The schedule of administration of canakinumab in cryopyrin associated periodic syndrome is driven by the phenotype severity rather than the age

Arthritis Research and Therapy

Volumn 15, Issue 1, 2013, Pages

  Caorsi, Roberta ^a   Lepore, Loredana ^b   Zulian, Francesco ^c   Alessio, Maria ^d   Stabile, Achille ^e   Insalaco, Antonella ^f   Finetti, Martina ^a   Battagliese, Antonella ^d   Martini, Giorgia ^c   Bibalo, Chiara ^b   Martini, Alberto ^a   Gattorno, Marco ^a  

^a UNIVERSITY OF GENOA   (Italy)

^b UNIVERSITY OF TRIESTE   (Italy)

^c UNIVERSITY OF PADOVA   (Italy)

^d FEDERICO II UNIVERSITY HOSPITAL   (Italy)

^e FONDAZIONE POLICLINICO UNIVERSITARIO A GEMELLI IRCCS   (Italy)

^f BAMBINO GESÙ CHILDREN S HOSPITAL   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

CANAKINUMAB; RECOMBINANT INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; MONOCLONAL ANTIBODY;

ADOLESCENT; ADULT; AGE; ARTICLE; CHILD; CINCA SYNDROME; CLINICAL ARTICLE; CLINICAL PRACTICE; COMORBIDITY; CONTROLLED STUDY; DISEASE ASSOCIATION; DISEASE SEVERITY; DIZZINESS; DRUG DOSE INCREASE; DRUG DOSE REDUCTION; DRUG DOSE REGIMEN; DRUG EFFECT; DRUG WITHDRAWAL; FEMALE; FOLLOW UP; HUMAN; MALE; MUCKLE WELLS SYNDROME; PATIENT COMPLIANCE; PATIENT SATISFACTION; PHENOTYPE; PSYCHOLOGICAL ASPECT; QUALITY OF LIFE; SCHOOL CHILD; SIDE EFFECT; TREATMENT RESPONSE; CLINICAL TRIAL; CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES; INFANT; MULTICENTER STUDY; NEWBORN; PRESCHOOL CHILD; RETROSPECTIVE STUDY; TREATMENT OUTCOME;

AGE FACTORS; ANTIBODIES, MONOCLONAL; CHILD; CHILD, PRESCHOOL; CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES; FEMALE; FOLLOW-UP STUDIES; HUMANS; INFANT; INFANT, NEWBORN; MALE; PHENOTYPE; RETROSPECTIVE STUDIES; TREATMENT OUTCOME;

EID: 84874213007     PISSN: 14786354     EISSN: 14786362     Source Type: Journal    
DOI: 10.1186/ar4184     Document Type: Article

References (21)

1. 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, 29:301-305. 10.1038/ng756, 11687797.
2. Neven B, Callebaut I, Prieur AM, Feldmann J, Bodemer C, Lepore L, Derfalvi B, Benjaponpitak S, Vesely R, Sauvain MJ, Oertle S, Allen R, Morgan G, Borkhardt A, Hill C, Gardner-Medwin J, Fischer A, de Saint Basile G. Molecular basis of the spectral expression of CIAS1 mutations associated with phagocytic cell-mediated autoinflammatory disorders CINCA/NOMID, MWS, and FCU. Blood 2004, 103:2809-2815. 10.1182/blood-2003-07-2531, 14630794.
3. Kile RL, Rusk HA. A case of cold urticaria with an unusual family history. JAMA 1940, 114:1067-1068.
4. Muckle TJ, Wells M. Urticaria, deafness, and amyloidosis: a new heredo-familial syndrome. Q J Med 1962, 31:235-248.
5. Prieur AM, Griscelli C. Arthropathy with rash, chronic meningitis, eye lesions, and mental retardation. J Pediatr 1981, 99:79-83. 10.1016/S0022-3476(81)80961-4, 7252669.
6. Martinon F, Tschopp J. Inflammatory caspases: linking an intracellular innate immune system to autoinflammatory diseases. Cell 2004, 117:561-574. 10.1016/j.cell.2004.05.004, 15163405.
7. Gattorno M, Tassi S, Carta S, Delfino L, Ferlito F, Pelagatti MA, D'Osualdo A, Buoncompagni A, Alpigiani MG, Alessio M, Martini A, Rubartelli A. 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, 56:3138-3148. 10.1002/art.22842, 17763411.
8. Hawkins PN, Lachmann HJ, McDermott MF. Interleukin-1-receptor antagonist in the Muckle-Wells syndrome. N Engl J Med 2003, 348:2583-2584. 10.1056/NEJM200306193482523, 12815153.
9. Hoffman HM, Rosengren S, Boyle DL, Cho JY, Nayar J, Mueller JL, Anderson JP, Wanderer AA, Firestein GS. Prevention of cold-associated acute inflammation in familial cold autoinflammatory syndrome by interleukin-1 receptor antagonist. Lancet 2004, 364:1779-1785. 10.1016/S0140-6736(04)17401-1, 15541451.
10. Goldbach-Mansky R, Dailey NJ, Canna SW, Gelabert A, Jones J, Rubin BI, Kim HJ, Brewer C, Zalewski C, Wiggs E, Hill S, Turner ML, Karp BI, Aksentijevich I, Pucino F, Penzak SR, Haverkamp MH, Stein L, Adams BS, Moore TL, Fuhlbrigge RC, Shaham B, Jarvis JN, O'Neil K, Vehe RK, Beitz LO, Gardner G, Hannan WP, Warren RW, Horn W, et al. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1beta inhibition. N Engl J Med 2006, 355:581-592. 10.1056/NEJMoa055137, 16899778.
11. Lepore L, Paloni G, Caorsi R, Alessio M, Rigante D, Ruperto N, Cattalini M, Tommasini A, Zulian F, Ventura A, Martini A, Gattorno M. Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with anakinra. J Pediatr 2010, 157:310-315. 10.1016/j.jpeds.2010.02.040, 20472245.
12. Neven B, Marvillet I, Terrada C, Ferster A, Boddaert N, Couloignier V, Pinto G, Pagnier A, Bodemer C, Bodaghi B, Tardieu M, Prieur AM, Quartier P. Long-term efficacy of the interleukin-1 receptor antagonist anakinra in ten patients with neonatal-onset multisystem inflammatory disease/chronic infantile neurologic, cutaneous, articular syndrome. Arthritis Rheum 2010, 62:258-267.
13. Sibley CH, Plass N, Snow J, Wiggs E, Brewer C, King K, Zalewski C, Kim HJ, Bishop R, Hill S, Paul SM, Kicker P, Phillips Z, Dolan JG, Widemann B, Jayaprakash N, Pucino F, Stone DL, Chapelle D, Snyder C, Butman JA, Wesley R, Goldbach-Mansky R. Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease (NOMID) treated with anakinra. Arthritis Rheum 2012, 64:2375-86. 10.1002/art.34409, 22294344.
14. Hoffman HM, Throne ML, Amar NJ, Sebai M, Kivitz AJ, Kavanaugh A, Weinstein SP, Belomestnov P, Yancopoulos GD, Stahl N, Mellis SJ. 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, 58:2443-2452. 10.1002/art.23687, 18668535.
15. Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, Leslie KS, Hachulla E, Quartier P, Gitton X, Widmer A, Patel N, Hawkins PN. Use of canakinumab in the cryopyrin-associated periodic syndrome. N Engl J Med 2009, 360:2416-2425. 10.1056/NEJMoa0810787, 19494217.
16. Kuemmerle-Deschner JB, Hachulla E, Cartwright R, Hawkins PN, Tran TA, Bader-Meunier B, Hoyer J, Gattorno M, Gul A, Smith J, Leslie KS, Jiménez S, Morell-Dubois S, Davis N, Patel N, Widmer A, Preiss R, Lachmann HJ. 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, 70:2095-2102. 10.1136/ard.2011.152728, 21859692.
17. Caroli F, Pontillo A, D'Osualdo A, Travan L, Ceccherini I, Crovella S, Alessio M, Stabile A, Gattorno M, Tommasini A, Martini A, Lepore L. Clinical and genetic characterization of Italian patients affected by CINCA syndrome. Rheumatology (Oxford) 2007, 46:473-478.
18. Tassi S, Carta S, Delfino L, Caorsi R, Martini A, Gattorno M, Rubartelli A. Altered redox state of monocytes from cryopyrin-associated periodic syndromes causes accelerated IL-1beta secretion. Proc Natl Acad Sci USA 2010, 107:9789-9794. 10.1073/pnas.1000779107, 2906851, 20445104.
19. Fox E, Jayaprakash N, Pham TH, Rowley A, McCully CL, Pucino F, Goldbach-Mansky R. The serum and cerebrospinal fluid pharmacokinetics of anakinra after intravenous administration to non-human primates. J Neuroimmunol 2010, 223:138-140. 10.1016/j.jneuroim.2010.03.022, 2887614, 20421138.
20. Kone-Paut I, Lachmann HJ, Kuemmerle-Deschner JB, Hachulla E, Leslie KS, Mouy R, Ferreira A, Lheritier K, Patel N, Preiss R, Hawkins PN. 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:R202. 10.1186/ar3535, 3334655, 22152723.
21. Ruperto N, Quartier P, Wulffraat N, Woo P, Ravelli A, Mouy R, Bader-Meunier B, Vastert SJ, Noseda E, D'Ambrosio D, Lecot J, Chakraborty A, Martini A, Chioato A. 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, 64:557-67. 10.1002/art.33342, 21953497.