Canakinumab for the treatment of autoinflammatory recurrent fever syndromes

New England Journal of Medicine

Volumn 378, Issue 20, 2018, Pages 1908-1919

  De Benedetti, Fabrizio ^a   Gattorno, Marco ^b   Anton, Jordi ^e   Eldad, Ben Chetrit ^h   Frenkel, Joost ^k   Hoffman, Hal M ^m   Isabelle, Koné Paut ⁿ   Lachmann, Helen J ^p   Ozen, Seza ^r   Simon, Anna ^l   Zeft, Andrew ^u   Penades, Inmaculada Calvo ^g   Moutschen, Michel ^v   Quartier, Pierre ^o   Kasapcopur, Ozgur ^s   Shcherbina, Anna ^x   Hofer, Michael ^y   Hashkes, Philip J ⁱ   Van der Hilst, Jeroen ^w   Hara, Ryoki ^aa   Segundo, Bujan Rivas ^f   Constantin, Tamas ^ab   Gul, Ahmet ^t   Livneh, Avi ^j   Brogan, Paul ^q   Cattalini, Marco ^c   Obici, Laura ^d   Lheritier, Karine ^z   Speziale, Antonio ^ac   Junge, Guido ^z   more..

^a BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

^b G GASLINI INSTITUTE   (Italy)

^c UNIVERSITY OF BRESCIA   (Italy)

^d FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^e UNIVERSITY OF BARCELONA   (Spain)

^f HOSPITAL UNIVERSITARI VALL D HEBRON   (Spain)

^g HOSPITAL UNIVERSITARIO LA FE   (Spain)

^h HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

ⁱ SHAARE ZEDEK MEDICAL CENTER   (Israel)

^j TEL AVIV UNIVERSITY   (Israel)

^k UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^l RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^m RADY CHILDREN'S HOSPITAL   (United States)

ⁿ UNIVERSITÉ PARIS SACLAY   (France)

^o UNIVERSITÉ PARIS DESCARTES   (France)

^p UNIVERSITY COLLEGE LONDON   (United Kingdom)

^q UNIVERSITY COLLEGE LONDON   (United Kingdom)

^r HACETTEPE UNIVERSITY   (Turkey)

^s ISTANBUL UNIVERSITY   (Turkey)

^t ISTANBUL UNIVERSITY   (Turkey)

^u LERNER RESEARCH INSTITUTE   (United States)

^v UNIVERSITY OF LIÈGE   (Belgium)

^w HASSELT UNIVERSITY   (Belgium)

^x Clinical and Research Center for Allergy and Immunology   (Russian Federation)

^y UNIVERSITY OF LAUSANNE   (Switzerland)

^z NOVARTIS PHARMA AG   (Switzerland)

^aa YOKOHAMA CITY UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^ab SEMMELWEIS UNIVERSITY   (Hungary)

^ac NONE

more..

Author keywords

[No Author keywords available]

Indexed keywords

C REACTIVE PROTEIN; CANAKINUMAB; COLCHICINE; PLACEBO; SERUM AMYLOID A; INTERLEUKIN 1BETA; MONOCLONAL ANTIBODY;

ABDOMINAL PAIN; ADOLESCENT; ADULT; ARTICLE; CELLULITIS; CLINICAL OUTCOME; COHORT ANALYSIS; CONJUNCTIVITIS; CONTROLLED STUDY; DISEASE COURSE; DISEASE DURATION; DISEASE EXACERBATION; DOUBLE BLIND PROCEDURE; DRUG DOSE INCREASE; DRUG DOSE REGIMEN; DRUG EFFICACY; DRUG RESPONSE; DRUG SAFETY; DRUG WITHDRAWAL; FAMILIAL MEDITERRANEAN FEVER; FEMALE; GASTROENTERITIS; HEADACHE; HUMAN; INFECTION; INFECTIOUS DIARRHEA; INJECTION SITE REACTION; LARYNGITIS; MAJOR CLINICAL STUDY; MALE; MEVALONATE KINASE DEFICIENCY; NEUTROPENIA; OPEN STUDY; PELVIS ABSCESS; PHARYNGITIS; PHARYNGOTONSILLITIS; PHASE 3 CLINICAL TRIAL; PNEUMONIA; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RECURRENT FEVER; TONSILLITIS; TREATMENT OUTCOME; TREATMENT RESPONSE; TUMOR NECROSIS FACTOR RECEPTOR ASSOCIATED PERIODIC SYNDROME; YOUNG ADULT; ANTAGONISTS AND INHIBITORS; CHILD; CLINICAL TRIAL; DOSE RESPONSE; FEVER; HEREDITARY PERIODIC FEVER; MULTICENTER STUDY; PRESCHOOL CHILD; SUBCUTANEOUS DRUG ADMINISTRATION;

ADOLESCENT; ADULT; ANTIBODIES, MONOCLONAL; CHILD; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; DOUBLE-BLIND METHOD; FAMILIAL MEDITERRANEAN FEVER; FEMALE; FEVER; HEREDITARY AUTOINFLAMMATORY DISEASES; HUMANS; INJECTIONS, SUBCUTANEOUS; INTERLEUKIN-1BETA; MALE; MEVALONATE KINASE DEFICIENCY; YOUNG ADULT;

EID: 85047323731     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1706314     Document Type: Article

References (35)

1. Ter Haar NM, 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: 2795-805.
2. Lachmann HJ, Papa R, Gerhold K, et al. The phenotype of TNF receptor-associated autoinflammatory syndrome (TRAPS) at presentation: a series of 158 cases from the Eurofever/EUROTRAPS international registry. Ann Rheum Dis 2014;73:2160-7.
3. Özen S, Batu ED, Demir S. Familial Mediterranean fever: recent developments in pathogenesis and new recommendations for management. Front Immunol 2017;8:253.
4. Sahin S, Yalcin I, Senel S, et al. Assessment life quality of familial Mediterranean fever patients by Short Form-36 and its relationship with disease parameters. Eur Rev Med Pharmacol Sci 2013;17: 958-63.
5. 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 (Baltimore) 2008; 87:301-10.
6. Gattorno M, Obici L, Cattalini M, et al. Canakinumab treatment for patients with active recurrent or chronic TNF receptor-associated periodic syndrome (TRAPS): an open-label, phase II study. Ann Rheum Dis 2017;76:173-8.
7. Papa R, Doglio M, Lachmann HJ, et al. A Web-based collection of genotype-phenotype associations in hereditary recurrent fevers from the Eurofever registry. Orphanet J Rare Dis 2017;12:167.
8. Ozen S, Demirkaya E, Erer B, et al. EULAR recommendations for the management of familial Mediterranean fever. Ann Rheum Dis 2016;75:644-51.
9. Park YH, Wood G, Kastner DL, Chae JJ. Pyrin inflammasome activation and RhoA signaling in the autoinflammatory diseases FMF and HIDS. Nat Immunol 2016;17:914-21.
10. Omenetti A, Carta S, Delfino L, Martini A, Gattorno M, Rubartelli A. Increased NLRP3-dependent interleukin 1β secretion in patients with familial Mediterranean fever: correlation with MEFV genotype. Ann Rheum Dis 2014;73:462-9.
11. Akula MK, Shi M, Jiang Z, et al. Control of the innate immune response by the mevalonate pathway. Nat Immunol 2016; 17:922-9.
12. Drenth JP, Göertz J, Daha MR, van der Meer JW. Immunoglobulin D enhances the release of tumor necrosis factor-alpha, and interleukin-1 beta as well as interleukin-1 receptor antagonist from human mononu-clear cells. Immunology 1996;88:355-62.
13. Frenkel J, Rijkers GT, Mandey SH, et al. Lack of isoprenoid products raises ex vivo interleukin-1beta secretion in hyperimmunoglobulinemia D and periodic fever syndrome. Arthritis Rheum 2002;46: 2794-803.
14. Bachetti T, Chiesa S, Castagnola P, et al. Autophagy contributes to inflammation in patients with TNFR-associated periodic syndrome (TRAPS). Ann Rheum Dis 2013;72:1044-52.
15. Lobito AA, Kimberley FC, Muppidi JR, et al. Abnormal disulfide-linked oligo-merization results in ER retention and altered signaling by TNFR1 mutants in TNFR1-associated periodic fever syndrome (TRAPS). Blood 2006;108:1320-7.
16. Bulua AC, Simon A, Maddipati R, et al. Mitochondrial reactive oxygen species promote production of proinflammatory cytokines and are elevated in TNFR1-associated periodic syndrome (TRAPS). J Exp Med 2011;208:519-33.
17. Simon A, Park H, Maddipati R, et al. Concerted action of wild-type and mutant TNF receptors enhances inflammation in TNF receptor 1-associated periodic fever syndrome. Proc Natl Acad Sci U S A 2010; 107:9801-6.
18. Gül A, Ozdogan H, Erer B, et al. Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever. Arthritis Res Ther 2015;17:243.
19. Brik R, Butbul-Aviel Y, Lubin S, et al. Canakinumab for the treatment of children with colchicine-resistant familial Mediterranean fever: a 6-month open-label, single-arm pilot study. Arthritis Rheumatol 2014;66:3241-3.
20. Alpay N, Sumnu A, Calışkan Y, Yazıcı H, Türkmen A, Gül A. Efficacy of anakinra treatment in a patient with colchicine-resistant familial Mediterranean fever. Rheumatol Int 2012;32:3277-9.
21. Ozen S, Bilginer Y, Aktay Ayaz N, Cal-guneri M. Anti-interleukin 1 treatment for patients with familial Mediterranean fever resistant to colchicine. J Rheumatol 2011;38:516-8.
22. Stankovic Stojanovic K, Delmas Y, Torres PU, et al. Dramatic beneficial effect of interleukin-1 inhibitor treatment in patients with familial Mediterranean fever complicated with amyloidosis and renal failure. Nephrol Dial Transplant 2012;27:1898-901.
23. Arostegui JI, Anton J, Calvo I, et al. Open-label, phase II study to assess the efficacy and safety of canakinumab treatment in active hyperimmunoglobulinemia D with periodic fever syndrome. Arthritis Rheumatol 2017;69:1679-88.
24. 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;58:1516-20.
25. Obici L, Meini A, Cattalini M, et al. Favourable and sustained response to anakinra in tumour necrosis factor receptor-associated periodic syndrome (TRAPS) with or without AA amyloidosis. Ann Rheum Dis 2011;70:1511-2.
26. Livneh A, Langevitz P, Zemer D, et al. Criteria for the diagnosis of familial Mediterranean fever. Arthritis Rheum 1997; 40:1879-85.
27. Shinar Y, Obici L, Aksentijevich I, et al. Guidelines for the genetic diagnosis of hereditary recurrent fevers. Ann Rheum Dis 2012;71:1599-605.
28. Hashkes PJ, Spalding SJ, Giannini EH, et al. Rilonacept for colchicine-resistant or -intolerant familial Mediterranean fever: a randomized trial. Ann Intern Med 2012;157:533-41.
29. 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;69:854-62.
30. Lachmann HJ, Kone-Paut I, Kuem-merle-Deschner JB, et al. Use of canakinumab in the cryopyrin-associated periodic syndrome. N Engl J Med 2009;360:2416-25.
31. Lachmann HJ, Brogan PA. Autoin-flammation: when is familial Mediterranean fever ‘severe’? Nat Rev Rheumatol 2016;12:256-8.
32. Piram M, Koné-Paut I, Lachmann HJ, et al. Validation of the Auto-Inflammatory Diseases Activity Index (AIDAI) for hereditary recurrent fever syndromes. Ann Rheum Dis 2014;73:2168-73.
33. Lachmann HJ, Goodman HJB, Gil-bertson JA, et al. Natural history and outcome in systemic AA amyloidosis. N Engl J Med 2007;356:2361-71.
34. Kuemmerle-Deschner JB, Hoffman H, Hawkins PN, et al. Long-term safety and efficacy of canakinumab in patients with CAPS: final results from the Beta-Confident Registry. Pediatr Rheumatol Online J 2015;13(Suppl 1):P3.
35. Brogan P, Hofer M, Kuemmerle-Deschner J, et al. FRI0503 efficacy and safety of canakinumab in patients with cryopyrin associated periodic syndromes: an open-label, phase-III, extension study. Ann Rheum Dis 2016;75:Suppl 2:620-1.