Nienke M Ter Haar1, Joost Frenkel. 1. aDepartment of Pediatric Rheumatology bDepartment of General Pediatrics, University Medical Center Utrecht, Utrecht, The Netherlands.
Abstract
PURPOSE OF REVIEW: The purpose of this review is to summarize recent advances in the treatment of the hereditary autoinflammatory diseases, focusing on Familial Mediterranean fever (FMF), tumor necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS) and mevalonate kinase deficiency (MKD). We discuss recently published studies and their implications for current patient care and future clinical research. RECENT FINDINGS: Interleukin (IL)-1 blockade is effective in most autoinflammatory conditions. Younger patients require a higher dose per kg of body weight. In FMF, colchicine remains the treatment of choice. Single daily dosing appears adequate. When colchicine fails, IL-1 blockade is effective. In CAPS, the beneficial effect of IL-1 blockade is sustained, and side-effects are limited. There is no evidence that one IL-1 blocker is superior to the other. In TRAPS and MKD, IL-1 blockade appears effective. Some patients have sufficient suppression of inflammatory symptoms with NSAIDs or corticosteroids. SUMMARY: Apart from CAPS and FMF, therapy of autoinflammatory diseases is based on small and retrospective studies. IL-1 blockade appears effective, but larger prospective trials are needed, especially in MKD, TRAPS and colchicine-resistant FMF patients.
PURPOSE OF REVIEW: The purpose of this review is to summarize recent advances in the treatment of the hereditary autoinflammatory diseases, focusing on Familial Mediterranean fever (FMF), tumornecrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS) and mevalonate kinase deficiency (MKD). We discuss recently published studies and their implications for current patient care and future clinical research. RECENT FINDINGS:Interleukin (IL)-1 blockade is effective in most autoinflammatory conditions. Younger patients require a higher dose per kg of body weight. In FMF, colchicine remains the treatment of choice. Single daily dosing appears adequate. When colchicine fails, IL-1 blockade is effective. In CAPS, the beneficial effect of IL-1 blockade is sustained, and side-effects are limited. There is no evidence that one IL-1 blocker is superior to the other. In TRAPS and MKD, IL-1 blockade appears effective. Some patients have sufficient suppression of inflammatory symptoms with NSAIDs or corticosteroids. SUMMARY: Apart from CAPS and FMF, therapy of autoinflammatory diseases is based on small and retrospective studies. IL-1 blockade appears effective, but larger prospective trials are needed, especially in MKD, TRAPS and colchicine-resistant FMFpatients.
Authors: Hella Luksch; Stefan Winkler; Michael C Heymann; Felix Schulze; Sigrun R Hofmann; Joachim Roesler; Angela Rösen-Wolff Journal: Curr Rheumatol Rep Date: 2015-07 Impact factor: 4.592