New age of biological therapies in paediatric rheumatology

Archives of Disease in Childhood

Volumn 99, Issue 7, 2014, Pages 679-685

  Sen, Ethan S ^a   Ramanan, A V ^a  

^a BRISTOL ROYAL HOSPITAL FOR CHILDREN   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ABATACEPT; ADALIMUMAB; AZATHIOPRINE; BELIMUMAB; BIOLOGICAL PRODUCT; CANAKINUMAB; CERTOLIZUMAB PEGOL; CORTICOSTEROID; ETANERCEPT; GOLIMUMAB; HYDROXYCHLOROQUINE; INFLIXIMAB; INTERLEUKIN 1 ANTIBODY; METHYLPREDNISOLONE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PLACEBO; RECOMBINANT INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; RILONACEPT; RITUXIMAB; STEROID; TOCILIZUMAB; TOFACITINIB; TUMOR NECROSIS FACTOR INHIBITOR;

ARTHRITIS; BIOLOGICAL THERAPY; CHILDHOOD DISEASE; CORTICOSTEROID THERAPY; DRUG EFFICACY; DRUG HALF LIFE; DRUG MECHANISM; DRUG SAFETY; ENTHESITIS; ENTHESITIS RELATED ARTHRITIS; GENE EXPRESSION; HUMAN; JUVENILE; JUVENILE DERMATOMYOSITIS; JUVENILE RHEUMATOID ARTHRITIS; LUPUS ERYTHEMATOSUS NEPHRITIS; PEDIATRICS; POLYARTHRITIS; PRIORITY JOURNAL; PSORIATIC ARTHRITIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RHEUMATIC DISEASE; RHEUMATOLOGY; STEROID THERAPY; SYSTEMIC LUPUS ERYTHEMATOSUS; VISUAL ANALOG SCALE; ADOLESCENT DISEASE; DISEASE ACTIVITY;

ANTIRHEUMATIC AGENTS; BIOLOGICAL THERAPY; HUMANS; PEDIATRICS; RHEUMATIC DISEASES; RHEUMATOLOGY; TREATMENT OUTCOME;

EID: 84902377101     PISSN: 00039888     EISSN: 14682044     Source Type: Journal    
DOI: 10.1136/archdischild-2013-304396     Document Type: Review

References (63)

1. Wahren-Herlenius M, Dörner T. Immunopathogenic mechanisms of systemic autoimmune disease. Lancet 2013;382:819-31.
2. Matricon J, Barnich N, Ardid D. Immunopathogenesis of infl ammatory bowel disease. Self Nonself 2010;1:299-309.
3. Beresford MW, Baildam EM. New advances in the management of juvenile idiopathic arthritis-2: the era of biologicals. Arch Dis Child Educ Pract Ed 2009;94:151-6.
4. Lovell DJ, Giannini EH, Reiff A, et al. Etanercept in children with polyarticular juvenile rheumatoid arthritis. Pediatric Rheumatology Collaborative Study Group. N Engl J Med 2000;342:763-9. (Pubitemid 30151920)
5. Ruperto N, Lovell DJ, Cuttica R, et al. A randomized, placebo-controlled trial of infliximab plus methotrexate for the treatment of polyarticular-course juvenile rheumatoid arthritis. Arthritis Rheum 2007;56:3096-106. (Pubitemid 47502757)
6. Lovell DJ, Ruperto N, Goodman S, et al. Adalimumab with or without methotrexate in juvenile rheumatoid arthritis. N Engl J Med 2008;359:810-20.
7. A Multicenter, Double-Blind, Randomized-Withdrawal Trial of Subcutaneous Golimumab, a Humanized Anti-TNFa Antibody, in Subjects With Active Polyarticular Juvenile Idiopathic Arthritis (JIA) Despite Standard Therapy. NCT01230827. http://www.clinicaltrials.gov (accessed 17 Nov 2013).
8. A multicenter, open-label study to assess the pharmacokinetics, safety and efficacy of certolizumab pegol in children and adolescents with moderately to severely active polyarticular-course juvenile idiopathic arthritis (JIA). NCT01550003. http://www. clinicaltrials.gov (accessed 17 Nov 2013).
9. 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 2011;70:747-54.
10. Ruperto N, Brunner HI, Quartier P, et al. Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med 2012;367:2396-406.
11. 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 2008;58:2443-52.
12. Yokota S, Imagawa T, Mori M, et al. Efficacy and safety of tocilizumab in patients with systemic-onset juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled, withdrawal phase III trial. Lancet 2008;371:998-1006. (Pubitemid 351389534)
13. De Benedetti F, Brunner HI, Ruperto N, et al. Randomized trial of tocilizumab in systemic juvenile idiopathic arthritis. N Engl J Med 2012;367:2385-95.
14. Brunner HI, Ruperto N, Zuber Z, et al. Efficacy and safety of tocilizumab in patients with polyarticular juvenile idiopathic arthritis: data from a phase 3 trial. Arthritis Rheum 2012;64:S682.
15. Ruperto N, Lovell DJ, Quartier P, et al. Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trial. Lancet 2008;372:383-91.
16. Ruperto N, Lovell DJ, Quartier P, et al. Long-term safety and ef ficacy of abatacept in children with juvenile idiopathic arthritis. Arthritis Rheum 2010;62:1792-802.
17. Alexeeva EI, Valieva SI, Bzarova TM, et al. Efficacy and safety of repeat courses of rituximab treatment in patients with severe refractory juvenile idiopathic arthritis. Clin Rheumatol 2011;30:1163-72.
18. Jansson AF, Sengler C, Kuemmerle-Deschner J, et al. B cell depletion for autoimmune diseases in paediatric patients. Clin Rheumatol 2011;30:87-97.
19. A multi-center, randomized, placebo-controlled trial to evaluate the safety, efficacy, and pharmacokinetics of belimumab, a human monoclonal anti-BLyS antibody, plus standard therapy in pediatric patients with systemic lupus erythematosus. NCT01649765. http://www.clinicaltrials.gov (accessed 17 Nov 2013).
20. An open-label multiple dose study to evaluate the pharmacokinetics, safety and tolerability of CP-690,550 in pediatric patients from 2 to less than 18 years of age with juvenile idiopathic arthritis (JIA). NCT01513902. http://www.clinicaltrials.gov (accessed 17 Nov 2013).
21. A long-term, open-label follow-up study of CP-690,550 for treatment of juvenile idiopathic arthritis (JIA). NCT01500551. http://www.clinicaltrials.gov (accessed 17 Nov 2013).
22. Woodrick RS, Ruderman EM. Safety of biologic therapy in rheumatoid arthritis. Nat Rev Rheumatol 2011;7:639-52.
23. Vitale A, Rigante D, Lucherini OM, et al. Biological treatments: new weapons in the management of monogenic autoinflammatory disorders. Mediators Inflamm 2013;2013:939847.
24. Prakken B, Albani S, Martini A. Juvenile idiopathic arthritis. Lancet 2011;377:2138-49.
25. Petty RE, Southwood TR, Manners P, et al. International League of Associations for Rheumatology classi fication of juvenile idiopathic arthritis: second revision, Edmonton, 2001. J Rheumatol 2004;31:390-2. (Pubitemid 38160763)
26. Harris JG, Kessler EA, Verbsky JW. Update on the treatment of juvenile idiopathic arthritis. Curr Allergy Asthma Rep 2013;13:337-46.
27. Wahezi DM, Ilowite NT. Juvenile idiopathic arthritis: an update on current pharmacotherapy and future perspectives. Expert Opin Pharmacother 2013;14:975-89.
28. Horneff G. Update on biologicals for treatment of juvenile idiopathic arthritis. Expert Opin Biol Ther 2013;13:361-76.
29. Breda L, Del Torto M, De Sanctis S, et al. Biologics in children's autoimmune disorders: efficacy and safety. Eur J Pediatr 2011;170:157-67.
30. Ramanan AV, Grom AA. Does systemic-onset juvenile idiopathic arthritis belong under juvenile idiopathic arthritis? Rheumatology (Oxford) 2005;44:1350-3. (Pubitemid 41631856)
31. Sikora KA, Grom AA. Update on the pathogenesis and treatment of systemic idiopathic arthritis. Curr Opin Pediatr 2011;23:640-6.
32. Pascual V, Allantaz F, Arce E, et al. Role of interleukin-1 (IL-1) in the pathogenesis of systemic onset juvenile idiopathic arthritis and clinical response to IL-1 blockade. J Exp Med 2005;201:1479-86. (Pubitemid 40664091)
33. Ogilvie EM, Khan A, Hubank M, et al. Specific gene expression profiles in systemic juvenile idiopathic arthritis. Arthritis Rheum 2007;56:1954-65. (Pubitemid 47110394)
34. Southwood TR, Foster HE, Davidson JE, et al. Duration of etanercept treatment and reasons for discontinuation in a cohort of juvenile idiopathic arthritis patients. Rheumatology (Oxford) 2011;50:189-95.
35. Nigrovic PA, Mannion M, Prince FH, et al. Anakinra as first-line disease-modifying therapy in systemic juvenile idiopathic arthritis: report of forty-six patients from an international multicenter series. Arthritis Rheum 2011;63:545-55.
36. Hedrich CM, Bruck N, Fiebig B, et al. Anakinra: a safe and effective first-line treatment in systemic onset juvenile idiopathic arthritis (SoJIA). Rheumatol Int 2012;32:3525-30.
37. Yokota S, Imagawa T, Mori M, et al. Long-term treatment of systemic juvenile idiopathic arthritis with tocilizumab: results of an open-label extension study in Japan. Ann Rheum Dis 2013;72:627-8.
38. Giannini EH, Ruperto N, Ravelli A, et al. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum 1997;40:1202-9. (Pubitemid 27430130)
39. Riley P, McCann LJ, Maillard SM, et al. Effectiveness of infliximab in the treatment of refractory juvenile dermatomyositis with calcinosis. Rheumatology (Oxford) 2008;47:877-80. (Pubitemid 351796377)
40. Baechler EC, Bilgic H, Reed AM. Type I interferon pathway in adult and juvenile dermatomyositis. Arthritis Res Ther 2011;13:249.
41. Palucka AK, Blanck JP, Bennett L, et al. Cross-regulation of TNF and IFN-alpha in autoimmune diseases. Proc Natl Acad Sci U S A 2005;102:3372-7. (Pubitemid 40328052)
42. Rouster-Stevens KA, Ferguson L, Morgan G, et al. Pilot study of etanercept in patients with refractory juvenile dermatomyositis. Arthritis Care Res (Hoboken) 2013. doi:10.1002/acr.22198. [Epub ahead of print]
43. Oddis CV, Reed AM, Aggarwal R, et al. Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: a randomized, placebo-phase trial. Arthritis Rheum 2013;65:314-24.
44. Barsalou J, Levy DM, Silverman ED. An update on childhood-onset systemic lupus erythematosus. Curr Opin Rheumatol 2013;25:616-22.
45. Podolskaya A, Stadermann M, Pilkington C, et al. B cell depletion therapy for 19 patients with refractory systemic lupus erythematosus. Arch Dis Child 2008;93:401-6. (Pubitemid 352025791)
46. Marks SD, Tullus K. Successful outcomes with rituximab therapy for refractory childhood systemic lupus erythematosus. Pediatr Nephrol 2006;21:598-9.
47. Rovin BH, Furie R, Latinis K, et al. Efficacy and safety of rituximab in patients with active proliferative lupus nephritis: the Lupus Nephritis Assessment with Rituximab study. Arthritis Rheum 2012;64:1215-26.
48. Merrill JT, Neuwelt CM, Wallace DJ, et al. Efficacy and safety of rituximab in moderately-to-severely active systemic lupus erythematosus: the randomized, double-blind, phase II/III systemic lupus erythematosus evaluation of rituximab trial. Arthritis Rheum 2010;62:222-33.
49. Reddy V, Jayne D, Close D, et al. B-cell depletion in SLE: clinical and trial experience with rituximab and ocrelizumab and implications for study design. Arthritis Res Ther 2013;15(Suppl 1):S2.
50. Merrill JT, Burgos-Vargas R, Westhovens R, et al. The efficacy and safety of abatacept in patients with non-life-threatening manifestations of systemic lupus erythematosus: results of a twelve-month, multicenter, exploratory, phase IIb, randomized, double-blind, placebo-controlled trial. Arthritis Rheum 2010;62:3077-87.
51. van Vollenhoven RF, Parodis I, Levitsky A. Biologics in SLE: Towards new approaches. Best Pract Res Clin Rheumatol 2013;27:341-9.
52. A phase Ib, open label, multicentre study to investigate the pharmacokinetics, pharmacodynamics, and safety of tocilizumab following subcutaneous administration in patients with polyarticular-course juvenile idiopathic arthritis. NCT01904279. http://www.clinicaltrials.gov (accessed 17 Nov 2013).
53. A phase Ib, open label, multicentre study to investigate the pharmacokinetics, pharmacodynamics, and safety of tocilizumab following subcutaneous administration in patients with systemic juvenile idiopathic arthritis. NCT01904292. http://www. clinicaltrials.gov (accessed 17 Nov 2013).
54. A Phase 3 multi-center, open-label study to evaluate pharmacokinetics, efficacy and safety of abatacept administered subcutaneously (SC) in children and adolescents with active polyarticular juvenile idiopathic arthritis (pJIA) and inadequate response (IR) to biologic or non biologic disease modifying anti-rheumatic drugs (DMARDs). NCT01844518. http://www.clinicaltrials.gov (accessed 17 Nov 2013).
55. Gurion R, Singer NG. Tocilizumab in pediatric rheumatology: the clinical experience. Curr Rheumatol Rep 2013;15:338.
56. van Vollenhoven RF. Small molecular compounds in development for rheumatoid arthritis. Curr Opin Rheumatol 2013;25:391-7.
57. European Medicines Agency. Refusal of the marketing authorisation for Xeljanz (tofacitinib). EMA/460814/2013, 26 July 2013. http://www.ema.europa.eu/ docs/en-GB/document-library/Summary-of-opinion---Initial-authorisation/human/ 002542/ WC500146629.pdf (accessed 17 Nov 2013).
58. Heiligenhaus A, Heinz C, Edelsten C, et al. Review for disease of the year: epidemiology of juvenile idiopathic arthritis and its associated uveitis: the probable risk factors. Ocul Immunol Inflamm 2013;21:180-91.
59. Sen ES, Sharma S, Hinchcliffe A, et al. Use of adalimumab in refractory non-infectious childhood chronic uveitis: efficacy in ocular disease - a case cohort interventional study. Rheumatology (Oxford) 2012;51:2199-203.
60. Sharma SM, Ramanan AV, Riley P, et al. Use of infliximab in juvenile onset rheumatological disease-associated refractory uveitis: ef ficacy in joint and ocular disease. Ann Rheum Dis 2007;66:840-1. (Pubitemid 46846714)
61. Zulian F, Balzarin M, Falcini F, et al. Abatacept for severe anti-tumor necrosis factor alpha refractory juvenile idiopathic arthritis-related uveitis. Arthritis Care Res 2010;62:821-5.
62. Randomised controlled trial of the clinical effectiveness, SafetY and Cost effectiveness of Adalimumab in combination with MethOtRExate for the treatment of juvenile idiopathic arthritis associated uveitis (SYCAMORE Trial). ISRCTN10065623. http://www.clinicaltrials.gov (accessed 17 Nov 2013).
63. A phase 2, multicenter, open-label, dose-escalation study to evaluate the safety and tolerability of intravenous dose of MEDI-546, a human monoclonal antibody directed against type I interferon receptor (IFNAR), in Japanese subjects with active systemic lupus erythematosus (SLE). NCT01559090. http://www.clinicaltrials.gov (accessed 17 Nov 2013).