Abstract
The remarkable effectiveness of interleukin (IL)-1β blockade in treating the cryopyrin-associated periodic syndromes (CAPS), which present with unexplained fevers and severe localized inflammation, has provided strong evidence for a direct role of IL-1β in the pathogenesis of these diseases. Since 2003, patients with CAPS have been treated with anakinra, a recombinant nonglycosylated human IL-1 receptor antagonist. The short half-life of anakinra, however, necessitates daily injections; therefore, new IL-1β antagonists with longer half-lives, such as the fusion protein rilonacept (IL-1 Trap), have been developed. In an open-label pilot study of rilonacept, five patients with CAPS showed sustained responses and the agent was well tolerated, whilst a phase III clinical study in a large number of patients demonstrated the drug's safety and clinical efficacy. Rilonacept is the first therapy approved by the FDA for the treatment of CAPS and offers a new alternative to anakinra. Further trials are necessary to assess the long-term safety and efficacy of rilonacept.
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Church, L., McDermott, M. Rilonacept in cryopyrin-associated periodic syndromes: the beginning of longer-acting interleukin-1 antagonism. Nat Rev Rheumatol 5, 14–15 (2009). https://doi.org/10.1038/ncprheum0959
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DOI: https://doi.org/10.1038/ncprheum0959
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