title: Targeting interleukin-1β in CAPS (cryopyrin-associated periodic) syndromes: what did we learn?
abstract: CAPS is the prototype of an IL-1β driven auto inflammatory disorder. Features of recurrent systemic inflammation compromises patient's quality of life, and may reduce life expectancy by inducing definite organ damage. Recent treatment targeting IL-1 have shown dramatic effect on patient's clinical symptoms and quality of life. Anakinra was first used successfully in treating small series of patients with all CAPS phenotypes. Two pivotal randomized placebo-controlled studies allowed licensing of rilonacept and canakinumab as orphan drugs for CAPS patients. The use of anti-IL-1 drugs in CAPS is still relatively new, and their effect on a long term is not well known. As we can suppress the clinical symptoms of patients with CAPS, important questions remain regarding the full effect of anti-IL-1 treatment on organ involvement and their potential ability to prevent them. As important variations of treatment doses and schedules appear in reaching effectiveness, pharmacologic studies are still warranted to determine a potential diffusion of anti-IL-1 drugs in the fluids and tissues. More studies evaluating the efficacy and safety of anti-IL-1 drugs given in patients before 2 years of age are warranted, since it is believed that the earliest treatment could avoid secondary CAPS complications to develop.
authors.affiliation: Division of Pediatric Rheumatology and Division of Pediatrics, CEREMAI, University of Paris SUD, Bicêtre Hospital, Assistance Publique Hôpitaux de Paris, Le Kremlin Bicêtre, Paris, France. isabelle.kone-paut@bct.aphp.fr
authors.forename: Isabelle
authors.initials: I
authors.lastname: Koné-Paut
authors.forename: Maryam
authors.initials: M
authors.lastname: Piram
mesh_terms: D056587:Cryopyrin-Associated Periodic Syndromes / Q000188:drug therapy*; D006801:Humans; D007155:Immunologic Factors / Q000009:adverse effects* / Q000627:therapeutic use; D053590:Interleukin 1 Receptor Antagonist Protein / Q000009:adverse effects* / Q000627:therapeutic use; D053583:Interleukin-1beta / Q000037:antagonists & inhibitors*; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic
chemical_list: D007155:Immunologic Factors; D053590:Interleukin 1 Receptor Antagonist Protein; D053583:Interleukin-1beta