{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T11:59:16Z","timestamp":1790251156866,"version":"4.1.0"},"reference-count":20,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2009,12,28]],"date-time":"2009-12-28T00:00:00Z","timestamp":1261958400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Arthritis &amp; Rheumatism"],"published-print":{"date-parts":[[2010,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Cryopyrin\u2010associated periodic syndromes (CAPS) are a group of rare autoinflammatory diseases. Neonatal\u2010onset multisystem inflammatory disease (NOMID)\/chronic infantile neurologic, cutaneous, articular syndrome (CINCA syndrome) is the most severe phenotype, with fever, rash, articular manifestations, and neurologic and neurosensory involvement. CAPS are caused by mutations in <jats:italic>CIAS1<\/jats:italic>, the gene encoding NLRP3, which plays a critical role in interleukin\u20101 (IL\u20101) processing. Anakinra, an IL\u20101 receptor antagonist, has been shown to be an effective treatment; however, data on long\u2010term efficacy and safety have been sparse. This study was undertaken to assess the long\u2010term efficacy and safety of anakinra treatment in patients with NOMID\/CINCA syndrome.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We retrospectively analyzed the medical records of NOMID\/CINCA syndrome patients referred to 2 centers, who had started anakinra treatment before June 2007.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>There were 10 patients with NOMID\/CINCA syndrome who had been treated with anakinra. The patients' ages at the time anakinra treatment was initiated ranged from 3 months to 20 years. They had been followed up for 26\u201342 months. Sustained efficacy in the treatment of systemic inflammation and, in some cases, neurologic involvement and growth parameters, was achieved. The dosage of anakinra required for efficacy ranged from 1 to 3 mg\/kg\/day in the 8 oldest patients and from 6 to 10 mg\/kg\/day in the 2 youngest. Residual central nervous system inflammation and deafness persisted in some patients, especially if there had been a delay in diagnosis and treatment. Secondary amyloidosis persisted in cases in which it was present at treatment initiation, but no new lesions developed. No effect on overgrowth arthropathy was observed. Adverse events consisted of mild injection\u2010site reactions.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The present results indicate that anakinra treatment is effective over the long term in NOMID\/CINCA syndrome. However, treatment has to be initiated before irreversible lesions develop, and, particularly in very young patients, dosage adjustment is required.<\/jats:p><\/jats:sec>","DOI":"10.1002\/art.25057","type":"journal-article","created":{"date-parts":[[2009,12,28]],"date-time":"2009-12-28T21:08:57Z","timestamp":1262034537000},"page":"258-267","source":"Crossref","is-referenced-by-count":240,"title":["Long\u2010term efficacy of the interleukin\u20101 receptor antagonist anakinra in ten patients with neonatal\u2010onset multisystem inflammatory disease\/chronic infantile neurologic, cutaneous, articular syndrome"],"prefix":"10.1002","volume":"62","author":[{"given":"B\u00e9n\u00e9dicte","family":"Neven","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Marvillet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Celine","family":"Terrada","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Ferster","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathalie","family":"Boddaert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincent","family":"Couloignier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Graziella","family":"Pinto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne","family":"Pagnier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"Bodemer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bahram","family":"Bodaghi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"Tardieu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne Marie","family":"Prieur","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Quartier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2009,12,28]]},"reference":[{"key":"e_1_2_7_2_2","first-page":"103","article-title":"A recently recognised chronic inflammatory disease of early onset characterised by the triad of rash, central nervous system involvement and arthropathy","volume":"19","author":"Prieur AM","year":"2001","journal-title":"Clin Exp Rheumatol"},{"key":"e_1_2_7_3_2","doi-asserted-by":"publisher","DOI":"10.1038\/ng756"},{"key":"e_1_2_7_4_2","doi-asserted-by":"publisher","DOI":"10.1086\/341357"},{"key":"e_1_2_7_5_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.10688"},{"key":"e_1_2_7_6_2","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M401178200"},{"key":"e_1_2_7_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1074-7613(04)00046-9"},{"key":"e_1_2_7_8_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200306193482523"},{"key":"e_1_2_7_9_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.20033"},{"key":"e_1_2_7_10_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(04)17401-1"},{"key":"e_1_2_7_11_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.20497"},{"key":"e_1_2_7_12_2","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/keh547"},{"key":"e_1_2_7_13_2","doi-asserted-by":"publisher","DOI":"10.1080\/03009740410010236"},{"key":"e_1_2_7_14_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.20953"},{"key":"e_1_2_7_15_2","doi-asserted-by":"publisher","DOI":"10.1001\/archderm.142.12.1591"},{"key":"e_1_2_7_16_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa055137"},{"key":"e_1_2_7_17_2","doi-asserted-by":"publisher","DOI":"10.1136\/ard.2004.032060"},{"key":"e_1_2_7_18_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.21965"},{"key":"e_1_2_7_19_2","doi-asserted-by":"publisher","DOI":"10.1359\/jbmr.071002"},{"key":"e_1_2_7_20_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.23620"},{"key":"e_1_2_7_21_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.23687"}],"container-title":["Arthritis &amp; 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