Critical appraisal of canakinumab in the treatment of adults and children with cryopyrin-associated periodic syndrome (CAPS)

Ori Toker¹, Philip J Hashkes²

¹Department of Pediatrics, Shaare Zedek Medical Center, affiliated with the Hadassah-Hebrew University Medical School, Jerusalem, Israel; ²Pediatric Rheumatology Unit, Shaare Zedek Medical Center, Jerusalem, Israel, Cleveland Clinic Lerner Medical School, Case Western Reserve University, Cleveland, OH, USA

Abstract: The cryopyrin-associated syndromes (CAPS) include three autosomal-dominant syndromes, that are caused by a mutation in the NLRP3 gene on chromosome 1, encoding the cryopyrin protein. These syndromes, familial cold autoinflammatory syndrome, Muckle-Wells syndrome and neonatal-onset multisystem inflammatory disease, are characterized by urticaria-like rash, fever, central nervous system inflammation, an arthropathy and a risk of the development of amyloidosis in a respectively escalating degree of severity between the various syndromes. Recently the role of cryopyrin in the regulation of interleukin (IL)-1 production and activation was described and anti IL-1 therapies were found to be very effective in treating these syndromes. There are several types of anti IL-1 medications based on different mechanisms of antagonizing IL-1. This paper focuses on the efficacy and safety of canakinumab, a long-acting humanized anti IL-1 antibody, in treating these syndromes.

Keywords: canakinumab, cryopyrin-associated periodic syndromes, biologics, treatment, autoinflammatory diseases