National CAPS (Cryopyrin-Associated Periodic Syndrome) Registry
نویسندگان
چکیده
Patients and methods The Italian CAPS Registry started in 2004 and has currently enrolled 29 patients: 16 with Chronic Infantile Neurologic Cutaneous Articular Syndrome (CINCAs), 8 with Muckles Welles syndrome (MWS) and 5 with Familial Cold Urticaria (FCU). 16 patients were treated with Anakinra (IL-1 receptor antagonist) at 1 mg/kg/ day. The Child Health Questionnaire (CHQ-PF 50) was used to assess the health related quality of life.
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 کاملβ-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 کاملPReS-FINAL-2241: Cases of cryopyrin-associated periodic syndromes (CAPS) in one single rheumatologic center of Russia
Introduction Cryopyrin-associated periodic syndromes (CAPS) are the rare hereditary autoinflammatory diseases. CAPS include three similar conditions are distinguished which lie along a phenotypical continuum with increasing levels of severity: familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS) and CINCA/ NOMID. Distinguishing features include cutaneous, neurological, op...
متن کامل[CAPS: cryopyrin-associated periodic syndrome].
Cryopyrin-associated periodic syndrome (CAPS) is an autoinflammatory syndrome caused by heterozygous mutations of NLRP3 gene. CAPS consists of three phenotypically similar but distinct syndromes: familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome and CINCA syndrome. Among them, FCAS shows the mildest phenotype while CINCA is the severest. Common symptoms include sporadic or c...
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عنوان ژورنال:
دوره 9 شماره
صفحات -
تاریخ انتشار 2011