Current options for the treatment of cryopyrin-associated periodic syndromes. (September 2013)

• Record Type: Journal Article
• Authors: Galeotti, Caroline; Koné-Paut, Isabelle
• Is Part Of: Expert opinion on orphan drugs. Volume 1:Number 8(2013:Aug.)
• Page Start: 589
• Page End: 597
• Publication Date: 2013-09
• Subjects: anakinra -- canakinumab -- chronic infantile neurologic cutaneous articular/neonatal onset multi-system inflammatory disorder -- cryopyrin-associated periodic syndromes -- familial cold auto-inflammatory syndrome -- inflammasome -- IL-1 -- Muckle–Wells -- rilonacept; Orphan drugs -- Periodicals; Rare diseases -- Periodicals; Chemotherapy -- Periodicals; 615.1
• DOI: 10.1517/21678707.2013.821946
• Languages: English
• ISSNs: 2167-8707

Abstract

Abstract : Introduction: Cryopyrin-associated periodic syndrome (CAPS), a rare hereditary auto-inflammatory disease, is associated with mutations in the NLRP3 gene, encoding cryopyrin, a key component of the inflammasome that activates caspase-1 and IL-1β production. Symptoms associated with CAPS are directly linked to excessive IL-1β secretion. Impressive clinical results were obtained with IL-1 blocking agents in patients with all CAPS phenotypes, which are now considered as IL-1-driven human diseases prototypes. Areas covered: Anakinra, the first biologic agent designed for the selective blockade of IL-1 in sepsis, was successfully used off-label in patients with all CAPS phenotypes. Two more recent IL-1 antagonists with longer half-life have been licensed for CAPS, rilonacept and canakinumab, both with highly favorable safety profile. Expert opinion: Clinical trials using anti-IL-1 drugs proved the dramatic effect of this strategy on both patients' symptoms and quality of life. Important variations of treatment doses and schedules appeared in reaching effectiveness. The full effect of anti-IL-1 drugs in damaged organs is still undefined. More studies evaluating their efficacy and safety in patients before 2 years are warranted, since it is believed that the earliest treatment could avoid secondary CAPS complications to develop.