Long-term safety and efficacy of Canakinumab in cryopyrin-associated periodic syndrome (CAPS) patients: results from beta-confident registry
نویسندگان
چکیده
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, we report interim data for the complete cohort of enrolled patients.
منابع مشابه
β-Confident-registry: aiming to be largest-ever studied cohort of cryopyrin-associated periodic syndromes (CAPS) patients. Study design and baseline characteristics
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/progressio...
متن کاملMethodological challenges in monitoring new treatments for rare diseases: lessons from the cryopyrin-associated periodic syndrome registry
BACKGROUND The Cryopyrin-Associated Periodic Syndromes (CAPS) are a group of rare hereditary autoinflammatory diseases and encompass Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), and Neonatal Onset Multisystem Inflammatory Disease (NOMID). Canakinumab is a monoclonal antibody directed against IL-1 beta and approved for CAPS patients but requires post-approval moni...
متن کاملCanakinumab (ILARIS®) in cryopyrin-associated periodic syndrome (CAPS) patients below 2 years of age: Review of four cases and literature.
Curr Pediatr Res 2017 Volume 21 Issue 4 646 Cryopyrin-Associated Periodic Syndrome (CAPS) is combination of different auto-inflammatory hereditary disorders with same genetic basis. CAPS caused mostly due to mutation of NLRP3 gene (nucleotide-binding domain, leucine-rich family (NLR), pyrin domain containing 3), which is an early onset in children. There are many anti-INF agents, anti-IL blocke...
متن کاملCanakinumab (ACZ885, a fully human IgG1 anti-IL-1β mAb) induces sustained remission in pediatric patients with cryopyrin-associated periodic syndrome (CAPS)
INTRODUCTION Cryopyrin-associated periodic syndrome (CAPS) represents a spectrum of three auto-inflammatory syndromes, familial cold auto-inflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and neonatal-onset multisystem inflammatory disease/chronic infantile neurological cutaneous and articular syndrome (NOMID/CINCA) with etiology linked to mutations in the NLRP3 gene resulting in eleva...
متن کاملEfficacy, safety, and post-vaccination antibody titer data in children with CAPS treated with Canakinumab
Background Canakinumab (CAN) is indicated for the treatment of cryopyrin-associated periodic syndrome (CAPS) in patients ≥2 years of age [1]. However, patients may require treatment in infancy where CAN has not yet been studied. IL-1 inhibition has not affected antibody production after vaccination in healthy volunteers [2], but no data in patients receiving standard childhood vaccines are avai...
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عنوان ژورنال:
دوره 13 شماره
صفحات -
تاریخ انتشار 2015