Long-term safety and efficacy of canakinumab in cryopyrin-associated periodic syndrome: Results from an open-label, phase III pivotal study in Japanese patients

Clinical and Experimental Rheumatology

Volumn 35, Issue , 2017, Pages S19-S26

  Yokota, Shumpei ^a   Imagawa, Tomoyuki ^a   Nishikomori, Ryuta ^b   Takada, Hidetoshi ^c   Abrams, Ken ^d   Lheritier, Karine ^d   Heike, Toshio ^b   Hara, Toshiro ^e  

^a TOKYO MEDICAL UNIVERSITY   (Japan)

^b KYOTO UNIVERSITY   (Japan)

^c KYUSHU UNIVERSITY   (Japan)

^d NOVARTIS PHARMA AG   (Switzerland)

^e FUKUOKA CHILDREN S HOSPITAL   (Japan)

Author keywords

Auto inflammatory syndrome; canakinumab; Cryopyrin associated periodic syndrome; Interleukin 1

Indexed keywords

CANAKINUMAB; IMMUNOSUPPRESSIVE AGENT; MONOCLONAL ANTIBODY;

ABDOMINAL PAIN; ACNE; ACUTE OTITIS MEDIA; ADOLESCENT; ADVERSE EVENT; AGE; ALLERGIC CONJUNCTIVITIS; ALLERGIC RHINITIS; ALLERGY; ANTIINFLAMMATORY ACTIVITY; APHTHOUS STOMATITIS; ARTICLE; ASTHMA; BRONCHITIS; CHILD; CINCA SYNDROME; CONJUNCTIVITIS; CONSTIPATION; COUGHING; DENTAL CARIES; DIARRHEA; DISEASE ACTIVITY; DRUG EFFICACY; DRUG RESPONSE; DRUG SAFETY; DRUG WITHDRAWAL; DRY SKIN; ECZEMA; FEMALE; GASTROENTERITIS; HEADACHE; HEARING IMPAIRMENT; HEAT RASH; HORDEOLUM; HUMAN; HYPERTENSION; IMPETIGO; INFECTION; INFLUENZA; JAPANESE (PEOPLE); LONG TERM CARE; MALE; OROPHARYNX PAIN; OTITIS MEDIA; PHASE 3 CLINICAL TRIAL; PNEUMONIA; PRIORITY JOURNAL; RELAPSE; RHINOPHARYNGITIS; RHINORRHEA; SINUSITIS; STOMATITIS; TREATMENT DURATION; UPPER RESPIRATORY TRACT INFECTION; URTICARIA; VOMITING; ADULT; CLINICAL TRIAL; DISEASE FREE SURVIVAL; DRUG ADMINISTRATION; IMMUNOLOGY; JAPAN; MIDDLE AGED; MULTICENTER STUDY; PRESCHOOL CHILD; RECURRENT DISEASE; REMISSION; SUBCUTANEOUS DRUG ADMINISTRATION; TIME FACTOR; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ADULT; ANTIBODIES, MONOCLONAL; CHILD; CHILD, PRESCHOOL; CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES; DISEASE-FREE SURVIVAL; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INJECTIONS, SUBCUTANEOUS; JAPAN; MALE; MIDDLE AGED; RECURRENCE; REMISSION INDUCTION; TIME FACTORS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 85035814255     PISSN: 0392856X     EISSN: 1593098X     Source Type: Journal    
DOI: None     Document Type: Article

References (30)

1. HOFFMAN HM, WANDERER AA, BROIDE DH: Familial cold autoinflammatory syndrome: phenotype and genotype of an autosomal dominant periodic fever. J Allergy Clin Immunol 2001; 108: 615-20
2. MUCKLE TJ, WELLS M: Urticaria, deafness, and amyloidosis: a new heredo-familial syndrome. Q J Med 1962; 31: 235-48
3. PRIEUR AM, GRISCELLI C, LAMPERT F et al.: A chronic, infantile, neurological, cutaneous and articular (CINCA) syndrome: a specific entity analysed in 30 patients. Scand J Rheumatol 1987; 66 (Suppl.): 57-68
4. GOLDBACH-MANSKY R, DAILEY NJ, CANNA SW et al.: Neonatal-onset multisystem inflammatory disease responsive to interleukin-1 beta inhibition. N Engl J Med 2006; 355: 581-92
5. AGANNA E, MARTINON F, HAWKINS PN et al.: Association of mutations in the NALP3/CIAS1/PYPAF1 gene with a broad phenotype including recurrent fever, cold sensitivity, sensorineural deafness, and AA amyloidosis. Arthritis Rheum 2002; 46: 2445-52
6. DODé C, LE DÛ N, CUISSET L et al.: New mutations of CIAS1 that are responsible for Muckle-Wells syndrome and familial cold urticaria: a novel mutation underlies both syndromes. Am J Hum Genet 2002; 70: 1498-506
7. HOFFMAN HM, MUELLER JL, BROIDE DH, WANDERER AA, KOLODNER RD: Mutation of a new gene encoding a putative pyrin-like protein causes familial cold autoinflammatory syndrome and Muckle-Wells syndrome. Nat Genet 2001; 29: 301-5
8. FELDMANN J, PRIEUR AM, QUARTIER P et al.: Chronic infantile neurological cutaneous and articular syndrome is caused by mutations in CIAS1, a gene highly expressed in polymorphonuclear cells and chondrocytes. Am J Hum Genet 2002; 71: 198-203
9. AKSENTIJEVICH I, NOWAK M, MALLAH M et al.: De novo CIAS1 mutations, cytokine activation, and evidence for genetic heterogeneity in patients with neonatal-onset multisystem inflammatory disease (NOMID): a new member of the expanding family of pyrin-associated autoinflammatory diseases. Arthritis Rheum 2002; 46: 3340-8
10. GATTORNO M, TASSI S, CARTA S et al.: Pattern of interleukin-1 beta secretion in response to lipopolysaccharide and ATP before and after interleukin-1 blockade in patients with CIAS1 mutations. Arthritis Rheum 2007; 56: 3138-48
11. DINARELLO CA: Biologic basis for interleukin-1 in disease. Blood 1996; 87: 2095-147
12. YAMAZAKI T, MASUMOTO J, AGEMATSU K et al.: Anakinra improves sensory deafness in a Japanese patient with Muckle-Wells syndrome, possibly by inhibiting the cryopyrin inflammasome. Arthritis Rheum 2008; 58: 864-8
13. LESLIE KS, LACHMANN HJ, BRUNING E et al.: Phenotype, genotype, and sustained response to anakinra in 22 patients with autoinflammatory disease associated with CIAS-1/NALP3 mutations. Arch Dermatol 2006; 142: 1591-7
14. ROSS JB, FINLAYSON LA, KLOTZ PJ et al.: Use of anakinra (Kineret) in the treatment of familial cold autoinflammatory syndrome with a 16-month follow-up. J Cutan Med Surg 2008; 12: 8-16
15. WITTKOWSKI H, KUEMMERLE-DESCHNER B, AUSTERMANN J et al.: MRP8 and MRP14, phagocyte-specific danger signals, are sensitive biomarkers of disease activity in cryopyrin-associated periodic syndromes. Arthritis Rheum Dis 2011; 70: 2075-81
16. HOFFMAN HM, THRONE ML, AMAR NJ et al.: Efficacy and safety of rilonacept (interleukin-1 Trap) in patients with cryopyrinassociated periodic syndromes: results from two sequential placebo-controlled studies. Arthritis Rheum 2008; 58: 2443-52
17. PALONI G, PASTORE S, TOMMASINI A, LEPORE L, TADDIO A: Delayed reactivation of chronic infantile neurologic, cutaneous, articular syndrome (CINCA) in a patient with somatic mosaicism of CIAS1/NLRP3 gene after withdrawal of anti-IL-1 beta therapy. Clin Exp Rheumatol 2015; 33: 766
18. LACHMANN HJ, LOWE P, FELIX SD et al.: In vivo regulation of interleukin 1 beta in patients with cryopyrin-associated periodic syndromes. J Exp Med 2009; 206: 1029-36
19. CHURCH LD, McDERMOTT MF: Canakinumab, a fully-human mAb against IL-1 beta for the potential treatment of inflammatory disorders. Curr Opin Mol Ther 2009; 11: 81-9
20. ILARIS [Prescribing information]. http://www.pharma.us.novartis.com/products/ilaris. shtml. Accessed January 2016
21. ILARIS [Prescribing information]. US Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/125319s081lbl.pdf. Accessed January 2016
22. ILARIS [Summary of product characteristics]. European Medicines Agency. http://www. ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/001109/WC500031680.pdf. Accessed January 2016
23. Pharmaceuticals and Medical Devices Agency. http://www.pmda.go.jp/english/service/list_s.html. Accessed January 2016
24. LACHMANN HJ, KONE-PAUT I, KUEMMERLE-DESCHNER JB et al.: Use of canakinumab in the cryopyrin-associated periodic syndrome. N Engl J Med 2009; 360: 2416-25
25. IMAGAWA T, NISHIKOMORI R, TAKADA H et al.: Safety and efficacy of canakinumab in Japanese patients with phenotypes of cryopyrin-associated periodic syndrome as established in the first open-label, phase-3 pivotal study (24-week results). Clin Exp Rheumatol 2013; 31: 302-9
26. CAORSI R, LEPORE L, ZULIAN F et al.: The schedule of administration of canakinumab in cryopyrin associated periodic syndrome is driven by the phenotype severity rather than the age. Arthritis Res Ther 2013; 15: R33
27. ANTON J, CALVO I, FERNáNDEZ-MARTIN J et al.: Efficacy and safety of canakinumab in cryopyrin-associated periodic syndromes: results from a Spanish cohort. Clin Exp Rheumatol 2015; 33 (Suppl. 94): S67-71
28. RUPERTO N, BRUNNER HI, QUARTIER P et al.: Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med 2012; 367: 2396-406
29. LEPORE L, PALONI G, CAORSI R et al.: Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with Anakinra. J Pediatr 2010; 157: 310-15
30. SIBLEY CH, PLASS N, SNOW J et al.: Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra. A cohort study to determine three-and five-year outcomes. Arthritis Rheum 2012; 64: 2375-86