Efficacy and safety of canakinumab in systemic juvenile idiopathic arthritis: 48-week results from an open-label phase III study in Japanese patients

References

• Ravelli A, Martini A. Juvenile idiopathic arthritis. Lancet. 2007;369(9563):767–78.
   PubMed Web of Science ®Google Scholar
• Takei S. Systemic JIA as an autoinflammatory disease. Inflamm Regen. 2011;31(1):52–62.
   Google Scholar
• Takei S, Yamashita S, Kato T. Nation-wide survey for patients with juvenile idiopathic arthritis in Japan. Annual Report on Children with Chronic Refractory Diseases from the Japanese Ministry of Health, Labor and Welfare 2008. p. 102–13.
   Google Scholar
• Fujikawa S, Okuni M. Clinical analysis of 570 cases with juvenile rheumatoid arthritis: results of a nationwide retrospective survey in Japan. Acta Paediatr Jpn. 1997;39(2):245–9.
   PubMedGoogle Scholar
• Okamoto N, Yokota S, Takei S, Okura Y, Kubota T, Shimizu M, et al. Clinical practice guidance for juvenile idiopathic arthritis (JIA) 2018. Mod Rheumatol. 2019;29(1):41–59.
   PubMed Web of Science ®Google Scholar
• Martini A, Ravelli A, Di Fuccia G, Rosti V, Cazzola M, Barosi G. Intravenous iron therapy for severe anaemia in systemic-onset juvenile chronic arthritis. Lancet. 1994;344(8929):1052–4.
   PubMed Web of Science ®Google Scholar
• Prakken B, Albani S, Martini A. Juvenile idiopathic arthritis. Lancet. 2011;377(9783):2138–49.
   PubMed Web of Science ®Google Scholar
• Schneider R, Laxer RM. Systemic onset juvenile rheumatoid arthritis. Baillieres Clin Rheumatol. 1998;12(2):245–71.
   PubMed Web of Science ®Google Scholar
• Henderson CJ, Cawkwell GD, Specker BL, Sierra RI, Wilmott RW, Campaigne BN, et al. Predictors of total body bone mineral density in non-corticosteroid-treated prepubertal children with juvenile rheumatoid arthritis. Arthritis Rheum. 1997;40(11):1967–75.
   PubMed Web of Science ®Google Scholar
• De Benedetti F, Alonzi T, Moretta A, Lazzaro D, Costa P, Poli V, et al. Interleukin 6 causes growth impairment in transgenic mice through a decrease in insulin-like growth factor-I. A model for stunted growth in children with chronic inflammation. J Clin Invest. 1997;99(4):643–50.
   PubMed Web of Science ®Google Scholar
• Simon D, Lucidarme N, Prieur AM, Ruiz JC, Czernichow P. Effects on growth and body composition of growth hormone treatment in children with juvenile idiopathic arthritis requiring steroid therapy. J Rheumatol. 2003;30(11):2492–9.
   PubMed Web of Science ®Google Scholar
• Ravelli A, Minoia F, Davi S, Horne A, Bovis F, Pistorio A, et al. 2016 classification criteria for macrophage activation syndrome complicating systemic juvenile idiopathic arthritis: a European League Against Rheumatism/American College of Rheumatology/Paediatric Rheumatology International Trials Organisation Collaborative Initiative. Ann Rheum Dis. 2016;75(3):481–9.
   PubMed Web of Science ®Google Scholar
• Pascual V, Allantaz F, Arce E, Punaro M, Banchereau J. Role of interleukin-1 (IL-1) in the pathogenesis of systemic onset juvenile idiopathic arthritis and clinical response to IL-1 blockade. J Exp Med. 2005;201(9):1479–86.
   PubMed Web of Science ®Google Scholar
• Gattorno M, Piccini A, Lasiglie D, Tassi S, Brisca G, Carta S, et al. The pattern of response to anti-interleukin-1 treatment distinguishes two subsets of patients with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum. 2008;58(5):1505–15.
   PubMed Web of Science ®Google Scholar
• De Benedetti F, Martini A. Is systemic juvenile rheumatoid arthritis an interleukin 6 mediated disease? J Rheumatol. 1998;25(2):203–7.
   PubMed Web of Science ®Google Scholar
• Rose-John S, Scheller J, Elson G, Jones SA. Interleukin-6 biology is coordinated by membrane-bound and soluble receptors: role in inflammation and cancer. J Leukoc Biol. 2006;80(2):227–36.
   PubMed Web of Science ®Google Scholar
• Chen O, Shan N, Zhu X, Wang Y, Ren P, Wei D, et al. The imbalance of IL-18/IL-18BP in patients with systemic juvenile idiopathic arthritis. Acta Biochim Biophys Sin (Shanghai). 2013;45(4):339–41.
   PubMed Web of Science ®Google Scholar
• Beukelman T, Patkar NM, Saag KG, Tolleson-Rinehart S, Cron RQ, DeWitt EM, et al. 2011 American College of Rheumatology recommendations for the treatment of juvenile idiopathic arthritis: initiation and safety monitoring of therapeutic agents for the treatment of arthritis and systemic features. Arthritis Care Res (Hoboken). 2011;63(4):465–82.
   PubMed Web of Science ®Google Scholar
• Malattia C, Martini A. Glucocorticoids in juvenile idiopathic arthritis. Ann N Y Acad Sci. 2014;1318:65–70.
   PubMed Web of Science ®Google Scholar
• Toplak N, Blazina S, Avcin T. The role of IL-1 inhibition in systemic juvenile idiopathic arthritis: current status and future perspectives. Drug Des Devel Ther. 2018;12:1633–43.
   PubMed Web of Science ®Google Scholar
• Ringold S, Weiss PF, Beukelman T, Dewitt EM, Ilowite NT, Kimura Y, et al. 2013 update of the 2011 American College of Rheumatology recommendations for the treatment of juvenile idiopathic arthritis: recommendations for the medical therapy of children with systemic juvenile idiopathic arthritis and tuberculosis screening among children receiving biologic medications. Arthritis Care Res (Hoboken). 2013;65(10):1551–63.
   PubMed Web of Science ®Google Scholar
• Ruperto N, Brunner HI, Quartier P, Constantin T, Wulffraat N, Horneff G, et al. Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med. 2012;367(25):2396–406.
   PubMed Web of Science ®Google Scholar
• Yokota S, Imagawa T, Mori M, Miyamae T, Aihara Y, Takei S, et al. Efficacy and safety of tocilizumab in patients with systemic-onset juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled, withdrawal phase III trial. Lancet. 2008;371(9617):998–1006.
   PubMed Web of Science ®Google Scholar
• DeWitt EM, Kimura Y, Beukelman T, Nigrovic PA, Onel K, Prahalad S, et al. Consensus treatment plans for new-onset systemic juvenile idiopathic arthritis. Arthritis Care Res (Hoboken). 2012;64(7):1001–10.
   PubMed Web of Science ®Google Scholar
• Woerner A, Uettwiller F, Melki I, Mouy R, Wouters C, Bader-Meunier B, et al. Biological treatment in systemic juvenile idiopathic arthritis: achievement of inactive disease or clinical remission on a first, second or third biological agent. RMD Open. 2015;1(1):e000036.
   PubMed Web of Science ®Google Scholar
• De Benedetti F, Brunner H, Ruperto N, Schneider R, Xavier R, Allen R, et al. Catch-up growth during tocilizumab therapy for systemic juvenile idiopathic arthritis: results from a phase III trial. Arthritis Rheumatol. 2015;67(3):840–8.
   PubMed Web of Science ®Google Scholar
• Ruperto N, Brunner HI, Quartier P, Constantin T, Wulffraat NM, Horneff G, et al. Canakinumab in patients with systemic juvenile idiopathic arthritis and active systemic features: results from the 5-year long-term extension of the phase III pivotal trials. Ann Rheum Dis. 2018;77(12):1710–9.
   PubMed Web of Science ®Google Scholar
• Stanbury RM, Graham EM. Systemic corticosteroid therapy-side effects and their management. Br J Ophthalmol. 1998;82(6):704–8.
   PubMed Web of Science ®Google Scholar
• Mushtaq T, Ahmed SF. The impact of corticosteroids on growth and bone health. Arch Dis Child. 2002;87(2):93–6.
   PubMed Web of Science ®Google Scholar
• Yokota S, Itoh Y, Morio T, Origasa H, Sumitomo N, Tomobe M, et al. Tocilizumab in systemic juvenile idiopathic arthritis in a real-world clinical setting: results from 1 year of postmarketing surveillance follow-up of 417 patients in Japan. Ann Rheum Dis. 2016;75(9):1654–60.
   PubMed Web of Science ®Google Scholar
• Horneff G, Schulz AC, Klotsche J, Hospach A, Minden K, Foeldvari I, et al. Experience with etanercept, tocilizumab and interleukin-1 inhibitors in systemic onset juvenile idiopathic arthritis patients from the BIKER registry. Arthritis Res Ther. 2017;19(1):256.
   PubMed Web of Science ®Google Scholar
• Alten R, Gram H, Joosten LA, van den Berg WB, Sieper J, Wassenberg S, et al. The human anti-IL-1 beta monoclonal antibody ACZ885 is effective in joint inflammation models in mice and in a proof-of-concept study in patients with rheumatoid arthritis. Arthritis Res Ther. 2008;10(3):R67.
   PubMed Web of Science ®Google Scholar
• Kuemmerle-Deschner JB, Ramos E, Blank N, Roesler J, Felix SD, Jung T, et al. Canakinumab (ACZ885, a fully human IgG1 anti-IL-1beta mAb) induces sustained remission in pediatric patients with cryopyrin-associated periodic syndrome (CAPS). Arthritis Res Ther. 2011;13(1):R34.
   PubMed Web of Science ®Google Scholar
• Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, Leslie KS, Hachulla E, Quartier P, et al. Use of canakinumab in the cryopyrin-associated periodic syndrome. N Engl J Med. 2009;360(23):2416–25.
   PubMed Web of Science ®Google Scholar
• Ruperto N, Quartier P, Wulffraat N, Woo P, Ravelli A, Mouy R, et al. A phase II, multicenter, open-label study evaluating dosing and preliminary safety and efficacy of canakinumab in systemic juvenile idiopathic arthritis with active systemic features. Arthritis Rheum. 2012;64(2):557–67.
   PubMed Web of Science ®Google Scholar
• Petty RE, Southwood TR, Manners P, Baum J, Glass DN, Goldenberg J, et al. International League of Associations for Rheumatology classification of juvenile idiopathic arthritis: second revision, Edmonton, 2001. J Rheumatol. 2004;31(2):390–2.
   PubMed Web of Science ®Google Scholar
• Ruperto N, Ravelli A, Pistorio A, Malattia C, Cavuto S, Gado-West L, et al. Cross-cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries. Review of the general methodology. Clin Exp Rheumatol. 2001;19:S1–S9.
   PubMed Web of Science ®Google Scholar
• Ruperto N, Martini A. Networking in paediatrics: the example of the Paediatric Rheumatology International Trials Organisation (PRINTO). Arch Dis Child. 2011;96(6):596–601.
   PubMed Web of Science ®Google Scholar
• Ruperto N, Giannini EH, Pistorio A, Brunner HI, Martini A, Lovell DJ. Is it time to move to active comparator trials in juvenile idiopathic arthritis? A review of current study designs. Arthritis Rheum. 2010;62(11):3131–9.
   PubMed Web of Science ®Google Scholar
• Giannini EH, Ruperto N, Ravelli A, Lovell DJ, Felson DT, Martini A. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum. 1997;40(7):1202–9.
   PubMed Web of Science ®Google Scholar
• Wallace CA, Ruperto N, Giannini E, et al.; Childhood Arthritis and Rheumatology Research Alliance; Pediatric Rheumatology International Trials Organization; Pediatric Rheumatology Collaborative Study Group. Preliminary criteria for clinical remission for select categories of juvenile idiopathic arthritis. J Rheumatol. 2004;31:2290–4.
   PubMed Web of Science ®Google Scholar
• Ravelli A, Magni-Manzoni S, Pistorio A, Besana C, Foti T, Ruperto N, et al. Preliminary diagnostic guidelines for macrophage activation syndrome complicating systemic juvenile idiopathic arthritis. J Pediatr. 2005;146(5):598–604.
   PubMed Web of Science ®Google Scholar
• Grevich S, Shenoi S. Update on the management of systemic juvenile idiopathic arthritis and role of IL-1 and IL-6 inhibition. Adolesc Health Med Ther. 2017;8:125–35.
   PubMed Web of Science ®Google Scholar
• Consolaro A, Negro G, Lanni S, Solari N, Martini A, Ravelli A. Toward a treat-to-target approach in the management of juvenile idiopathic arthritis. Clin Exp Rheumatol. 2012;30:S157–S62.
   PubMed Web of Science ®Google Scholar
• Brunner HI, Klein-Gitelman MS, Miller MJ, Barron A, Baldwin N, Trombley M, et al. Minimal clinically important differences of the childhood health assessment questionnaire. J Rheumatol. 2005;32(1):150–61.
   PubMed Web of Science ®Google Scholar
• Woo P. Systemic juvenile idiopathic arthritis: diagnosis, management, and outcome. Nat Clin Pract Rheumatol. 2006;2(1):28–34.
   PubMedGoogle Scholar
• Chakraborty A, Tannenbaum S, Rordorf C, Lowe PJ, Floch D, Gram H, et al. Pharmacokinetic and pharmacodynamic properties of canakinumab, a human anti-interleukin-1β monoclonal antibody. Clin Pharmacokinet. 2012;51(6):e1.
   PubMedGoogle Scholar
• Gohar F, Kessel C, Lavric M, Holzinger D, Foell D. Review of biomarkers in systemic juvenile idiopathic arthritis: helpful tools or just playing tricks? Arthritis Res Ther. 2016;18:163.
   PubMed Web of Science ®Google Scholar
• Put K, Avau A, Brisse E, Mitera T, Put S, Proost P, et al. Cytokines in systemic juvenile idiopathic arthritis and haemophagocytic lymphohistiocytosis: tipping the balance between interleukin-18 and interferon-γ. Rheumatology (Oxford). 2015;54(8):1507–17.
   PubMed Web of Science ®Google Scholar
• Grom AA, Ilowite NT, Pascual V, Brunner HI, Martini A, Lovell D, et al. Rate and clinical presentation of macrophage activation syndrome in patients with systemic juvenile idiopathic arthritis treated with canakinumab. Arthritis Rheumatol. 2016;68(1):218–28.
   PubMed Web of Science ®Google Scholar