444
Views
55
CrossRef citations to date
0
Altmetric
Drug Evaluations

Rilonacept for the treatment of cryopyrin-associated periodic syndromes (CAPS)

, MD
Pages 519-531 | Published online: 03 Apr 2009

Bibliography

  • Hull KM, Shoham N, Chae JJ, et al. The expanding spectrum of systemic autoinflammatory disorders and their rheumatic manifestations. Curr Opin Rheumatol 2003;15:61-9
  • Aksentijevich I, Putnam DC, 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
  • Hoffman HM. Familial cold autoinflammatory syndrome. Orphanet Encyclopedia 2005;1-4
  • 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
  • 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
  • Goldbach-Mansky R, Dailey NJ, et al. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1β inhibition. N Engl J Med 2006;355(6):581-92
  • Hawkins PN, Lachmann HJ, Aganna E, McDermott MF. Spectrum of clinical features in Muckle–Wells syndrome and response to anakinra. Arthritis Rheum 2004;50:607-12
  • 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
  • Kastner DL. Hereditary periodic fever syndromes. Hematology 2005 – American Society of Hematology Education Program. 2005;74-81
  • Boschan C, Witt O, Lohse P, et al. Neonatal-onset multisystem inflammatory disease (NOMID) due to a novel S331R mutation of the CIAS1 gene and response to interleukin-1 receptor antagonist treatment. Am J Med Genet A 2006;140:883-6
  • Stefaniak R, Maurer M, Mahony M, et al. Burden of disease in patients with Muckle Wells Syndrome (MWS): baseline results from a natural history study. Abstract presented at American College of Rheumatology, 2008
  • Stych B, Dobrovolny D. Familial cold auto-inflammatory syndrome (FCAS): characterization of symptomatology and impact on patients' lives. Curr Med Res Opin 2008;24:1577-82
  • Hoffman HM, Wolfe F, Belomestnov P, Mellis SJ. Cryopyrin-associated periodic syndromes: development of a patient-reported outcomes instrument to assess the pattern and severity of clinical disease activity. Curr Med Res Opin 2008;29:2531-43
  • 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
  • Hoffman HM, Mueller JL, Broide DH, et al. Mutation of a new gene encoding a putative pyrin-like protein causes familial cold autoinflammatory syndrome and Muckle-Wells syndrome. Nature 2001;29:301-5
  • Milhavet F, Cuisset L, Hoffman HM, et al. The infevers autoinflammatory mutation online registry: update with new genes and functions. Hum Mutat 2008;29:803-8
  • Hoffman HM, Gregory SG, Mueller JL, et al. Fine structure mapping of CIAS1: identification of an ancestral haplotype and a common FCAS mutation, L353P. Hum Genet 2003;112(2):209-16
  • Hoffman HM, Patel DD. Genomic-based therapy: targeting interleukin-1 for autoinflammatory diseases. Arthritis Rheum 2004;50:345-9
  • Saito M, Nishikomori R, Kambe N, et al. Disease-associated CIAS1 mutations induce monocyte death, revealing low-level mosaicism in mutation-negative cryopyrin-associated periodic syndrome patients. Blood 2008;111:2132-41
  • Boutin H, Kimber I, Rothwell NJ, Pinteaux E. The expanding interleukin-1 family and its receptors: do alternative IL-1 receptor/signaling pathways exist in the brain? Mol Neurobiol 2003;27:239-48
  • Dinarello CA. Biologic basis for interleukin-1 in disease. Blood 1996;87:2095-147
  • Dinarello CA. Unraveling the NALP-3/IL-1β inflammasome: a big lesson from a small mutation. Immunity 2004;20:243-6
  • Agostini L, Martinon F, Burns K, et al. NALP3 forms an IL-1β-processing inflammasome with increased activity in Muckle–Wells autoinflammatory disorder. Immunity 2004;20:319-25
  • Janssen R, Verhard E, Lankester A, et al. Enhanced interleukin-1β and interleukin-18 release in a patient with chronic infantile neurologic, cutaneous, articular syndrome. Arthritis Rheum 2004;50:3329-33
  • Hawkins PN, Lachmann HJ, McDermott MF. Interleukin-1 receptor antagonist in the Muckle–Wells syndrome. N Engl J Med 2003;48:2583-4
  • Hoffman HM, Throne ML, Amar NJ, et al. Efficacy and safety of rilonacept (IL-1 trap) in cryopyrin-associated periodic syndromes (CAPS): results from two sequential placebo-controlled studies. Arthritis Rheum 2008;58:2443-52
  • 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;24:85-9
  • Hoffman HM, Rosengren S, Boyle DL, et al. Prevention of cold-associated acute inflammation in familial cold autoinflammatory syndrome by interleukin-1 receptor antagonist. Lancet 2004;364:1779-85
  • 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(1):8-16
  • Chang DM, Chang SY, Yeh MK, Lai JH. The pharmacokinetics of interleukin-1 receptor antagonist in Chinese subjects with rheumatoid arthritis. Pharmacol Res 2004;50:371-6
  • Cohen S, Hurd E, Cush J, et al. Treatment of rheumatoid arthritis with anakinra, a recombinant human interleukin-1 receptor antagonist, in combination with methotrexate: results of a twenty-four-week, multicenter, randomized, double-blind, placebo-controlled trial. Arthritis Rheum 2002;46:614-24
  • Burger D, Dayer JM, Palmer G, Gabay C. Is IL-1 a good therapeutic target in the treatment of arthritis? Best Pract Res Clin Rheumatol 2006;20:879-96
  • Pope RM, Tschopp J. The role of interleukin-1 and the inflammasome in gout: implications for therapy. Arthritis Rheum 2007;56(10):3183-8
  • Dinarello CA. Blocking IL-1 in systemic inflammation. J Exp Med 2005;201:1355-9
  • Dinarello CA. Setting the cytokine trap for autoimmunity. Nat Med 2003;1:20-2
  • Economides AN, Carpenter LR, Rudge JS, et al. Cytokine traps: multi-component, high-affinity blockers of cytokine action. Nat Med 2003;9:47-52
  • ARCALYST™ (rilonacept) full prescribing information. Regeneron Pharmaceuticals, Inc. 2008
  • Data on file, Regeneron Pharmaceuticals, Tarrytown, NY
  • Genovese MC, Cohen S, Moreland L, et al. Combination therapy with etanercept and anakinra in the treatment of patients with rheumatoid arthritis who have been treated unsuccessfully with methotrexate. Arthritis Rheum 2004;50:1412-9
  • 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
  • Hoffman HM, Amar NJ, Cartwright RC, et al. Durability of response to rilonacept (IL-1 Trap) in a Phase 3 study of patients with cryopyrin-associated periodic syndromes (CAPS): familial cold autoinflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS) [abstract]. American Academy of Allergy, Asthma & Immunology 8th Annual Meeting, March 14-18, 2008, Philadelphia, PA
  • Braddock M, Quinn A, Canvin J. Therapeutic potential targeting IL-1 and IL-18 inflammation. Expert Opin Biol Ther 2004;4:847-60
  • Bresnihan B, Alvaro-Gracia JM, Cobby M, et al. Treatment of rheumatoid arthritis with recombinant human interleukin-1 receptor antagonist. Arthritis Rheum 1998;41:2196-204
  • Jiang Y, Genant HK, Watt I, et al. A multicenter, double-blind, dose-ranging, randomized, placebo-controlled study of recombinant human interleukin-1 receptor antagonist in patients with rheumatoid arthritis: radiologic progression and correlation of Genant and Larsen scores. Arthritis Rheum 2000;43:1001-9
  • Rosengren S, Mueller JL, Anderson JP, et al. Monocytes from familial cold autoinflammatory syndrome patients are activated by mild hypothermia. J Allergy Clin Immunol 2007;119:991-6
  • Picard C, Puel A, Bonnet M, et al. Pyogenic bacterial infections in humans with IRAK-4 deficiency. Science 2003;299:2076-9
  • Wanderer A. An “allergy” to cold. Hosp Pract 1979;14:135-7

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.