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)

Clinical and Experimental Rheumatology

Volumn 31, Issue 2, 2013, Pages 0302-0309

  Imagawa, Tomoyuki ^a   Nishikomori, Ryuta ^b   Takada, Hidetoshi ^c   Takeshita, Saoko ^a   Patel, Neha ^d   Kim, Dennis ^d   Lheritier, Karine ^d   Heike, Toshio ^b   Hara, Toshiro ^c   Yokota, Shumpei ^a  

^a YOKOHAMA CITY UNIVERSITY HOSPITAL   (Japan)

^b KYOTO UNIVERSITY   (Japan)

^c KYUSHU UNIVERSITY   (Japan)

^d NOVARTIS PHARMA AG   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

C REACTIVE PROTEIN; CANAKINUMAB; SERUM AMYLOID A; AUTACOID; BIOLOGICAL MARKER; IMMUNOSUPPRESSIVE AGENT; MONOCLONAL ANTIBODY;

ACNE; ADOLESCENT; ADULT; ARTICLE; CHILD; CINCA SYNDROME; CLINICAL ARTICLE; COUGHING; DIARRHEA; DISEASE ACTIVITY; DRUG DOSE INCREASE; DRUG DOSE TITRATION; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRY SKIN; EPSTEIN BARR VIRUS INFECTION; FEMALE; GASTROENTERITIS; GASTROINTESTINAL DISEASE; HUMAN; HYPERTENSION; IMMUNOGENICITY; INFECTION; INFESTATION; INJECTION SITE REACTION; JAPANESE; MALE; MEDIASTINUM DISEASE; MUCKLE WELLS SYNDROME; NEUROLOGIC DISEASE; OPEN STUDY; PARVOVIRUS INFECTION; PHASE 3 CLINICAL TRIAL; PNEUMONIA; PRESCHOOL CHILD; PRIORITY JOURNAL; RELAPSE; REMISSION; RESPIRATORY TRACT DISEASE; RHINOPHARYNGITIS; RHINORRHEA; SCHOOL CHILD; SKIN DISEASE; STOMATITIS; THORAX DISEASE; UPPER ABDOMINAL PAIN; UPPER RESPIRATORY TRACT INFECTION; URTICARIA; VASCULAR DISEASE; BLOOD; DRUG ADMINISTRATION; IMMUNOLOGY; JAPAN; MIDDLE AGED; RECURRENT DISEASE; SUBCUTANEOUS DRUG ADMINISTRATION; TIME; TREATMENT OUTCOME;

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

MLCS; MLOWN;

EID: 84875796225     PISSN: 0392856X     EISSN: 1593098X     Source Type: Journal    
DOI: None     Document Type: Article

References (27)

1. AKSENTIJEVICH I, D PUTNAM C, REMMERS EF et al.: The clinical continuum of cryopyrinopathies: novel CIAS1 mutations in North American patients and a new cryopyrin model. Arthritis Rheum 2007; 56: 1273-85.
2. 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.
3. 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.
4. 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.
5. 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.
6. 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.
7. DINARELLO CA: Mutations in cryopyrin: bypassing roadblocks in the caspase 1 inflammasome for interleukin-1beta secretion and disease activity. Arthritis Rheum 2007; 56: 2817-22.
8. GATTORNO M, TASSI S, CARTA S et al.: Pattern of interleukin-1beta secretion in response to lipopolysaccharide and ATP before and after interleukin-1 blockade in patients with CIAS1 mutations. Arthritis Rheum 2007; 56: 3138-48.
9. 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.
10. 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.
11. 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.
12. 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.
13. 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.
14. MAKSIMOVIC L, STIRNEMANN J, CAUX F et al.: New CIAS1 mutation and anakinra efficacy in overlapping of Muckle-Wells and familial cold autoinflammatory syndromes. Rheumatology (Oxford) 2008; 47: 309-10.
15. O'CONNELL SM, O'REGAN GM, BOLGER T et al.: Response to IL-1-receptor antagonist in a child with familial cold autoinflammatory syndrome. Pediatr Dermatol 2007: 24: 85.
16. HOFFMAN HM: Rilonacept for the treatment of cryopyrin-associated periodic syndromes (CAPS). Expert Opin Biol Ther 2009; 9: 519-31.
17. GOLDBACH-MANSKY R, SHROFF SD, WILSON M et al.: A pilot study to evaluate the safety and efficacy of the long-acting interleukin- 1 inhibitor rilonacept (interleukin-1 Trap) in patients with familial cold autoinflammatory syndrome. Arthritis Rheum 2008; 58: 2432-42.
18. LACHMANN HJ, LOWE P, FELIX SD et al.: In vivo regulation of interleukin 1beta 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-1beta for the potential treatment of inflammatory disorders. Curr Opin Mol Ther 2009; 11: 81-9.
20. Novartis. Ilaris prescribing information. http://wwwpharmausnovartiscom/product/pi/pdf/ilarispdf2010, Accessed Feb 17. http://wwwpharmausnovartiscom/product/pi/pdf/ilarispdf
21. 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.
22. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsare-DevelopedandApproved/DrugandBiologicApprovalReports/PriorityNDAandBLAApprovals/UCM090995.pdfhttp://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApproval-Reports/PriorityNDAandBLAApprovals/UCM090995.pdf accessed July 2010.
23. http://www.ema.europa.eu/pdfs/human/opinion/illaris_44782909en.pdf,http://www.ema.europa.eu/pds/human/opinion/illaris_44782909en.pdf. accessed2010.
24. NEVEN B, PRIEUR AM, QUARTIER DIT MAIRE P.: Cryopyrinopathies: update on pathogenesis and treatment. Nat Clin Pract Rheumatol 2008; 4: 481-9.
25. KUEMMERLE-DESCHNER JB, RAMOS E, BLANK N et al.: Canakinumab (ACZ885, a fully human IgG1 anti-IL-1β mAb) induces sustained remission in pediatric patients with cryopyrin-associated periodic syndrome (CAPS). Arthritis Res Ther 2011; 13: R34 [Epub ahead of print].
26. KUEMMERLE-DESCHNER JB, HACHULLA E, CARTWRIGHT R et al.: Two-year results from an open-label, multicentre, phase III study evaluating the safety and efficacy of canakinumab in patients with cryopyrin-associated periodic syndrome across different severity phenotypes. Ann Rheum Dis 2011; 70: 2095-102.
27. FDA DRUG SAFETY COMMUNICATION: Early communication about an ongoing safety review of Tumor Necrosis Factor (TNF) blockers (marked as Remicade, Embrel, Humira, and Cimzia).