicine, Nazarbayev University School of Medicine, Nur-Sultan, Kazakhstan
Clinical Academic Department of Pediatrics, National Research Center of Maternal and Child Health, University Medical Center, Nur-Sultan, Kazakhstan
Schulich School of Medicine & Dentistry, Department of Pediatrics, Division of Pediatric Rheumatology, University of Western Ontario, London, ON, Canada
Schulich School of Medicine & Dentistry, Department of Pediatrics, Canadian Behcet and Autoinflammatory Center (CAN BE AID), University of Western Ontario, London, ON, Canada
Schulich School of Medicine & Dentistry, Department of Pediatrics, Division of Pharmacology, University of Western Ontario, London, ON, Canada
Schulich School of Medicine & Dentistry, Department of Epidemiology and Biostatistics, University of Western Ontario, London, ON, Canada
Department of Medicine
Clinical Academic Department of Pediatrics
Schulich School of Medicine & Dentistry
Schulich School of Medicine & Dentistry
Schulich School of Medicine & Dentistry
Schulich School of Medicine & Dentistry
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