Current treatment options for monogenic periodic fever syndromes – the role of interleukin 1 inhibitors
Authors:
Tomáš Milota 1,2; Hana Malcová 1; Zuzana Střížová 1,2; Dita Cebecauerová 1; Rudolf Horváth 1
Authors place of work:
Oddělení revmatologie dětí a dospělých, FN Motol, Praha
1; Ústav imunologie 2. LF UK a FN Motol, Praha
2
Published in the journal:
Čas. Lék. čes. 2022; 161: 3-10
Category:
Review Article
Summary
Monogenic periodic fever syndromes are heterogeneous group of autoinflammatory diseases including distinct syndromes, such as cryopyrin-associated periodic syndrome (CAPS), tumor necrosis factor alpha receptor-associated periodic syndrome (TRAPS), mevalonate kinase deficiency/hyper IgD syndrome (MKD/HIDS), and familial Mediterranean fever (FMF). Individual diseases differ in pathogenesis, clinical manifestations, and severity. However, cytokines from the interleukin 1 (IL-1) family play a key role in all of them. Inhibition of these cytokines, especially IL-1, thus plays a crucial role in their treatment.
At present, we have a wide range of drugs that differ in structure, mechanism of action, efficacy, and spectrum of side effects. The most available are anakinra, canakinumab and rilonacept. Moreover, several clinical trials are currently underway with other very promising drugs, such as gevokizumab, tadekinig alfa or tranilast. In the following review, we provide a new perspective on the efficacy and safety of IL-1 inhibitors that have provided the novel results coming from recently published clinical trials.
Keywords:
interleukin 1 – canakinumab – anakinra – monogenic periodic fever syndromes – rilonacept
Zdroje
- Georgin-Lavialle S, Fayand A, Rodrigues F et al. Autoinflammatory diseases: state of the art. Presse Med 2019; 48 (1 Pt 2): e25–e48.
- Cantarini L, Vitale A, Lucherini OM et al. The labyrinth of autoinflammatory disorders: A snapshot on the activity of a third-level center in Italy. Clinical Rheumatology 2015; 34(1): 17–28.
- Cattalini M, Soliani M, Lopalco G et al. Systemic and organ involvement in monogenic autoinflammatory disorders: a global review filtered through internists' lens. Intern Emerg Med 2016; 11(6): 781–791.
- Cantarini L, Lopalco G, Selmi C et al. Autoimmunity and autoinflammation as the yin and yang of idiopathic recurrent acute pericarditis. Autoimmu Rev 2015; 14(2): 90–97.
- Obici L, Merlini G. Amyloidosis in autoinflammatory syndromes. Autoimmun Rev 2012; 12(1): 14–17.
- Feist E, Mitrovic S, Fautrel B. Mechanisms, biomarkers and targets for adult-onset Still's disease. Nat R Rheumatol 2018; 14(10): 603–618.
- Lee JJY, Schneider R. Systemic juvenile idiopathic arthritis. Pediatr Clin North Am 2018; 65(4): 691–709.
- Dinarello CA. Overview of the IL-1 family in innate inflammation and acquired immunity. Immunol Rev 2018; 281(1): 8–27.
- Dinarello CA. The IL-1 family of cytokines and receptors in rheumatic diseases. Nature reviews. Rheumatology 2019; 15(10): 612–632.
- Dinarello CA. Interleukin-1 in the pathogenesis and treatment of inflammatory diseases. Blood 2011; 117(14): 3720–32.
- Junge G, Mason J, Feist E. Adult-onset Still's disease – the evidence that anti-interleukin-1 treatment is effective and well-tolerated (a comprehensive literature review). Semin Arthritis Rheum 2017; 47(2): 295–302.
- Tarp S, Amarilyo G, Foeldvari I et al. Efficacy and safety of biological agents for systemic juvenile idiopathic arthritis: a systematic review and meta-analysis of randomized trials. Rheumatology 2016; 55(4): 669–679.
- Gattorno M, Hofer M, Federici S et al. Classification criteria for autoinflammatory recurrent fevers. Ann Rheum Dis 2019; 78(8): 1025–1032.
- Ter Haar NM, Oswald M, Jeyaratnam J et al. Recommendations for the management of autoinflammatory diseases. Ann Rheum Dis 2015; 74(9): 1636–1644.
- Owyang AM, Issafras H, Corbin J et al. XOMA 052, a potent, high-affinity monoclonal antibody for the treatment of IL-1beta-mediated diseases. mAbs 2011; 3(1): 49–60.
- Gabay C, Fautrel B, Rech J et al. Open-label, multicentre, dose-escalating phase II clinical trial on the safety and efficacy of tadekinig alfa (IL-18BP) in adult-onset Still's disease. Ann Rheum Dis 2018; 77(6): 840–7.
- Huang Y, Jiang H, Chen Y et al. Tranilast directly targets NLRP3 to treat inflammasome-driven diseases. EMBO Mol Med 2018; 10(4): e8689.
- Sanchez-Fernandez A, Skouras DB, Dinarello CA, Lopez-Vales R. OLT1177 (dapansutrile), a selective NLRP3 inflammasome inhibitor, ameliorates experimental autoimmune encephalomyelitis pathogenesis. Front Immunol 2019; 10: 2578.
- Kluck V, Jansen T, Janssen M et al. Dapansutrile, an oral selective NLRP3 inflammasome inhibitor, for treatment of gout flares: an open-label, dose-adaptive, proof-of-concept, phase 2a trial. Lancet Rheumatol 2020; 2(5): e270–e280.
- McKee CM, Coll RC. NLRP3 inflammasome priming: A riddle wrapped in a mystery inside an enigma. J Leukoc Biol 2020; 108(3): 937–952.
- La Manna S, Di Natale C, Florio D, Marasco D. Peptides as therapeutic agents for inflammatory-related diseases. Int J Mol Sci 2018; 19(9): 2714.
- Caseley EA, Poulter JA, Rodrigues F et al. Inflammasome inhibition under physiological and pharmacological conditions. Genes Immun 2020; 21(4): 211–223.
- Gasparyan AY, Ayvazyan L, Blackmore H, Kitas GD. Writing a narrative biomedical review: Considerations for authors, peer reviewers, and editors. Rheumatol Int 2011; 31(11): 1409–1417.
- Kuemmerle-Deschner JB, Dembi Samba S, Tyrrell PN et al. Challenges in diagnosing Muckle-Wells syndrome: Identifying two distinct phenotypes. Arthritis Care Res 2014; 66(5): 765–772.
- Tran TA. Muckle-Wells syndrome: Clinical perspectives. Open Access Rheumatol 2017; 9: 123–129.
- Finetti M, Omenetti A, Federici S et al. Chronic infantile neurological cutaneous and articular (CINCA) syndrome: A review. Orphanet Journal Rare Dis 2016; 11(1): 167.
- Cantarini L, Lucherini OM, Frediani B et al. Bridging the gap between the clinician and the patient with cryopyrin-associated periodic syndromes. Int J Immunopathol Pharmacol 2011; 24(4): 827–836.
- Goldbach-Mansky R, Dailey NJ, Canna SW et al. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1beta inhibition. New Engl J Med 2006; 355(6): 581–592.
- Kuemmerle-Deschner JB, Tyrrell PN, Koetter I et al. Efficacy and safety of anakinra therapy in pediatric and adult patients with the autoinflammatory Muckle-Wells syndrome. Arthritis Rheum 2011; 63(3): 840–849.
- Kullenberg T, Lofqvist M, Leinonen M et al. Long-term safety profile of anakinra in patients with severe cryopyrin-associated periodic syndromes. Rheumatology 2016; 55(8): 1499–1506.
- Kone-Paut I, Lachmann HJ, Kuemmerle-Deschner JB et al. Sustained remission of symptoms and improved health-related quality of life in patients with cryopyrin-associated periodic syndrome treated with canakinumab: results of a double-blind placebo-controlled randomized withdrawal study. Arthritis Res Ther 2011; 13(6): R202.
- Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB et al. Use of canakinumab in the cryopyrin-associated periodic syndrome. New Engl J Med 2009; 360(23): 2416–2425.
- Hoffman HM, Throne ML, Amar NJ et al. Efficacy and safety of rilonacept (interleukin-1 Trap) in patients with cryopyrin-associated periodic syndromes: Results from two sequential placebo-controlled studies. Arthritis Rheum 2008; 58(8): 2443–2452.
- Rigante D, Lopalco G, Vitale A et al. Key facts and hot spots on tumor necrosis factor receptor-associated periodic syndrome. Clin Rheumatol 2014; 33(9): 1197–1207.
- Magnotti F, Vitale A, Rigante D et al. The most recent advances in pathophysiology and management of tumour necrosis factor receptor-associated periodic syndrome (TRAPS): Personal experience and literature review. Clin Exp Rheumatol 2013; 31(3 Suppl. 77): 141–149.
- Cantarini L, Lopalco G, Vitale A et al. Delights and let-downs in the management of tumor necrosis factor receptor-associated periodic syndrome: The canakinumab experience in a patient with a high-penetrance T50M TNFRSF1A variant. Int J Rheum Dis 2015; 18(4): 473–475.
- Lopalco G, Rigante D, Vitale A et al. Tumor necrosis factor receptor-associated periodic syndrome managed with the couple canakinumab-alendronate. Clin Rheumatol 2015; 34(4): 807–809.
- Gattorno M, Pelagatti MA, Meini A et al. Persistent efficacy of anakinra in patients with tumor necrosis factor receptor-associated periodic syndrome. Arthritis Rheum 2008; 58(5): 1516–1520.
- de Benedetti F, Gattorno M, Anton J et al. Canakinumab for the treatment of autoinflammatory recurrent fever syndromes. New Engl J Med 2018; 378(20): 1908–1919.
- Brik R, Shinawi M, Kasinetz L, Gershoni-Baruch R. The musculoskeletal manifestations of familial Mediterranean fever in children genetically diagnosed with the disease. Arthritis Rheum 2001; 44(6): 1416–1419.
- Uthman I, Hajj-Ali RA, Arayssi T et al. Arthritis in familial Mediterranean fever. Rheumatol Int 2001; 20(4): 145–148.
- Rigante D, Lopalco G, Tarantino G et al. Non-canonical manifestations of familial Mediterranean fever: A changing paradigm. Clin Rheumatol 2015; 34(9): 1503–11.
- Alghamdi M. Familial Mediterranean fever, review of the literature. Clin Rheumatol 2017; 36(8): 1707–1713.
- El Hasbani G, Jawad A, Uthman I. Update on the management of colchicine resistant Familial Mediterranean Fever (FMF). Orphanet Journal Rare Dis 2019; 14(1): 224.
- Ozen S, Kone-Paut I, Gul A. Colchicine resistance and intolerance in familial Mediterranean fever: definition, causes, and alternative treatments. Semin Arthritis Rheum 2017; 47(1): 115–120.
- Tombetti E, Mule A, Tamanini S et al. Novel pharmacotherapies for recurrent pericarditis: Current options in 2020. Curr Cardiol Rep 2020; 22(8): 59.
- Lazaros G, Antonopoulos AS, Antonatou K et al. Hydroxychloroquine for colchicine-resistant glucocorticoid-dependent idiopathic recurrent pericarditis: A pilot observational prospective study. Int J Cardiol 2020; 311: 77–82.
- Ben-Zvi I, Kukuy O, Giat E et al. Anakinra for colchicine-resistant familial Mediterranean fever: A randomized, double-blind, placebo-controlled trial. Arthritis Rheumatol 2017; 69(4): 854–862.
- Ozen S, Ben-Cherit E, Foeldvari I et al. Long-term efficacy and safety of canakinumab in patients with colchicine-resistant familial Mediterranean fever: results from the randomised phase III CLUSTER trial. Ann Rheum Dis 2020; 79(10): 1362–1369.
- Sag E, Akal F, Atalay E et al. Anti-IL1 treatment in colchicine-resistant paediatric FMF patients: real life data from the HELIOS registry. Rheumatology 2020; 59(11): 3324–3329.
- Hashkes PJ, Spalding SJ, Giannini EH et al. Rilonacept for colchicine-resistant or -intolerant familial Mediterranean fever: a randomized trial. Ann Inter Med 2012; 157(8): 533–541.
- Houten SM, Kuis W, Duran M et al. Mutations in MVK, encoding mevalonate kinase, cause hyperimmunoglobulinaemia D and periodic fever syndrome. Nature Genet 1999; 22(2): 175–177.
- Haar NM ter, Jeyaratnam J, Lachmann HJ et al. The phenotype and genotype of mevalonate kinase deficiency: a series of 114 cases from the Eurofever registry. Arthritis Rheumatol 2016; 68(11): 2795–2805.
- van der Hilst JC, Bodar EJ, Barron KS et al. Long-term follow-up, clinical features, and quality of life in a series of 103 patients with hyperimmunoglobulinemia D syndrome. Medicine 2008; 87(6): 301–310.
- Malcová H, Milota T, Střížová Z et al. Interleukin-1 blockade in polygenic autoinflammatory disorders: Where are we now? Front Pharmacol 2020; 11: 619273.
- Malcová H, Střížová Z, Milota T et al. IL-1 Inhibitors in the treatment of monogenic periodic fever syndromes: from the past to the future perspectives. Front Immunol 2020; 11: 619257.
Štítky
Addictology Allergology and clinical immunology Angiology Audiology Clinical biochemistry Dermatology & STDs Paediatric gastroenterology Paediatric surgery Paediatric cardiology Paediatric neurology Paediatric ENT Paediatric psychiatry Paediatric rheumatology Diabetology Pharmacy Vascular surgery Pain management Dental HygienistČlánok vyšiel v časopise
Journal of Czech Physicians
2022 Číslo 1
- Metamizole at a Glance and in Practice – Effective Non-Opioid Analgesic for All Ages
- Advances in the Treatment of Myasthenia Gravis on the Horizon
- Metamizole vs. Tramadol in Postoperative Analgesia
- Spasmolytic Effect of Metamizole
- What Effect Can Be Expected from Limosilactobacillus reuteri in Mucositis and Peri-Implantitis?
Najčítanejšie v tomto čísle
- Physiology of ageing
- Tobacco dependence treatment guidelines
- Do clinical pharmacists contribute to fall prevention in healthcare facilities also in the Czech Republic? 10 years of monitoring and experience from ÚVN Prague
- Multi-branch cooperation on SARS-CoV-2 omicron capture: a case report