448
Views
38
CrossRef citations to date
0
Altmetric
Drug Profile

Anakinra for cryopyrin-associated periodic syndrome

&

Recommended reading

  • Masters SL, Simon A, Aksentijevich I, Kastner DL. Horror autoinflammaticus: The molecular pathophysiology of autoinflammatory disease. Annu. Rev. Immunol. 27, 621–668 (2009).
  • Park H, Bourla AB, Kastner DL, Colbert RA, Siegel RM. Lighting the fires within: the cell biology of autoinflammatory diseases. Nat. Rev. Immunol. 12(8), 570–580 (2012).
  • Koné-Paut I, Piram M. Targeting interleukin-1β in CAPS (cryopyrin-associated periodic) syndromes: what did we learn? Autoimmun. Rev. 12 (1), 77–80 (2012).
  • Goldbach-Mansky R. Immunology in clinic review series; focus on autoinflammatory diseases: update on monogenic autoinflammatory diseases: the role of interleukin (IL)-1 and an emerging role for cytokines beyond IL-1. Clin. Exp. Immunol. 167(3), 391–404 (2012).

References

  • Muckle TJ, Wells M. Urticaria, deafness, and amyloidosis: a new heredo-familial syndrome. Q. J. Med. 31, 235–248 (1962).
  • 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. 29(3), 301–305 (2001).
  • Prieur AM, Griscelli C, Lampert F et al. Chronic, infantile, neurological, cutaneous and articular (CINCA) syndrome. A specific entity analysed in 30 patients. Scand. J. Rheumatol. Suppl. 66, 57–68 (1987).
  • Cuisset L, Jeru I, Dumont B et al. Mutations in the autoinflammatory cryopyrin-associated periodic syndrome gene: epidemiological study and lessons from eight years of genetic analysis in France. Ann. Rheum. Dis. 70 (3), 495–499 (2011).
  • Tran TA, Koné-Paut I, Marie I, Ninet J, Cuisset L, Meinzer U. Muckle-Wells syndrome and male hypofertility: a case series. Semin. Arthritis Rheum. 42(3), 327–331 (2012).
  • Aksentijevich ID, 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. 56(4), 1273–1285 (2007).
  • Stych B, Dobrovolny D. Familial cold auto-inflammatory syndrome (FCAS): characterization of symptomatology and impact on patients’ lives. Curr. Med. Res. Opin. 24(6), 1577–1582 (2008).
  • 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. 157(2), 310–315 (2010).
  • Kone-Paut I, Lachmann HJ, Kuemmerle-Deschner JB et al. 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. 13(6), R202 (2011).
  • Touitou I, Kone-Paut I. Autoinflammatory diseases. Best Pract. Res. Clin. Rheumatol. 22(5), 811–829 (2008).
  • Feldmann J, Prieur A, 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. 71(1), 198–203 (2002).
  • Agostini L, Martinon F, Burns K, McDermott MF, Hawkins PN, Tschopp J. NALP3 forms an IL-1β-processing inflammasome with increased activity in Muckle-Wells autoinflammatory disorder. Immunity 20, 319–325 (2004).
  • Janssen R, Verhard E, Lankester A, ten Cate R, van Dissel JT. Enhanced interleukin-1β and interleukin-18 release in a patient with chronic infantile neurologic, cutaneous, articular syndrome. Arthritis Rheum. 50, 3329–3333 (2004).
  • Dinarello CA. Immunological and Inflammatory Functions of the Interleukin-1 Family. Annu. Rev. Immunol. 27, 519–550 (2009).
  • Milhavet F, Cuisset L, Hoffman H et al. The infevers autoinflammatory mutation online registry: update with new genes and functions. Hum. Mutat. 29(6), 803–808 (2008).
  • Rowczenio DM, Trojer H, Russell T et al. Clinical characteristics in subjects with NLRP3 V198M diagnosed at a single UK center and a review of the literature. Arthritis Res. Ther. 15(1), R30 (2013).
  • 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 111, 2132–2141 (2008).
  • Tanaka N, Izawa K, Saito MK et al. High incidence of NLRP3 somatic mosaicism in patients with chronic infantile neurologic, cutaneous, articular syndrome: results of an International Multicenter Collaborative Study. Arthritis Rheum. 63(11), 3625–3632 (2011).
  • 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. 206(5), 1029–1036 (2009).
  • Hawkins PN, Lachmann HJ, McDermott MF. Interleukin-1-receptor antagonist in the Muckle-Wells syndrome. N. Engl. J. Med. 348(25), 2583–2584 (2003).
  • Hawkins PN, Lachmann HJ, Aganna E, McDermott MF. Spectrum of clinical features in Muckle-Wells syndrome and response to anakinra. Arthritis Rheum. 50, 607–612 (2004).
  • 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 364, 1779–1785 (2004).
  • Lovell DJ, Bowyer SL, Solinger AM. Interleukin-1 blockade by anakinra improves clinical symptoms in patients with neonatal onset multisystem inflammatory disease. Arthritis Rheum. 52, 1283–1286 (2005).
  • Goldbach-Mansky R, Dailey NJ, Canna SW et al. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1 inhibition. N. Engl. J. Med. 355, 581–592 (2006).
  • 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. 142, 1591–1597 (2006).
  • 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 47, 309–310 (2008).
  • Kuemmerle-Deschner JB, Tyrrell PN, Koetter I et al. Efficacy and safety of anakinra therapy in pediatric and adult patients with the autoinflammatory Muckle-Wells syndrome. Arthritis Rheum. 63(3), 840–849 (2011).
  • Hoffman HM, Throne ML, Amar NJ et al. Efficacy and safety of rilonacept (interleukin-1 Trap) in patients with cryopyrin-associated periodic syndromes: results from two sequential placebo-controlled studies. Arthritis Rheum. 58(8), 2443–2452 (2008).
  • Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB et al. Use of canakinumab in the cryopyrin-associated periodic syndrome. N. Engl. J. Med. 360(23), 2416–2425 (2009).
  • Lachmann HJ, Goodman HJ, Gilbertson JA et al. Natural history and outcome in systemic AA amyloidosis. N. Engl. J. Med. 356(23), 2361–2371 (2007).
  • Blech M, Peter D, Fischer P et al. One target-two different binding modes: structural insights into gevokizumab and canakinumab interactions to interleukin-1β. J. Mol. Biol. 425(1), 94–111 (2013).
  • Gül A, Tugal-Tutkun I, Dinarello CA et al. Interleukin-1β-regulating antibody XOMA 052 (gevokizumab) in the treatment of acute exacerbations of resistant uveitis of Behcet’s disease: an open-label pilot study. Ann. Rheum. Dis. 71(4), 563–566 (2012).
  • Aksentijevich I, Masters SL, Ferguson PJ et al. An Autoinflammatory Disease with Deficiency of the Interleukin-1–Receptor Antagonist. N. Engl. J. Med. 360(23), 2426–2437 (2009).
  • Lequerré T, Quartier P, Rosellini D et al. Interleukin-1 receptor antagonist (anakinra) treatment in patients with systemic-onset juvenile idiopathic arthritis or adult onset Still disease: preliminary experience in France. Ann. Rheum. Dis. 67(3), 302–308 (2008).
  • Quartier P, Allantaz F, Cimaz R et al. A multicentre, randomised, double-blind, placebo-controlled trial with the interleukin-1 receptor antagonist anakinra in patients with systemic-onset juvenile idiopathic arthritis (ANAJIS trial). Ann. Rheum. Dis. 70(5), 747–754 (2011).
  • Ter Haar N, Lachmann H, Özen S et al. Treatment of autoinflammatory diseases: results from the Eurofever Registry and a literature review. Ann. Rheum. Dis. 72 (5), 678–685 (2012).
  • Bishop R, Hill S, Paul SM 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. 62b, 258–267 (2010).
  • Neven B, Marvillet I, Terrada C et al. 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. 62(1), 258–266 (2010).
  • 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. 64(7), 2375–2386 (2012).
  • Stankovic Stojanovic K, Delmas Y 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. 27(5), 1898–1901 (2012).
  • Olivecrona H, Aldén Raboisson M, Söderberg M et al. Kineret® (anakinra) controls severe disease symptoms in patients with severe Cryopyrin-associated periodic syndrome (CAPS): up to 5 years follow-up data. Ann. Rheum. Dis. 72 (Suppl. 3), 70–71 (2013).
  • Giampietro C, Costedoat Chalumeau N et al. Anakinra in adult-onset Still’s disease: long-term treatment in patients resistant to conventional therapy. Arthritis Care. Res. 65(5), 822–826 (2013).
  • 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. 70(12), 2095–2102 (2011).
  • Kuemmerle-Deschner JB Wittkowski H, Tyrrell PN et al. Treatment of Muckle-Wells syndrome: analysis of two IL-1-blocking regimens. Arthritis Res. Ther. 15(3), R64 (2013)
  • Hoffman HM, Throne ML, Amar NJ et al. Long-term efficacy and safety profile of rilonacept in the treatment of cryopryin-associated periodic syndromes: results of a 72-week open-label extension study. Clin. Ther. 34(10), 2091–2103 (2012).
  • Izawa K, Hijikata A, Tanaka N et al. Detection of base substitution-type somatic mosaicism of the NLRP3 gene with >99.9% statistical confidence by massively parallel sequencing. DNA Res. 19(2), 143–152 (2012).

Websites

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.