Current treatment recommendations and considerations for cryopyrin-associated periodic syndrome

Expert Rev Clin Immunol. 2015;11(10):1083-92. doi: 10.1586/1744666X.2015.1077702. Epub 2015 Aug 27.

Abstract

Cryopyrin-associated periodic syndrome (CAPS) encompasses a spectrum of three phenotypes of increasing severity. The syndrome is due to dominant mutations in NLRP3, which encodes a key component of the innate immunity that regulates the secretion of IL-1β. CAPS manifests as systemic inflammation, which compromises quality of life and leads to serious complications and handicap. Anti-IL-1 drugs have shown remarkable efficacy in treating CAPS symptoms and have significantly changed patients' lives. They have acceptable safety profiles but do have some differences. We review three drugs that are currently marketed for CAPS, give additional information for the practical use of these drugs, and provide some recommendations for management.

Keywords: Muckle–Wells syndrome; anakinra; autoinflammatory diseases; canakinumab; chronic infantile neurological cutaneous and articular syndrome; cryopyrin-associated periodic syndrome; familial cold autoinflammatory syndrome; interleukin 1; neonatal-onset multisystem inflammatory disease (NOMID); recommendations; rilonacept; treatment.

Publication types

  • Review

MeSH terms

  • Animals
  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Humanized
  • Carrier Proteins / genetics*
  • Cryopyrin-Associated Periodic Syndromes / drug therapy*
  • Cryopyrin-Associated Periodic Syndromes / genetics
  • Humans
  • Immunity, Innate / genetics
  • Interleukin 1 Receptor Antagonist Protein / therapeutic use*
  • Interleukin-1beta / antagonists & inhibitors
  • Interleukin-1beta / immunology*
  • Mutation / genetics
  • NLR Family, Pyrin Domain-Containing 3 Protein
  • Receptors, Interleukin-1 / antagonists & inhibitors
  • Recombinant Fusion Proteins / therapeutic use*
  • Treatment Outcome

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Carrier Proteins
  • Interleukin 1 Receptor Antagonist Protein
  • Interleukin-1beta
  • NLR Family, Pyrin Domain-Containing 3 Protein
  • NLRP3 protein, human
  • Receptors, Interleukin-1
  • Recombinant Fusion Proteins
  • canakinumab
  • rilonacept