title: Biological treatments: new weapons in the management of monogenic autoinflammatory disorders.
abstract: Treatment of monogenic autoinflammatory disorders, an expanding group of hereditary diseases characterized by apparently unprovoked recurrent episodes of inflammation, without high-titre autoantibodies or antigen-specific T cells, has been revolutionized by the discovery that several of these conditions are caused by mutations in proteins involved in the mechanisms of innate immune response, including components of the inflammasome, cytokine receptors, receptor antagonists, and oversecretion of a network of proinflammatory molecules. Aim of this review is to synthesize the current experience and the most recent evidences about the therapeutic approach with biologic drugs in pediatric and adult patients with monogenic autoinflammatory disorders.
authors: Research Center of Systemic Autoimmune and Autoinflammatory Diseases, Rheumatology Unit, Policlinico Le Scotte, University of Siena, Viale Bracci 1, 53100 Siena, Italy.
authors: Antonio
authors: A
authors: Vitale
authors: Donato
authors: D
authors: Rigante
authors: Orso Maria
authors: OM
authors: Lucherini
authors: Francesco
authors: F
authors: Caso
authors: Isabella
authors: I
authors: Muscari
authors: Flora
authors: F
authors: Magnotti
authors: Maria Giuseppina
authors: MG
authors: Brizi
authors: Susanna
authors: S
authors: Guerrini
authors: Maria
authors: M
authors: Patti
authors: Leonardo
authors: L
authors: Punzi
authors: Mauro
authors: M
authors: Galeazzi
authors: Luca
authors: L
authors: Cantarini
mesh_terms: D000152:Acne Vulgaris / Q000628:therapy; D000742:Anemia, Dyserythropoietic, Congenital / Q000628:therapy; D001168:Arthritis; D001170:Arthritis, Infectious / Q000628:therapy; D001688:Biological Products / Q000627:therapeutic use*; D003389:Cranial Nerve Diseases / Q000628:therapy; D056587:Cryopyrin-Associated Periodic Syndromes / Q000628:therapy; D010505:Familial Mediterranean Fever / Q000628:therapy; D005334:Fever; D056660:Hereditary Autoinflammatory Diseases / Q000628:therapy; D006801:Humans; D007113:Immunity, Innate; D007153:Immunologic Deficiency Syndromes; D007249:Inflammation / Q000378:metabolism* / Q000628:therapy*; D047908:Intracellular Signaling Peptides and Proteins / Q000378:metabolism; D054078:Mevalonate Kinase Deficiency / Q000628:therapy; D009154:Mutation; D010019:Osteomyelitis / Q000628:therapy; D017511:Pyoderma Gangrenosum / Q000628:therapy; D017472:Receptors, Interleukin-1 / Q000378:metabolism; D012507:Sarcoidosis; D013585:Synovitis / Q000628:therapy; D013601:T-Lymphocytes / Q000378:metabolism; D016896:Treatment Outcome; D014605:Uveitis / Q000628:therapy
chemical_list: D001688:Biological Products; D047908:Intracellular Signaling Peptides and Proteins; C476731:NLRP12 protein, human; D017472:Receptors, Interleukin-1
references: J Rheumatol. 2005 Jan;32(1):175-7
references: 15630744
references: Pediatr Rheumatol Online J. 2009 Aug 05;7:14
references: 19656398
references: Pediatr Dermatol. 2013 Nov-Dec;30(6):758-60
references: 22471702
references: N Engl J Med. 2009 Jun 4;360(23):2438-44
references: 19494219
references: Joint Bone Spine. 2008 May;75(3):370-2
references: 18313967
references: Arthritis Rheum. 2007 Nov;56(11):3805-13
references: 17968944
references: N Engl J Med. 1972 Dec 21;287(25):1302
references: 4636899
references: Clin Exp Rheumatol. 2010 Mar-Apr;28(2):290-1
references: 20483057
references: J Pediatr. 2004 Dec;145(6):851-5
references: 15580218
references: Medicine (Baltimore). 2002 Sep;81(5):349-68
references: 12352631
references: J Pediatr. 1989 Nov;115(5 Pt 1):730-4
references: 2809904
references: Arthritis Rheum. 2011 May;63(5):1459-64
references: 21538323
references: Br J Haematol. 2009 Sep;146(5):467-78
references: 19466978
references: J Pediatr. 2010 Aug;157(2):310-315.e1
references: 20472245
references: Arthritis Rheum. 2008 Aug;58(8):2443-52
references: 18668535
references: Ann Rheum Dis. 2011 Sep;70(9):1692-3
references: 21378401
references: Arthritis Rheum. 2011 Dec;63(12):4018-22
references: 21792839
references: Arthritis Rheum. 2008 May;58(5):1516-20
references: 18438813
references: Neth J Med. 2008 Dec;66(11):489-91
references: 19075317
references: Arthritis Rheum. 2009 Feb;60(2):619-25
references: 19180495
references: Arthritis Rheum. 2011 Mar;63(3):830-9
references: 21360512
references: Clin Rheumatol. 2006 Feb;25(1):83-7
references: 16172948
references: J Clin Rheumatol. 2010 Aug;16(5):229-32
references: 20661070
references: Rheumatol Int. 2011 Dec;31(12):1661-4
references: 21240490
references: Arthritis Rheum. 2003 Sep;48(9):2645-51
references: 13130485
references: Rheumatology (Oxford). 2012 Oct;51(10):1855-9
references: 22740624
references: Arch Dermatol. 2006 Dec;142(12):1591-7
references: 17178985
references: Genes Immun. 2004 Jun;5(4):289-93
references: 15071491
references: Intern Med. 2007;46(15):1247-9
references: 17675778
references: Int J Immunopathol Pharmacol. 2011 Jul-Sep;24(3):695-702
references: 21978701
references: Medicine (Baltimore). 2008 Nov;87(6):301-310
references: 19011501
references: Rheumatology (Oxford). 2006 Sep;45(9):1140-3
references: 16527883
references: N Engl J Med. 2006 Aug 10;355(6):581-92
references: 16899778
references: Semin Arthritis Rheum. 2011 Oct;41(2):265-71
references: 21277619
references: Ann Intern Med. 2007 Jun 5;146(11):825-6
references: 17548423
references: JAMA Dermatol. 2013 Feb;149(2):209-15
references: 23426477
references: Rheumatol Int. 2006 Nov;27(1):97-100
references: 16871408
references: Int J Immunopathol Pharmacol. 2010 Oct-Dec;23(4):1133-41
references: 21244762
references: Clin Exp Rheumatol. 2010 Sep-Oct;28(5):802
references: 21029567
references: Lancet. 2001 Jul 7;358(9275):24-9
references: 11454373
references: J Med Genet. 2005 Jul;42(7):551-7
references: 15994876
references: Clin Exp Rheumatol. 2012 Nov-Dec;30(6):943-6
references: 22935299
references: Clin Res Cardiol. 2012 Jul;101(7):525-31
references: 22311714
references: Arthritis Rheum. 2010 Jan;62(1):250-7
references: 20039400
references: N Engl J Med. 1986 Apr 17;314(16):1001-5
references: 3515182
references: Ren Fail. 2010;32(10):1230-2
references: 20954987
references: Int J Immunopathol Pharmacol. 2009 Oct-Dec;22(4):1051-8
references: 20074469
references: Arthritis Rheum. 2008 Aug;58(8):2432-42
references: 18668591
references: Ann Rheum Dis. 2008 Sep;67(9):1292-8
references: 18180277
references: Am J Med Genet A. 2006 Apr 15;140(8):883-6
references: 16532456
references: Clin Ther. 2012 Oct;34(10):2091-103
references: 23031624
references: Arthritis Rheum. 2007 Mar;56(3):960-4
references: 17330256
references: Arthritis Rheum. 2012 Mar;64(3):908-13
references: 22006113
references: J Clin Rheumatol. 2010 Aug;16(5):244-5
references: 20661073
references: Pediatrics. 2011 Jul;128(1):e152-9
references: 21708801
references: Ophthalmology. 2003 Oct;110(10):2040-4
references: 14522785
references: Proc Natl Acad Sci U S A. 2006 Jun 27;103(26):9982-7
references: 16785446
references: J AAPOS. 2011 Apr;15(2):205-7
references: 21596301
references: Arthritis Res Ther. 2011 Feb 28;13(1):R34
references: 21356079
references: Arthritis Rheum. 2002 Aug;46(8):2181-8
references: 12209523
references: Mayo Clin Proc. 1997 Jul;72(7):611-5
references: 9212761
references: Arthritis Rheum. 2013 Feb;65(2):513-8
references: 23124805
references: Clin Rheumatol. 2003 Oct;22(4-5):314-7
references: 14579163
references: Arthritis Rheum. 2007 Sep;56(9):3138-48
references: 17763411
references: Springer Semin Immunopathol. 2006 Jun;27(4):494-508
references: 16738958
references: J Clin Rheumatol. 2004 Jun;10(3):134-7
references: 17043487
references: Ann Rheum Dis. 2011 Jun;70(6):1168-70
references: 21325428
references: Childs Nerv Syst. 2006 Apr;22(4):334-7
references: 16525848
references: Pediatr Nephrol. 2003 Sep;18(9):853-9
references: 12836090
references: Autoimmun Rev. 2009 Jan;8(3):228-32
references: 18718560
references: Int J Clin Pract. 2005 Feb;59(2):202-5
references: 15854197
references: Intern Med. 2012;51(16):2239-44
references: 22892512
references: J Exp Med. 2009 May 11;206(5):1029-36
references: 19364880
references: Am J Ophthalmol. 2002 Dec;134(6):897-904
references: 12470760
references: J Rheumatol. 1994 Sep;21(9):1708-9
references: 7799354
references: Blood. 1995 Jun 15;85(12):3586-93
references: 7780142
references: Autoimmun Rev. 2012 Nov;12(1):44-51
references: 22884558
references: Nephrol Dial Transplant. 2009 Feb;24(2):676-8
references: 19033248
references: N Engl J Med. 2009 Jun 4;360(23):2416-25
references: 19494217
references: Arthritis Res Ther. 2011;13(6):R202
references: 22152723
references: Eur J Clin Invest. 2007 Nov;37(11):912-4
references: 17973784
references: Pediatrics. 1993 May;91(5):915-21
references: 8386351
references: Clin Exp Rheumatol. 2012 May-Jun;30(3):452
references: 22513199
references: Clin Rheumatol. 2008 Oct;27(10):1317-20
references: 18506569
references: Ann Rheum Dis. 2011 Jul;70(7):1347-8
references: 21345814
references: Br J Dermatol. 2009 Nov;161(5):1199-201
references: 19673875
references: N Engl J Med. 2003 Jun 19;348(25):2583-4
references: 12815153
references: Rheumatology (Oxford). 2001 May;40(5):579-84
references: 11371670
references: Ann Intern Med. 2012 Jun 19;156(12):907-8
references: 22711098
references: Curr Drug Targets Inflamm Allergy. 2005 Feb;4(1):117-24
references: 15720245
references: Arthritis Rheum. 2009 Feb;60(2):611-8
references: 19180500
references: N Engl J Med. 2007 Jun 28;356(26):2700-3
references: 17596604
references: Rheumatology (Oxford). 2007 Jul;46(7):1211-2
references: 16935919
references: Proc Natl Acad Sci U S A. 2003 Nov 11;100(23):13501-6
references: 14595024
references: Arch Dermatol. 2012 Jun;148(6):747-52
references: 22431714
references: Case Rep Rheumatol. 2011;2011:134106
references: 22937436
references: Clin Rheumatol. 2012 Aug;31(8):1267-71
references: 22673790
references: Acta Paediatr. 2011 Jan;100(1):21-5
references: 20712835
references: Pediatr Int. 2012 Aug;54(4):552-5
references: 22830546
references: Rheumatology (Oxford). 2006 Jan;45(1):31-7
references: 16287931
references: Autoimmun Rev. 2012 Nov;12(1):14-7
references: 22878269
references: N Engl J Med. 2009 Jun 4;360(23):2426-37
references: 19494218
references: Pediatr Radiol. 2012 Apr;42(4):495-8
references: 21789664
references: Ann Med. 2012 Nov;44(7):664-73
references: 21972825
references: Clin Exp Rheumatol. 2010 May-Jun;28(3):405-7
references: 20497634
references: Ann Rheum Dis. 2014 Apr;73(4):704-9
references: 23505237
references: Ann Rheum Dis. 2013 May;72(5):678-85
references: 22753383
references: Rheumatology (Oxford). 2011 May;50(5):990-2
references: 21278066
references: Arthritis Rheum. 2012 Jun;64(6):2022-7
references: 22161697
references: Immunity. 2004 Mar;20(3):319-25
references: 15030775
references: Proc Natl Acad Sci U S A. 2008 Feb 5;105(5):1614-9
references: 18230725
references: Arch Dermatol. 2012 Mar;148(3):301-4
references: 22431772
references: Eur Rev Med Pharmacol Sci. 2010 Jan;14(1):1-18
references: 20184084
references: Pediatr Dermatol. 2005 May-Jun;22(3):262-5
references: 15916580
references: Ann Rheum Dis. 2011 Dec;70(12):2095-102
references: 21859692
references: Joint Bone Spine. 2007 Dec;74(6):544-50
references: 17950649
references: Autoimmun Rev. 2012 Mar;11(5):348-56
references: 22024500
references: Clin Exp Rheumatol. 2013 Mar-Apr;31(2):302-9
references: 23380020
references: Cell. 1999 Apr 2;97(1):133-44
references: 10199409
references: Rheumatology (Oxford). 2005 Aug;44(8):1072-3
references: 15840596
references: Autoimmun Rev. 2010 Apr;9(6):436-40
references: 19948252
references: J Clin Rheumatol. 2000 Feb;6(1):30-4
references: 19078446
references: J Pharmacol Exp Ther. 2002 May;301(2):418-26
references: 11961039
references: J Pediatr. 2000 Sep;137(3):441-2
references: 10969284
references: Rheumatology (Oxford). 2005 Mar;44(3):406-8
references: 15637033
references: Rheumatology (Oxford). 2012 Jun;51(6):1041
references: 22403182
references: Ann Intern Med. 2012 Oct 16;157(8):533-41
references: 23070486
references: Curr Opin Allergy Clin Immunol. 2002 Dec;2(6):511-6
references: 14752334
references: Rheumatology (Oxford). 2004 Nov;43(11):1405-8
references: 15316120
references: Nephrol Dial Transplant. 2012 May;27(5):1898-901
references: 21931121
references: Eur J Pediatr. 2001 Dec;160(12):705-10
references: 11795677
references: Lancet. 2004 Nov 13-19;364(9447):1779-85
references: 15541451
references: Arthritis Rheum. 1997 Oct;40(10):1879-85
references: 9336425
references: APMIS. 2006 Dec;114(12):912-9
references: 17207093
references: Clin Rev Allergy Immunol. 2013 Aug;45(1):117-30
references: 23322405
references: Arthritis Rheum. 2011 Apr;63(4):1151-5
references: 21225679
references: Clin Chem Lab Med. 2011 May;49(5):783-91
references: 21275813
references: Clin Rheumatol. 2010 Feb;29(2):209-10
references: 19774408
references: Mod Rheumatol. 2011 Dec;21(6):684-90
references: 21567247
references: Clin Exp Rheumatol. 2006 Sep-Oct;24(5 Suppl 42):S99-103
references: 17067437
references: Ann Rheum Dis. 2011 Aug;70(8):1511-2
references: 21173015
references: J Clin Rheumatol. 2007 Feb;13(1):38-40
references: 17278949
references: Int J Immunopathol Pharmacol. 2011 Oct-Dec;24(4):827-36
references: 22230390
references: Med Hypotheses. 2004;62(2):297-8
references: 14962643
references: Ann Rheum Dis. 2010 Jul;69(7):1383-8
references: 19934105
references: Clin Pharmacol Ther. 2004 May;75(5):476-83
references: 15116060
references: J Pediatr. 1985 Nov;107(5):689-93
references: 4056967
references: Ann Rheum Dis. 2011 Dec;70(12):2155-8
references: 21859689
references: Int J Immunopathol Pharmacol. 2010 Jul-Sep;23(3):701-7
references: 20943039
references: Arthritis Rheum. 2011 Dec;63(12):4007-17
references: 22127713
references: Rheumatol Int. 2012 Apr;32(4):1095-7
references: 21431291
references: Eur Rev Med Pharmacol Sci. 2006 Jul-Aug;10(4):173-8
references: 16910346
references: Rheumatol Int. 2008 Nov;29(1):107-10
references: 18500460
references: Eur J Pediatr. 2005 Jan;164(1):13-6
references: 15549379
references: Arthritis Rheum. 2011 Jul;63(7):2142-8
references: 21480187
references: Rheumatol Int. 2012 Jan;32(1):249-51
references: 20020131
references: J Rheumatol. 2011 Mar;38(3):516-8
references: 21159830
references: Autoimmun Rev. 2012 Nov;12(1):10-3
references: 22878268
references: J Inherit Metab Dis. 2006 Dec;29(6):763
references: 17103011
references: Best Pract Res Clin Rheumatol. 2012 Aug;26(4):505-33
references: 23040364
references: Rheumatol Int. 2012 Oct;32(10):3277-9
references: 20386914
references: Rheumatology (Oxford). 2007 Dec;46(12):1865-6
references: 17967816
references: Ann Rheum Dis. 2013 Mar;72(3):410-3
references: 23087183