https://m2.mtmt.hu/ hun 2026-09-23 07:47 JournalArticle 32709603 VALIDATED true Department of Pediatrics, University Medical Center Utrecht, Utrecht, Netherlands Department of Internal Medicine, Radboud University Medical Center, Radboudumc Expertise Center for Immunodeficiency and Autoinflammation (REIA), Nijmegen, Netherlands Pediatric Rheumatology Unit, Hospital Universitario y Politcnico La Fe, Valencia, Spain 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary Department of Immunology, Dmitry Rogachev National Medical Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation Unit Centre Multisite Romande d'Immuno-e Rhumatologie Pediatrique, Centre Hospitalier Universitaire Vaudois (CHUV), Lausanne, Switzerland Center for Autoinflammatory Diseases and Immunodeficiencies, IRCCS, G. Gaslini, Italy University of Genoa, Genoa, Italy Department of Pediatric Immunology, Hematology and Rheumatology, Universite de Paris, Institut des Maladies Genetiques (IMAGINE Institute), Reference Centre for Rheumatic, AutoImmune, and Systemic Diseases in Children (RAISE), Necker Hospital, Assistance Publique-Hopitaux de Paris, Paris, France Department of Pediatric Immunology, University Medical Center Utrecht, Utrecht, Netherlands Novartis Pharma AG, Basel, Switzerland Division of Rheumatology, Ospedale Pediatrico Bambino Gesù, Roma, Italy Export Date: 25 January 2023 CODEN: RUMAF Correspondence Address: Frenkel, J.; Department of General Pediatrics, PO-Box 85090, Netherlands; email: j.frenkel@umcutrecht.nl Chemicals/CAS: C reactive protein, 9007-41-4; canakinumab, 402710-25-2, 402710-27-4, 914613-48-2; Antibodies, Monoclonal; Antibodies, Monoclonal, Humanized; canakinumab; Serum Amyloid A Protein 0 false 2026-06-12T17:48:49.740+0000 2022-06-02T11:15:29.297+0000 2022-02-28T10:43:54.804+0000 true 10080205 Szalóky-Siki Ágnes /api/admin/10080205 Admin true 2025-05-26T10:36:57.447+0000 2022-06-02T11:15:29.516+0000 true 10080205 Szalóky-Siki Ágnes /api/admin/10080205 Admin true true false true 24 24 /api/publicationtype/24 PublicationType 1 true 24 JournalArticle true 10000059 Article true 24 24 /api/publicationtype/24 PublicationType 1 true 24 JournalArticle /api/subtype/10000059 Szakcikk SubType 101 true 10000059 true 1 /api/category/1 Category true 1 Jeyaratnam, Jerold Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised Phase 3 CLUSTER trial true true /api/journal/25853 REVIEWED true false 25853 false 25853 true 1462-0324 1462-0332 Journal FOREIGN 61 5 2088 2094 2088 7 2022 Objectives. To evaluate the long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency during the open label extension (weeks 41-113) of the randomized controlled CLUSTER trial. Methods. During a 72-week period, patients received open-label canakinumab 150 or 300 mg, every 4 or 8 weeks. The disease activity was evaluated every 8 weeks using physician global assessment and counting the number of flares. Concentrations of CRP and serum amyloid A protein were measured. The safety was studied by determination and classification of observed adverse events. The safety and efficacy were analysed separately in three subgroups of patients receiving a cumulative dose of less than <35 mg/kg, >= 35 to <70 mg/kg or >= 70 mg/kg. Results Of the 74 patients who started the CLUSTER study, 66 entered Epoch 4 and 65 completed it. During the 72-week period, 42 (64%) patients experienced no flares, while 13 (20%) had one flare, as compared with a median of 12 flares per year reported at baseline. Low physician global assessment scores were seen at the end of the study for all groups with >90% reporting minimal disease activity or none at all. Median CRP concentrations were consistently equal or lower than 10 mg/l, while median serum amyloid A concentrations remained only slightly above the normal range of 10 mg/l. The study showed no new or unexpected adverse events. Conclusion Canakinumab proved effective to control disease activity and prevent flares in mevalonate kinase deficiency during the 72-week study period. 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Gyermekgyógyászati Klinika (SE / AOK / K) 1 5 1 14 0 true Language 10002 /api/language/10002 Angol English true 2 true PersonAuthorship 100846074 /api/authorship/100846074 1 0.071428575 true false false Jeyaratnam Jerold true false false AuthorshipType 1 /api/authorshiptype/1 0 true 0 true false true PersonAuthorship 100846075 /api/authorship/100846075 2 0.071428575 false false false Simon Anna true false false AuthorshipType 1 /api/authorshiptype/1 0 true 0 true false true PersonAuthorship 100846076 /api/authorship/100846076 3 0.071428575 false false false Calvo Inmaculada true false false AuthorshipType 1 /api/authorshiptype/1 0 true 0 true false true PersonAuthorship 100846077 /api/authorship/100846077 4 0.071 false false false Author 10023611 /api/author/10023611 Constantin Tamás true 10023611 true Constantin Tamas true false false AuthorshipType 1 /api/authorshiptype/1 0 true 0 true false true PersonAuthorship 100846078 /api/authorship/100846078 5 0.071428575 false false false 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src="/frontend/resources/grid/publication-core-icon.png"> <img title="Idézőközlemény" style="float: left" src="/frontend/resources/grid/publication-citation-icon.png"> <div class="autype autype0"> <span class="author-name" >Jeyaratnam Jerold </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Simon Anna </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Calvo Inmaculada </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" mtid="10023611"><a href="/gui2/?type=authors&mode=browse&sel=10023611" target="_blank">Constantin Tamas (<span class="authorship-author-name">Constantin Tamás</span> <span class="authorAux-mtmt"> Gyermekgyógyászat</span>) </a> </span> <span class="author-affil"><span title="Semmelweis Egyetem">SE</span>/<span title="Általános Orvostudományi Kar">AOK</span>/<span title="Klinikum">K</span>/II. Sz. Gyermekgyógyászati Klinika</span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Shcherbina Anna </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Hofer Michael </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Gattorno Marco </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Martini Alberto </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Bader-Meunier Brigitte </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Vastert Bas </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Levy Jeremy </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Dekker Elise </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >de Benedetti Fabrizio </span> ;&nbsp;&nbsp;&nbsp; <span class="author-name" >Frenkel Joost ✉ </span> </div> </div> <div class="title"><a href="/gui2/?mode=browse&params=publication;32709603" target="_blank">Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised Phase 3 CLUSTER trial</a></div> <div> <span class="journal-title">RHEUMATOLOGY (UNITED KINGDOM)</span> <span class="journal-issn">(<a target="_blank" href="https://portal.issn.org/resource/ISSN/1462-0324">1462-0324</a> <a target="_blank" href="https://portal.issn.org/resource/ISSN/1462-0332">1462-0332</a>)</span>: <span class="journal-volume">61</span> <span class="journal-issue">5</span> <span class="page"> pp 2088-2094 </span> <span class="year">(2022)</span> </div> <div class="pub-footer"> <span class="language" xmlns="http://www.w3.org/1999/html">Nyelv: Angol | </span> <span class="identifiers"> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="10.1093/rheumatology/keab696" target="_blank" href="https://doi.org/10.1093/rheumatology/keab696"> DOI </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="000756674500001" target="_blank" href="https://www.webofscience.com/wos/woscc/full-record/000756674500001"> WoS </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="85129997494" target="_blank" href="http://www.scopus.com/record/display.url?origin=inward&eid=2-s2.0-85129997494"> Scopus </a> </span> <span class="id identifier oa_none" title="none"> <a style="color:blue" title="34554243" target="_blank" href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=34554243&dopt=Abstract"> PubMed </a> </span> </span> <OnlyViewableByAuthor><div class="ratings"> <div class="journal-subject">Folyóirat szakterülete: Scopus - Pharmacology (medical)&nbsp;&nbsp;&nbsp;SJR indikátor:&nbsp;D1</div> <div class="journal-subject">Folyóirat szakterülete: Scopus - Rheumatology&nbsp;&nbsp;&nbsp;SJR indikátor:&nbsp;Q1</div> </div></OnlyViewableByAuthor> <div class="publication-citation" style="margin-left: 0.5cm;"> <span title="Nyilvános idézőközlemények összesen, említések nélkül" class="citingPub-count">Nyilvános idéző összesen: 14</span> | Független: 11 | Függő: 3 | Nem jelölt: 0 | WoS jelölt: 14 | Scopus jelölt:&nbsp;12 | WoS/Scopus jelölt:&nbsp;14 | DOI jelölt:&nbsp;14 </div> <div class="publication-citation"> <a target="_blank" href="/api/publication?cond=citations.related;eq;32709603&sort=publishedYear,desc&sort=title"> Idézett közlemények száma: 2 </a> </div> <div class="mtid"><span class="long-pub-mtid">Közlemény: 32709603</span> | <span class="status-data status-VALIDATED"> Egyeztetett </span> Forrás Idéző | <span class="type-subtype">Folyóiratcikk ( Szakcikk ) </span> | <span class="pub-category">Tudományos</span> | <span class="publication-sourceOfData">WOS TAGGED</span> </div> <div class="lastModified">Utolsó módosítás: 2022.06.02. 13:15 Szalóky-Siki Ágnes (SE_KK_Admin5_SZSA, admin) </div> <pre class="comment" style="margin-top: 0; margin-bottom: 0;"><u>Megjegyzés</u>: Department of Pediatrics, University Medical Center Utrecht, Utrecht, Netherlands Department of Internal Medicine, Radboud University Medical Center, Radboudumc Expertise Center for Immunodeficiency and Autoinflammation (REIA), Nijmegen, Netherlands Pediatric Rheumatology Unit, Hospital Universitario y Politcnico La Fe, Valencia, Spain ...</pre> </div></div>