Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)

Leigh D Church1, Sinisa Savic2, Michael F McDermott21Department of Rheumatology, Division of Immunity, Infection and Inflammation, Institute for Biomedical Research, The University of Birmingham, Birmingham, UK; 2Section of Musculoskeletal Disease, Leeds Institute of Molecular Medicine, Wellcome Tru...

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Autores principales:	Leigh D Church, Sinisa Savic, Michael F McDermott
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2008
Materias:	Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/7c1d1dc2c87d46f3a90f0e2b1d4596ab
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spelling	oai:doaj.org-article:7c1d1dc2c87d46f3a90f0e2b1d4596ab2021-12-02T02:33:31ZLong term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)1177-54751177-5491https://doaj.org/article/7c1d1dc2c87d46f3a90f0e2b1d4596ab2008-10-01T00:00:00Zhttp://www.dovepress.com/long-term-management-of-patients-with-cryopyrin-associated-periodic-sy-a2476https://doaj.org/toc/1177-5475https://doaj.org/toc/1177-5491Leigh D Church1, Sinisa Savic2, Michael F McDermott21Department of Rheumatology, Division of Immunity, Infection and Inflammation, Institute for Biomedical Research, The University of Birmingham, Birmingham, UK; 2Section of Musculoskeletal Disease, Leeds Institute of Molecular Medicine, Wellcome Trust Brenner Building, University of Leeds, Leeds, UKAbstract: Cryopyrin-associated periodic syndromes (CAPS) are a group of inherited inflammatory disorders consisting of familial cold-induced autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and neonatal-onset multisystem inflammatory disease (NOMID; also known as chronic infantile neurologic, cutaneous, articular [CINCA] syndrome). These rare disorders are associated with heterozygous mutations in the NLRP3 (CIAS1) gene, which encodes the protein NALP3 or cryopyrin, and inflammation driven by excessive production of the cytokine interleukin-1&beta; (IL-1&beta;). Amyloidosis is a serious complication with 25% of MWS patients developing amyloidosis, with occasional fatal consequences, whilst up to 20% of CINCA/NOMID patients die from various complications, before reaching the early adulthood. In some CINCA/NOMID adult survivors amyloidosis can also occur. Prior to the discovery of the CIAS1 gene mutations and the advent of IL-1 targeted therapy, treatment was aimed at suppressing inflammation, with limited success. The selective blockade of IL-1&beta;, with anakinra (IL-1 receptor antagonist), not only provided supportive evidence for the role of IL-1&beta; in CAPS, but also demonstrated the efficacy of targeting IL-1&beta; for treatment of these conditions. In February, 2008, &lsquo;Orphan Drug&rsquo; approval from the Food and Drug Administration (FDA) for rilonacept (IL-1 Trap/Arcalyst&trade;, Regeneron Pharmaceuticals, Inc) was given for the treatment of two CAPS disorders, FCAS and MWS in adults and children 12 years and older, making rilonacept the first therapy approved for the treatment of CAPS.Keywords: cryopyrin-associated periodic syndromes, interleukin-1, therapy, rilonacept Leigh D ChurchSinisa SavicMichael F McDermottDove Medical PressarticleMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol 2008, Iss Issue 4, Pp 733-742 (2008)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine (General) R5-920
spellingShingle	Medicine (General) R5-920 Leigh D Church Sinisa Savic Michael F McDermott Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)
description	Leigh D Church1, Sinisa Savic2, Michael F McDermott21Department of Rheumatology, Division of Immunity, Infection and Inflammation, Institute for Biomedical Research, The University of Birmingham, Birmingham, UK; 2Section of Musculoskeletal Disease, Leeds Institute of Molecular Medicine, Wellcome Trust Brenner Building, University of Leeds, Leeds, UKAbstract: Cryopyrin-associated periodic syndromes (CAPS) are a group of inherited inflammatory disorders consisting of familial cold-induced autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and neonatal-onset multisystem inflammatory disease (NOMID; also known as chronic infantile neurologic, cutaneous, articular [CINCA] syndrome). These rare disorders are associated with heterozygous mutations in the NLRP3 (CIAS1) gene, which encodes the protein NALP3 or cryopyrin, and inflammation driven by excessive production of the cytokine interleukin-1&beta; (IL-1&beta;). Amyloidosis is a serious complication with 25% of MWS patients developing amyloidosis, with occasional fatal consequences, whilst up to 20% of CINCA/NOMID patients die from various complications, before reaching the early adulthood. In some CINCA/NOMID adult survivors amyloidosis can also occur. Prior to the discovery of the CIAS1 gene mutations and the advent of IL-1 targeted therapy, treatment was aimed at suppressing inflammation, with limited success. The selective blockade of IL-1&beta;, with anakinra (IL-1 receptor antagonist), not only provided supportive evidence for the role of IL-1&beta; in CAPS, but also demonstrated the efficacy of targeting IL-1&beta; for treatment of these conditions. In February, 2008, &lsquo;Orphan Drug&rsquo; approval from the Food and Drug Administration (FDA) for rilonacept (IL-1 Trap/Arcalyst&trade;, Regeneron Pharmaceuticals, Inc) was given for the treatment of two CAPS disorders, FCAS and MWS in adults and children 12 years and older, making rilonacept the first therapy approved for the treatment of CAPS.Keywords: cryopyrin-associated periodic syndromes, interleukin-1, therapy, rilonacept
format	article
author	Leigh D Church Sinisa Savic Michael F McDermott
author_facet	Leigh D Church Sinisa Savic Michael F McDermott
author_sort	Leigh D Church
title	Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)
title_short	Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)
title_full	Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)
title_fullStr	Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)
title_full_unstemmed	Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)
title_sort	long term management of patients with cryopyrin-associated periodic syndromes (caps): focus on rilonacept (il-1 trap)
publisher	Dove Medical Press
publishDate	2008
url	https://doaj.org/article/7c1d1dc2c87d46f3a90f0e2b1d4596ab
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Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)

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