{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,24]],"date-time":"2026-08-24T16:07:13Z","timestamp":1787587633837,"version":"build-2736575974"},"reference-count":98,"publisher":"BMJ","issue":"2","license":[{"start":{"date-parts":[[2020,7,1]],"date-time":"2020-07-01T00:00:00Z","timestamp":1593561600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008272","name":"Novartis Pharmaceuticals Corporation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008272","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["RMD Open"],"accepted":{"date-parts":[[2020,6,17]]},"published-print":{"date-parts":[[2020,7]]},"abstract":"<jats:sec id=\"s1\">\n                  <jats:title>Objectives<\/jats:title>\n                  <jats:p>Several therapies are used for the treatment of rareautoinflammatory conditions like cryopyrin-associated periodic fever syndromes (CAPS), hyperimmunoglobulin Dsyndrome (HIDS)\/mevalonate kinase deficiency (MKD) and tumour necrosis factor receptor-associated periodic syndrome (TRAPS). However, reviews reporting on treatment outcomes of these therapies are lacking.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec id=\"s2\">\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>A systematic literature review was conducted using Embase, MEDLINE, MEDLINE-In Process and Cochrane databases to identify the randomised\/non-randomised controlled trials (RCTs\/non-RCTs) and real-world observational studies of CAPS, HIDS\/MKD and TRAPS published as full-texts (January 2000\u2013September 2017) or conference abstracts (January 2014\u2013September 2017). Studies with data for \u22651 biologic were included. Studies with &lt;5 patients were excluded.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec id=\"s3\">\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>Of the 3 342 retrieved publications, 72 studies were included (CAPS, n=43; HIDS\/MKD, n=9; TRAPS, n=7; studies with \u22652 cohorts, n=13). Most studies were full-text (n=56), published after 2010 (n=56) and real-world observational studies (n=58). Among included studies, four were RCTs (canakinumab, n=2 (CAPS, n=1; HIDS\/MKD and TRAPS, n=1); rilonacept, n=1 (in CAPS); simvastatin, n=1 (in HIDS\/MKD)). Canakinumab and anakinra were the most commonly used therapies for CAPS and HIDS\/MKD, whereas etanercept, canakinumab and anakinra were the most common for TRAPS. The available evidence suggested the efficacy or effectiveness of canakinumab and anakinra in CAPS, HIDS\/MKD and TRAPS, and of etanercept in TRAPS; asingle RCT demonstrated the efficacy of rilonacept in CAPS.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec id=\"s4\">\n                  <jats:title>Conclusions<\/jats:title>\n                  <jats:p>Canakinumab, anakinra, etanercept and rilonacept were reported to be well tolerated; however, injection-site reactions were observed frequently with anakinra, rilonacept and etanercept. Data on the use of tocilizumab, infliximab and adalimumab in these conditions were limited; thus, further research is warranted.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1136\/rmdopen-2020-001227","type":"journal-article","created":{"date-parts":[[2020,7,28]],"date-time":"2020-07-28T17:40:13Z","timestamp":1595958013000},"page":"e001227","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":35,"title":["Systematic literature review of efficacy\/effectiveness and safety of current therapies for the treatment of cryopyrin-associated periodic syndrome, hyperimmunoglobulin D syndrome and tumour necrosis factor receptor-associated periodic syndrome"],"prefix":"10.1136","volume":"6","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6365-6598","authenticated-orcid":false,"given":"Jasmin Beate","family":"Kuemmerle-Deschner","sequence":"first","affiliation":[{"name":"Division of Pediatric Rheumatology, Department of Pediatrics, University Hospital Tuebingen, T\u00fcbingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raju","family":"Gautam","sequence":"additional","affiliation":[{"name":"Patient Access Services, Novartis Healthcare Ltd, Hyderabad, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aneesh Thomas","family":"George","sequence":"additional","affiliation":[{"name":"Patient Access Services, Novartis Healthcare Ltd, Hyderabad, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Syed","family":"Raza","sequence":"additional","affiliation":[{"name":"Patient Access Services, Novartis Healthcare Ltd, Hyderabad, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen Graham","family":"Lomax","sequence":"additional","affiliation":[{"name":"Medical Affairs, Novartis Pharmaceuticals Corp, East Hanover, New Jersey, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Hur","sequence":"additional","affiliation":[{"name":"Health Economics and Outcomes Research (HEOR), Novartis Pharmaceuticals Corp, East Hanover, New Jersey, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2020,7,28]]},"reference":[{"key":"2025090313345540000_6.2.e001227.1","doi-asserted-by":"publisher","DOI":"10.1016\/j.berh.2017.12.001"},{"key":"2025090313345540000_6.2.e001227.2","first-page":"32","article-title":"A dermatologic perspective on autoinflammatory diseases","volume":"36","author":"Marzano","year":"2018","journal-title":"Clin Exp Rheumatol"},{"key":"2025090313345540000_6.2.e001227.3","doi-asserted-by":"crossref","first-page":"30","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"},{"key":"2025090313345540000_6.2.e001227.4","doi-asserted-by":"publisher","DOI":"10.1016\/j.clim.2012.09.011"},{"key":"2025090313345540000_6.2.e001227.5","doi-asserted-by":"publisher","DOI":"10.1136\/annrheumdis-2013-204184"},{"key":"2025090313345540000_6.2.e001227.6","unstructured":"U.S. Food and Drug Administration . 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