{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,28]],"date-time":"2026-07-28T22:51:11Z","timestamp":1785279071240,"version":"3.55.0"},"reference-count":29,"publisher":"Oxford University Press (OUP)","issue":"2","license":[{"start":{"date-parts":[[2022,5,16]],"date-time":"2022-05-16T00:00:00Z","timestamp":1652659200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008792","name":"Novartis Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008792","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023,3,2]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>To assess the real-world safety and effectiveness of canakinumab in patients in Japan with tumour necrosis factor receptor-associated periodic syndrome (TRAPS) or mevalonate kinase deficiency\/hyperimmunoglobulinaemia D with periodic fever syndrome (MKD\/HIDS).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>All patients with TRAPS or MKD\/HIDS who received canakinumab following drug approval in Japan were registered in a post-marketing all-patient surveillance with a 2-year observation period. Herein, the interim results are reported.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fifteen patients with TRAPS and seven with MKD\/HIDS were included in the safety and effectiveness analysis set. Adverse drug reactions were reported in 26.67% (n\u2009=\u20094) and 42.86% (n\u2009=\u20093) of TRAPS and MKD\/HIDS patients, respectively. Most common adverse drug reactions were upper respiratory tract inflammation (13.33%, n\u2009=\u20092) and pyrexia (42.86%, n\u2009=\u20093) in TRAPS and MKD\/HIDS patients, respectively. No serious adverse drug reactions were observed in either TRAPS or MKD\/HIDS patients. The proportion of responders was 46.67% and 14.29% in the TRAPS and MKD\/HIDS groups, respectively; 72.73% and 66.67% achieved clinical remission, while 90.91% and 66.67% achieved serological remission by Week 4 in the TRAPS and MKD\/HIDS groups, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>These interim results provide the first evidence of the real-world effectiveness of canakinumab in patients with TRAPS or MKD\/HIDS in Japan. No new safety concerns were identified.<\/jats:p><\/jats:sec>","DOI":"10.1093\/mr\/roac041","type":"journal-article","created":{"date-parts":[[2022,5,16]],"date-time":"2022-05-16T11:16:07Z","timestamp":1652699767000},"page":"381-391","source":"Crossref","is-referenced-by-count":13,"title":["Real-world safety and effectiveness of canakinumab in patients with tumour necrosis factor receptor-associated periodic syndrome or hyperimmunoglobulinaemia D syndrome: Interim results from post-marketing surveillance in Japan"],"prefix":"10.1093","volume":"33","author":[{"given":"Kumiko","family":"Hosono","sequence":"first","affiliation":[{"name":"Medical Division, Novartis Pharma K.K. , Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kazuko","family":"Matsumoto","sequence":"additional","affiliation":[{"name":"Medical Division, Novartis Pharma K.K. , Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miki","family":"Shimbo","sequence":"additional","affiliation":[{"name":"Medical Division, Novartis Pharma K.K. , Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isao","family":"Tsumiyama","sequence":"additional","affiliation":[{"name":"Clinical Development & Analytics Japan Integrated Biostatistics Japan Biostatistics Pharma, Novartis Pharma K.K. , Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chihiro","family":"Kato","sequence":"additional","affiliation":[{"name":"Clinical Development & Analytics Japan CDD and Re-examination CDD2, Novartis Pharma K.K. , Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2022,5,16]]},"reference":[{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"1129","DOI":"10.1016\/j.jaci.2009.11.001","article-title":"Therapy of autoinflammatory syndromes","volume":"124","author":"Hoffman","year":"2009","journal-title":"J Allergy Clin Immunol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1016\/j.det.2013.04.003","article-title":"Autoinflammatory diseases in pediatrics","volume":"31","author":"Hausmann","year":"2013","journal-title":"Dermatol Clin"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"2160","DOI":"10.1136\/annrheumdis-2013-204184","article-title":"The phenotype of TNF receptor-associated autoinflammatory syndrome (TRAPS) at presentation: a series of 158 cases from the Eurofever\/EUROTRAPS international registry","volume":"73","author":"Lachmann","year":"2014","journal-title":"Ann Rheum Dis"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","DOI":"10.1186\/s12969-016-0091-7","article-title":"Natural history of mevalonate kinase deficiency: a literature review","volume":"14","author":"Zhang","year":"2016","journal-title":"Pediatr Rheumatol Online J"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1097\/MD.0b013e318190cfb7","article-title":"Long-term follow-up, clinical features, and quality of life in a series of 103 patients with hyperimmunoglobulinemia D syndrome","volume":"87","author":"van der Hilst","year":"2008","journal-title":"Medicine (Baltimore)"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"2795","DOI":"10.1002\/art.39763","article-title":"The phenotype and genotype of mevalonate kinase deficiency: a series of 114 cases from the Eurofever registry","volume":"68","author":"Ter Haar","year":"2016","journal-title":"Arthritis Rheumatol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1007\/s10238-005-0071-6","article-title":"Hereditary periodic fever and reactive amyloidosis","volume":"5","author":"van der Hilst","year":"2005","journal-title":"Clin Exp Med"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"2010","DOI":"10.1002\/art.21901","article-title":"AA amyloidosis complicating hyperimmunoglobulinemia D with periodic fever syndrome: a report of two cases","volume":"54","author":"Lachmann","year":"2006","journal-title":"Arthritis Rheum"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"1957","DOI":"10.2169\/internalmedicine.54.3969","article-title":"Trapped in TRAPS: difficulty of diagnosis due to heterogeneity","volume":"54","author":"Horiuchi","year":"2015","journal-title":"Intern Med"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"2760","DOI":"10.1002\/art.39793","article-title":"Clinical and genetic features of patients with TNFRSF1A variants in Japan: findings of a nationwide survey","volume":"68","author":"Ueda","year":"2016","journal-title":"Arthritis Rheumatol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1080\/14397595.2018.1442639","article-title":"National survey of Japanese patients with mevalonate kinase deficiency reveals distinctive genetic and clinical characteristics","volume":"29","author":"Tanaka","year":"2019","journal-title":"Mod Rheumatol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1136\/annrheumdis-2011-200549","article-title":"An international registry on autoinflammatory diseases: the Eurofever experience","volume":"71","author":"Toplak","year":"2012","journal-title":"Ann Rheum Dis"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"914","DOI":"10.1038\/ni.3457","article-title":"Pyrin inflammasome activation and RhoA signaling in the autoinflammatory diseases FMF and HIDS","volume":"17","author":"Park","year":"2016","journal-title":"Nat Immunol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"462","DOI":"10.1136\/annrheumdis-2012-202774","article-title":"Increased NLRP3-dependent interleukin 1\u03b2 secretion in patients with familial Mediterranean fever: correlation with MEFV genotype","volume":"73","author":"Omenetti","year":"2014","journal-title":"Ann Rheum Dis"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"9982","DOI":"10.1073\/pnas.0602081103","article-title":"The B30.2 domain of pyrin, the familial Mediterranean fever protein, interacts directly with caspase-1 to modulate IL-1beta production","volume":"103","author":"Chae","year":"2006","journal-title":"Proc Natl Acad Sci U S A"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"1516","DOI":"10.1002\/art.23475","article-title":"Persistent efficacy of anakinra in patients with tumour necrosis factor receptor-associated periodic syndrome","volume":"58","author":"Gattorno","year":"2008","journal-title":"Arthritis Rheum"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"1679","DOI":"10.1002\/art.40146","article-title":"Open-label, phase II study to assess the efficacy and safety of canakinumab treatment in active hyperimmunoglobulinemia D with periodic fever syndrome","volume":"69","author":"Arostegui","year":"2017","journal-title":"Arthritis Rheumatol"},{"key":"2023030304341419800_","first-page":"302","article-title":"Safety and efficacy of canakinumab in Japanese patients with phenotypes of cryopyrin-associated periodic syndrome as established in the first open-label, phase-3 pivotal study (24-week results)","volume":"31","author":"Imagawa","year":"2013","journal-title":"Clin Exp Rheumatol"},{"key":"2023030304341419800_","first-page":"19","article-title":"Long-term safety and efficacy of canakinumab in cryopyrin-associated periodic syndrome: results from an open-label, phase III pivotal study in Japanese patients","volume":"35","author":"Yokota","year":"2017","journal-title":"Clin Exp Rheumatol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"1908","DOI":"10.1056\/NEJMoa1706314","article-title":"Canakinumab for the treatment of autoinflammatory recurrent fever syndromes","volume":"378","author":"De Benedetti","year":"2018","journal-title":"N Engl J Med"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"226","DOI":"10.1080\/14397595.2020.1783163","article-title":"Efficacy and safety of canakinumab in systemic juvenile idiopathic arthritis: 48-week results from an open-label phase III study in Japanese patients","volume":"31","author":"Nishimura","year":"2021","journal-title":"Mod Rheumatol"},{"key":"2023030304341419800_","first-page":"228","article-title":"An interim analysis of post-marketing all-patients surveillance of canakinumab in Japanese patients with familial Mediterranean fever","volume":"32","author":"Hosono","year":"2020","journal-title":"Clin Rheumatol Relat Res"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"410","DOI":"10.1093\/rheumatology\/keh157","article-title":"Clinical and genetic aspects of the hereditary periodic fever syndromes","volume":"43","author":"Grateau","year":"2004","journal-title":"Rheumatology (Oxford)"},{"key":"2023030304341419800_","article-title":"Ilaris (Canakinumab) Injection 2020","author":"US Food and Drug Administration"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"2102","DOI":"10.1093\/rheumatology\/kex305","article-title":"International multi-centre study of pregnancy outcomes with interleukin-1 inhibitors","volume":"56","author":"Youngstein","year":"2017","journal-title":"Rheumatology (Oxford)"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","DOI":"10.3389\/fphar.2020.612259","article-title":"Inflammasome targeted therapy in pregnancy: new insights from an analysis of real-world data from the FAERS database and a systematic review","volume":"11","author":"Carnovale","year":"2021","journal-title":"Front Pharmacol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1007\/s10875-017-0389-3","article-title":"Placental transfer of canakinumab in a patient with Muckle-Wells syndrome","volume":"37","author":"Egawa","year":"2017","journal-title":"J Clin Immunol"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","first-page":"1636","DOI":"10.1136\/annrheumdis-2015-207546","article-title":"Recommendations for the management of autoinflammatory diseases","volume":"74","author":"ter Haar","year":"2015","journal-title":"Ann Rheum Dis"},{"key":"2023030304341419800_","doi-asserted-by":"crossref","DOI":"10.1186\/s12969-020-0409-3","article-title":"Consensus protocols for the diagnosis and management of the hereditary autoinflammatory syndromes CAPS, TRAPS and MKD\/HIDS: a German PRO-KIND initiative","volume":"18","author":"Hansmann","year":"2020","journal-title":"Pediatr Rheumatol Online J"}],"container-title":["Modern Rheumatology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/academic.oup.com\/mr\/advance-article-pdf\/doi\/10.1093\/mr\/roac041\/44139986\/roac041.pdf","content-type":"application\/pdf","content-version":"am","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/mr\/article-pdf\/33\/2\/381\/49400530\/roac041.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/mr\/article-pdf\/33\/2\/381\/49400530\/roac041.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,21]],"date-time":"2023-11-21T17:07:23Z","timestamp":1700586443000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/mr\/article\/33\/2\/381\/6586274"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,5,16]]},"references-count":29,"journal-issue":{"issue":"2","published-online":{"date-parts":[[2022,5,16]]},"published-print":{"date-parts":[[2023,3,2]]}},"URL":"https:\/\/doi.org\/10.1093\/mr\/roac041","relation":{},"ISSN":["1439-7595","1439-7609"],"issn-type":[{"value":"1439-7595","type":"print"},{"value":"1439-7609","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2023,3,1]]},"published":{"date-parts":[[2022,5,16]]}}}