Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap).
Cryopyrin-associated periodic syndromes (CAPS) are a group of inherited inflammatory disorders consisting of familial cold-induced autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and neonatal-onset multisystem inflammatory disease (NOMID; also known as chronic infantile neurologic, cutaneous, articular [CINCA] syndrome). These rare disorders are associated with heterozygous mutations in the NLRP3 (CIAS1) gene, which encodes the protein NALP3 or cryopyrin, and inflammation driven by excessive production of the cytokine interleukin-1beta (IL-1beta). Amyloidosis is a serious complication with 25% of MWS patients developing amyloidosis, with occasional fatal consequences, whilst up to 20% of CINCA/NOMID patients die from various complications, before reaching the early adulthood. In some CINCA/NOMID adult survivors amyloidosis can also occur. Prior to the discovery of the CIAS1 gene mutations and the advent of IL-1 targeted therapy, treatment was aimed at suppressing inflammation, with limited success. The selective blockade of IL-1beta, with anakinra (IL-1 receptor antagonist), not only provided supportive evidence for the role of IL-1beta in CAPS, but also demonstrated the efficacy of targeting IL-1beta for treatment of these conditions. In February, 2008, 'Orphan Drug' approval from the Food and Drug Administration (FDA) for rilonacept (IL-1 Trap/Arcalyst(), Regeneron Pharmaceuticals, Inc) was given for the treatment of two CAPS disorders, FCAS and MWS in adults and children 12 years and older, making rilonacept the first therapy approved for the treatment of CAPS.
Church
Leigh D
LD
Department of Rheumatology, Division of Immunity, Infection and Inflammation, Institute for Biomedical Research, The University of Birmingham, Birmingham, UK.
Savic
Sinisa
S
McDermott
Michael F
MF
Radiology. 1986 Sep;160(3):741-6
3737913
Arch Dermatol. 2008 Mar;144(3):392-402
18347298
Q J Med. 1962 Apr;31:235-48
14476827
Arthritis Rheum. 2008 Aug;58(8):2432-42
18668591
J Allergy Clin Immunol. 2007 Apr;119(4):991-6
17320940
Am J Hum Genet. 2002 Jun;70(6):1498-506
11992256
Arch Ophthalmol. 2000 Oct;118(10):1386-92
11030821
N Engl J Med. 2003 Jun 19;348(25):2583-4
12815153
Arthritis Rheum. 2004 Feb;50(2):607-12
14872505
Skeletal Radiol. 1989;18(5):359-64
2781338
Am J Hum Genet. 2002 Jul;71(1):198-203
12032915
Scand J Rheumatol Suppl. 1987;66:57-68
3482735
Cell Death Differ. 2007 Sep;14(9):1583-9
17599094
Ann Rheum Dis. 2006 Apr;65(4):533-4
16531551
J Pediatr. 1997 Apr;130(4):513-5
9108844
Arthritis Rheum. 2008 Aug;58(8):2443-52
18668535
Arch Dermatol. 2006 Dec;142(12):1591-7
17178985
Nat Biotechnol. 2008 May;26(5):485
18464767
N Engl J Med. 2006 Aug 10;355(6):581-92
16899778
Proc Natl Acad Sci U S A. 2008 Jul 1;105(26):9035-40
18577586
Nature. 2006 Mar 9;440(7081):237-41
16407889
Arthritis Rheum. 2002 Dec;46(12):3340-8
12483741
Arthritis Rheum. 2002 Sep;46(9):2445-52
12355493
Br J Dermatol. 2004 Jul;151(1):99-104
15270877
Nat Clin Pract Rheumatol. 2008 Sep;4(9):481-9
18665151
Proc R Soc Med. 1973 Nov;66(11):1070-1
4777015
J Cutan Med Surg. 2008 Jan-Feb;12(1):8-16
18258152
Am J Hum Genet. 1999 Oct;65(4):1054-9
10486324
Nat Rev Immunol. 2006 Mar;6(3):183-95
16498449
Arthritis Rheum. 2004 Aug;50(8):2708-9
15334488
Nature. 2008 Jun 19;453(7198):1122-6
18496530
Expert Rev Clin Immunol. 2008 Jul;4(4):425-8
20477570
Nat Genet. 2001 Nov;29(3):301-5
11687797
Curr Pharm Des. 2003;9(14):1095-106
12769749
Immunity. 2004 Mar;20(3):319-25
15030775
Health Technol Assess. 2004 May;8(18):iii-iv, ix-x, 1-105
15130461
Rheumatology (Oxford). 2008 Mar;47(3):309-10
18174231
Mol Cell. 2002 Aug;10(2):417-26
12191486
Am J Hum Genet. 2000 May;66(5):1693-8
10741953
J Nephrol. 2003 May-Jun;16(3):435-7
12832748
Nat Med. 2003 Jan;9(1):47-52
12483208
Curr Opin Allergy Clin Immunol. 2002 Dec;2(6):511-6
14752334
Nat Clin Pract Rheumatol. 2008 Jan;4(1):34-42
18172447
Lancet. 2004 Nov 13-19;364(9447):1779-85
15541451
Nature. 2008 Mar 6;452(7183):103-7
18288107
Pediatr Dermatol. 2007 Jan-Feb;24(1):85-9
17300660
Arthritis Res Ther. 2008;10(3):R67
18534016
Nephrol Dial Transplant. 2002 Jul;17(7):1212-7
12105243
Arthritis Rheum. 2006 May;54(5):1697-700
16646042