β-Confident-registry: aiming to be largest-ever studied cohort of cryopyrin-associated periodic syndromes (CAPS) patients. Study design and baseline characteristics
نویسندگان
چکیده
Methods CAPS patients receiving canakinumab as part of their routine care are included in this study for a minimum of 5-years follow-up. Data from routine clinic assessments is supplied at 6-monthly intervals via a web-based application. Selected safety events potentially associated with anti-IL-1 therapy such as serious infections, malignancies, hypersensitivity and disease activity/progression is analyzed. Signs and symptoms of systemic inflammation, neurologic and ophthalmologic status and the
منابع مشابه
Long-term safety and efficacy of Canakinumab in cryopyrin-associated periodic syndrome (CAPS) patients: results from beta-confident registry
Background CAPS encompasses a spectrum of three phenotypes: familial cold auto-inflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and chronic infantile neurologic cutaneous and articular syndrome/neonatal onset multisystem inflammatory disease (CINCA/ NOMID)[1]. The b-Confident Registry, the largest CAPS cohort documented in a registry, enrolled the last patient in December 2014. Here, ...
متن کاملAn experience of use of canakinumab IL-1 inhibitor in children with cryopyrin-associated periodic syndromes (CAPS)
Introduction Interleukin-1b (IL-1b) is a basic mediator of cryopyrinassociated periodic syndromes (CAPS). In this respect an experience of the use of IL-1 inhibitors has been gained in patients with CAPS. Canakinumab was approved by FDA and EMEA in 2009 and registered in the Russian Federation in 2011 for treatment of CAPS. Canakinumab is shown to have high efficiency and good tolerability in p...
متن کاملThe role of somatic NLRP3 mosaicism and new gene discovery in mutation negative cryopyrin-associated periodic syndrome patients
Introduction Cryopyrin associated periodic syndromes (CAPS) are caused by autosomal dominant gain of function mutations in the NLRP3 gene. However, up to 50% of clinically diagnosed CAPS patients with typical clinical features and good response to anti-IL-1b treatment have no mutation detected by conventional Sanger DNA sequencing. Recent studies suggest that somatic NLRP3 mosaicism may account...
متن کاملRilonacept in the management of cryopyrin-associated periodic syndromes (CAPS)
Cryopyrin-associated periodic syndromes (CAPS) are a subgroup of the hereditary periodic fever syndromes, which are rare autoinflammatory and inherited disorders, characterized by recurrent inflammation and varying degrees of severity. CAPS are thought to be driven by excessive production of interleukin-1β (IL-1β), through over-activation of the inflammasome by gain of function mutations in the...
متن کاملExpanding spectrum of neurologic manifestations in patients with NLRP3 low-penetrance mutations
OBJECTIVE To evaluate the frequency of the cryoporin/NLRP3 low-penetrance mutations V198M and Q703K in patients who reported at least 2 symptoms compatible with cryopyrin-associated periodic syndromes (CAPS) and to characterize the phenotype in mutation-positive patients. METHODS The frequency of the V198M and Q703K mutations was investigated in a selected cohort of 108 patients from our neur...
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عنوان ژورنال:
دوره 9 شماره
صفحات -
تاریخ انتشار 2011