The development and assessment of biological treatments for children

British Journal of Clinical Pharmacology

Volumn 79, Issue 3, 2015, Pages 379-394

  Smith, Eve M D ^a   Foster, Helen E ^b,c   Beresford, Michael W ^a,d  

^a UNIVERSITY OF LIVERPOOL   (United Kingdom)

^b NEWCASTLE UNIVERSITY   (United Kingdom)

^c Infectious Diseases Allergy   (United Kingdom)

^d ALDER HEY CHILDREN'S NHS FOUNDATION TRUST   (United Kingdom)

Author keywords

adverse drug reactions; biologics; clinical trials; paediatric rheumatology

Indexed keywords

ABATACEPT; ADALIMUMAB; ANTIRHEUMATIC AGENT; BELIMUMAB; CANAKINUMAB; CERTOLIZUMAB PEGOL; CYTOTOXIC T LYMPHOCYTE ANTIGEN 4; ETANERCEPT; GOLIMUMAB; INFLIXIMAB; INTERLEUKIN 1; INTERLEUKIN 6; RECOMBINANT INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; RITUXIMAB; TOCILIZUMAB; BIOLOGICAL PRODUCT;

ARTICLE; CHILD; DISEASE EXACERBATION; DRUG EFFICACY; DRUG INDUSTRY; DRUG MONITORING; DRUG SAFETY; DRUG SURVEILLANCE PROGRAM; HUMAN; JUVENILE RHEUMATOID ARTHRITIS; MULTICENTER STUDY (TOPIC); PEDIATRICS; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REGISTER; RHEUMATOLOGY; STUDY DESIGN; AUTOIMMUNE DISEASES; DRUG DEVELOPMENT; IMMUNOLOGY; METHODOLOGY; PROCEDURES; TREATMENT OUTCOME;

AUTOIMMUNE DISEASES; BIOLOGICAL PRODUCTS; CHILD; DRUG DISCOVERY; HUMANS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; TREATMENT OUTCOME;

EID: 84929999872     PISSN: 03065251     EISSN: 13652125     Source Type: Journal    
DOI: 10.1111/bcp.12406     Document Type: Article

References (127)

1. Horneff G,. Update on biologicals for treatment of juvenile idiopathic arthritis. Expert Opin Biol Ther 2013; 13: 361-376.
2. Munro JE, Murray KJ,. Advances in paediatric rheumatology: beyond NSAIDs and joint replacement. J Paed Child Health 2004; 40: 161-169.
3. Brennan FM, Feldmann R,. Cytokines in autoimmunity. Curr Opin Autoimmune 1992; 4: 1520-1530.
4. Taylor PC,. Pharmacology of TNF blocade in rheumatoid arthrtis and other chronic inflammatory disease. Curr Opin Pharmacol 2010; 10: 308-315.
5. Kok MR, Tak PP,. Taking advances from bench to bedside during the last decade. Best Pract Res Clin Rheumatol 2012; 26: 225-236.
6. Mangge H, Kenzian H, Gallistl S, Neuwirth G, Liebmann P, Kaulfersch W, Beaufort F, Muntean W, Schauenstein K,. Serum cytokines in juvenile rheumatoid arthritis. Correlation with conventional inflammation parameters and clinical subtypes. Arthritis Rheum 1995; 38: 211-220.
7. Eberhard BA, Laxer RM, Andersson U, Silverman ED,. Local synthesis of both macrophage and T cell cytokines by synovial fluid cells from children with juvenile rheumatoid arthritis. Clin Exp Immunol 1994; 96: 260-266.
8. Giannini EH, Ruperto N, Ravelli A, Lovell DJ, Felson DT, Martini A,. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum 1997; 40: 1202-1209.
9. Wallace CA, Ruperto N, Giannini E,. Preliminary criteria for clinical remission for select categories of juvenile idiopathic arthritis. J Rheumatol 2004; 31: 2290-2294.
10. Brunner HI, Lovell DJ, Finck BK, Giannini EH,. Preliminary definition of disease flare in juvenile rheumatoid arthritis. J Rheumatol 2002; 29: 1058-1064.
11. Lehman TJ,. Are withdrawal trials in paediatric rheumatic disease helpful? Lancet 2008; 372: 348-350.
12. Irvine EJ, Whitehead WE, Chey WD, Matsueda K, Shaw M, Talley NJ, Veldhuyzen van Zanten SJ,. Design of treatment trials for functional gastrointestinal disorders. Gastroenterology 2006; 130: 1538-1551.
13. Fernandes R, Ferreira JJ, Sampaio C,. The placebo response in studies of acute migraine. J Pediatr 2008; 152: 527-533.
14. Rothner AD, Wasiewski W, Winner P, Lewis D, Stankowski J,. Zolmitriptan oral tablet in migraine treatment: high placebo responses in adolescents. Headache 2006; 46: 101-109.
15. Mohler KM, Torrance DS, Smith CA, Goodwin RG, Stremler KE, Fung VP, Madani H, Widmer MB,. Soluble tumor necrosis factor (TNF) receptors are effective therapeutic agents in lethal endotoxemia and function simultaneously as both TNF carriers and TNF antagonists. J Immunol 1993; 151: 1548-1561.
16. Lovell DJ, Giannini EH, Reiff A, Cawkwell GD, Silverman ED, Nocton JJ, Stein LD, Gedalia A, Ilowite NT, Wallace CA, Whitmore J, Finck BK,. Etanercept in children with polyarticular juvenile rheumatoid arthritis. Pediatric Rheumatology Collaborative Study Group. N Engl J Med 2000; 342: 763-769.
17. Lovell DJ, Giannini EH, Reiff A, Jones OY, Schneider R, Olson JC, Stein LD, Gedalia A, Ilowite NT, Wallace CA, Lange M, Finck BK, Burge DJ,. Long-term efficacy and safety of etanercept in children with polyarticular-course juvenile rheumatoid arthritis: interim results from an ongoing multicenter, open-label, extended-treatment trial. Arthritis Rheum 2003; 48: 218-226.
18. Quartier P, Taupin P, Bourdeaut F, Lemelle I, Pillet P, Bost M, Sibilia J, Koné-Paut I, Gandon-Laloum S, LeBideau M, Bader-Meunier B, Mouy R, Debré M, Landais P, Prieur AM,. Efficacy of etanercept for the treatment of juvenile idiopathic arthritis according to the onset type. Arthritis Rheum 2003; 48: 1093-1101.
19. Prince FH, Twilt M, ten Cate R, van Rossum MA, Armbrust W, Hoppenreijs EP, van Santen-Hoeufft M, Koopman-Keemink Y, Wulffraat NM, van Suijlekom-Smit LW,. Long-term follow-up on effectiveness and safety of etanercept in juvenile idiopathic arthritis: the Dutch national register. Ann Rheum Dis 2009; 68: 635-641.
20. Horneff G, Burgos-Vargas R, Constantin T, Foeldvari I, Vojinovic J, Chasnyk VG, Dehoorne J, Panaviene V, Susic G, Stanevica V, Kobusinska K, Zuber Z, Mouy R, Rumba-Rozenfelde I, Breda L, Dolezalova P, Job-Deslandre C, Wulffraat N, Alvarez D, Zang C, Wajdula J, Woodworth D, Vlahos B, Martini A, Ruperto N,. Efficacy and safety of open-label etanercept on extended oligoarticular juvenile idiopathic arthritis, enthesitis-related arthritis and psoriatic arthritis: part 1 (week 12) of the CLIPPER study. Ann Rheum Dis 2014; 73: 1114-1122.
21. Papsdorf V, Horneff G,. Complete control of disease activity and remission induced by treatment with etanercept in juvenile idiopathic arthritis. Rheumatol (Oxford) 2011; 50: 214-221.
22. Otten MH, Prince FH, Armbrust W, ten Cate R, Hoppenreijs EP, Twilt M, Koopman-Keemink Y, Gorter SL, Dolman KM, Swart JF, van den Berg JM, Wulffraat NM, van Rossum MA, van Suijlekom-Smit LW,. Factors associated with treatment response to etanercept in juvenile idiopathic arthritis. JAMA 2011; 306: 2340-2347.
23. European Medicines Agency. Opinion and decisions on paediatric investigation plans. 2014. Available at http://www.ema.europa.eu (last accessed 26 March 2014).
24. National Institute for Health and Care Excellence. NICE Guidance. 2014. Available at http://www.nice.org.uk (last accessed 26 March 2014).
25. Lovell DJ, Reiff A, Jones OY, Schneider R, Nocton J, Stein LD, Gedalia A, Ilowite NT, Wallace CA, Whitmore JB, White B, Giannini EH,. Long-term safety and efficacy of etanercept in children with polyarticular-course juvenile rheumatoid arthritis. Arthritis Rheum 2006; 54: 1987-1994.
26. Lovell DJ, Reiff A, Ilowite NT, Wallace CA, Chon Y, Lin SL, Baumgartner SW, Giannini EH,. Safety and efficacy of up to eight years of continuous etanercept therapy in patients with juvenile rheumatoid arthritis. Arthritis Rheum 2008; 58: 1496-1504.
27. Horneff G, De Bock F, Foeldvari I, Girschick HJ, Michels H, Moebius D, Schmeling H,. Safety and efficacy of combination of etanercept and methotrexate compared to treatment with etanercept only in patients with juvenile idiopathic arthritis (JIA): preliminary data from the German JIA Registry. Ann Rheum Dis 2009; 68: 519-525.
28. Giannini EH, Ilowite NT, Lovell DJ, Wallace CA, Rabinovich CE, Reiff A, Higgins G, Gottlieb B, Singer NG, Chon Y, Lin SL, Baumgartner SW,. Long-term safety and effectiveness of etanercept in children with selected categories of juvenile idiopathic arthritis. Arthritis Rheum 2009; 60: 2794-2804.
29. Gerloni V, Pontikaki I, Gattinara M, Fantini F,. Focus on adverse events of tumour necrosis factor alpha blockade in juvenile idiopathic arthritis in an open monocentric long-term prospective study of 163 patients. Ann Rheum Dis 2008; 67: 1145-1152.
30. Horneff G, Ebert A, Fitter S, Minden K, Foeldvari I, Kummerle-Deschner J, Thon A, Girschick HJ, Weller F, Huppertz HI,. Safety and efficacy of once weekly etanercept 0.8 mg/kg in a multicentre 12 week trial in active polyarticular course juvenile idiopathic arthritis. Rheumatol (Oxford) 2009; 48: 916-919.
31. Kuemmerle-Deschner JB, Horneff G,. Safety and efficacy of once-weekly application of Etanercept in children with juvenile idiopathic arthritis. Rheumatol Int 2007; 28: 153-156.
32. Prince FH, Twilt M, Jansen-Wijngaarden NC, van Suijlekom-Smit LW,. Effectiveness of a once weekly double dose of etanercept in patients with juvenile idiopathic arthritis: a clinical study. Ann Rheum Dis 2007; 66: 704-705.
33. Toussi SS, Pan N, Walters HM, Walsh TJ,. Infections in children and adolescents with juvenile idiopathic arthritis and inflammatory bowel disease treated with tumor necrosis factor-α inhibitors: a systematic review of the literature. Clin Infect Dis 2013; 57: 1318-1330.
34. Myers A, Clark J, Foster H,. Tuberculosis and treatment with infliximab. N Engl J Med 2002; 346: 623-626.
35. Southwood TR,. Adverse events in juvenile idiopathic arthritis patients treated with etanercept or the combination of etanercept and methotrexate. Arthritis Rheum 2005; 55 (Suppl.): S85.
36. Kaneko U, Kishi T, Kikuchi M, Hara R, Shinoki T, Miyamae T, Imagawa T, Mori M, Yokota S,. Two patients with childhood-onset Behcet's disease successfully treated by anti-tumor necrosis factor therapy. Jpn J Clin Immunol 2010; 33: 157-161.
37. Sakallioglu O, Duzova A, Ozen S,. Etanercept in the treatment of arthritis in a patient with familial Mediterranean fever. Clin Exp Rheumatol 2006; 24: 435-437.
38. Kusuhara K, Hoshina T, Saito M, Ishimura M, Inoue H, Horiuchi T, Sato T, Hara T,. Successful treatment of a patient with tumor necrosis factor receptor-associated periodic syndrome using a half-dose of etanercept. Pediatr Int 2012; 54: 552-555.
39. Choueiter NF, Olson AK, Shen DD, Portman MA,. Prospective open-label trial of etanercept as adjunctive therapy for kawasaki disease. J Pediatr 2010; 157: 960-966.
40. Lin JH, Liebhaber M, Roberts RL, Dyer Z, Stiehm ER,. Etanercept treatment of cutaneous granulomas in common variable immunodeficiency. J Aller Clin Immunol 2006; 117: 878-882.
41. Akobeng AK, Zachos M,. Tumor necrosis factor-alpha antibody for induction of remission in Crohn's disease. Cochrane Database Syst Rev 2004 (1): CD003574. (Online).
42. Paller AS, Siegfried EC, Langley RG, Gottlieb AB, Pariser D, Landells I, Hebert AA, Eichenfield LF, Patel V, Creamer K, Jahreis A,. Etanercept treatment for children and adolescents with plaque psoriasis. N Engl J Med 2008; 358: 241-251.
43. Lofgren S, Krol A,. New therapies in pediatric dermatology. Curr Opin Pediat 2011; 23: 399-402.
44. Lovell DJ, Ruperto N, Goodman S, Reiff A, Jung L, Jarosova K, Nemcova D, Mouy R, Sandborg C, Bohnsack J, Elewaut D, Foeldvari I, Gerloni V, Rovensky J, Minden K, Vehe RK, Weiner LW, Horneff G, Huppertz HI, Olson NY, Medich JR, Carcereri-De-Prati R, McIlraith MJ, Giannini EH, Martini A,. Adalimumab with or without methotrexate in juvenile rheumatoid arthritis. N Engl J Med 2008; 359: 810-820.
45. Kingsbury D, Quartier P, Santra S,. Safety and efficacy of adalimumab in children with active polyarticular juvenile idiopathic arthritis aged 2 to < 4 years or > 4 years weighing < 15 kg. Ann Rheum Dis 2012; 71: (Suppl 3): 428.
46. FDA Pediatric Advisory Committee. Pediatric focused safety review: adalimumab (humira). 2009. Available at http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM193197.pdf (last accessed 13 June 2013).
47. Anink J, Otten MH, Gorter SL, Prince FH, van Rossum MA, van den Berg JM, van Pelt PA, Kamphuis S, Brinkman DM, Swen WA, Swart JF, Wulffraat NM, Dolman KM, Koopman-Keemink Y, Hoppenreijs EP, Armbrust W, Ten Cate R, van Suijlekom-Smit LW,. Treatment choices of paediatric rheumatologists for juvenile idiopathic arthritis: etanercept or adalimumab? Rheumatol (Oxford) 2013; 52: 1674-1679.
48. Biester S, Deuter C, Michels H, Haefner R, Kuemmerle-Deschner J, Doycheva D, Zierhut M,. Adalimumab in the therapy of uveitis in childhood. Br J Ophthalmol 2007; 91: 319-324.
49. Tynjala P, Kotaniemi K, Lindahl P, Latva K, Aalto K, Honkanen V, Lahdenne P,. Adalimumab in juvenile idiopathic arthritis-associated chronic anterior uveitis. Rheumatol (Oxford) 2008; 47: 339-344.
50. Simonini G, Taddio A, Cattalini M, Caputo R, De Libero C, Naviglio S, Bresci C, Lorusso M, Lepore L, Cimaz R,. Prevention of flare recurrences in childhood-refractory chronic uveitis: an open-label comparative study of adalimumab versus infliximab. Arthritis Care Res 2011; 63: 612-618.
51. Randomised controlled trial of the clinical effectiveness, safety and cost effectiveness of Adalimumab for the treatment of juvenile idiopathic arthritis associated uveitis (SYCAMORE). 2013. Available at http://www.sycamoretrial.org.uk (last accessed 13 June 2013).
52. Sandborn WJ, Hanauer SB, Rutgeerts P, Fedorak RN, Lukas M, MacIntosh DG, Panaccione R, Wolf D, Kent JD, Bittle B, Li J, Pollack PF,. Adalimumab for maintenance treatment of Crohn's disease: results of the CLASSIC II trial. Gut 2007; 56: 1232-1239.
53. Hanauer SB, Sandborn WJ, Rutgeerts P, Fedorak RN, Lukas M, MacIntosh D, Panaccione R, Wolf D, Pollack P,. Human anti-tumor necrosis factor monoclonal antibody (adalimumab) in Crohn's disease: the CLASSIC-I trial. Gastroenterology 2006; 130: 323-333.
54. Feagan BG, Panaccione R, Sandborn WJ, D'Haens GR, Schreiber S, Rutgeerts PJ, Loftus EV, Jr, Lomax KG, Yu AP, Wu EQ, Chao J, Mulani P,. Effects of adalimumab therapy on incidence of hospitalization and surgery in Crohn's disease: results from the CHARM study. Gastroenterology 2008; 135: 1493-1499.
55. Colombel JF, Sandborn WJ, Rutgeerts P, Enns R, Hanauer SB, Panaccione R, Schreiber S, Byczkowski D, Li J, Kent JD, Pollack PF,. Adalimumab for maintenance of clinical response and remission in patients with Crohn's disease: the CHARM trial. Gastroenterology 2007; 132: 52-65.
56. Rosenbach Y, Hartman C, Shapiro R, Hirsch A, Avitzur Y, Shamir R,. Adalimumab treatment in children with refractory Crohn's disease. Dig Dis Sci 2010; 55: 747-753.
57. Wyneski MJ, Green A, Kay M, Wyllie R, Mahajan L,. Safety and efficacy of adalimumab in pediatric patients with Crohn disease. J Pediatr Gastroenterol Nutr 2008; 47: 19-25.
58. Viola F, Civitelli F, Di Nardo G, Barbato MB, Borrelli O, Oliva S, Conte F, Cucchiara S,. Efficacy of adalimumab in moderate-to-severe pediatric Crohn's disease. Am J Gastroenterol 2009; 104: 2566-2571.
59. Russell RK, Wilson ML, Loganathan S, Bourke B, Kiparissi F, Mahdi G, Torrente F, Rodrigues A, Davies I, Thomas A, Akobeng AK, Fagbemi A, Hyer W, Spray C, Vaish S, Rogers P, McGrogan P, Heuschkel RB, Ayub N, Fell JM, Afzal NA, Green M, Murphy MS, Rao P, Shah N, Ho GT, Naik S, Wilson DC,. A British Society of Paediatric Gastroenterology, Hepatology and Nutrition survey of the effectiveness and safety of adalimumab in children with inflammatory bowel disease. Aliment Pharmacol Ther 2011; 33: 946-953.
60. Rosh JR, Lerer T, Markowitz J, Goli SR, Mamula P, Noe JD, Pfefferkorn MD, Kelleher KT, Griffiths AM, Kugathasan S, Keljo D, Oliva-Hemker M, Crandall W, Carvalho RS, Mack DR, Hyams JS,. Retrospective Evaluation of the Safety and Effect of Adalimumab Therapy (RESEAT) in pediatric Crohn's disease. Am J Gastroenterol 2009; 104: 3042-3049.
61. Hyams JS, Griffiths A, Markowitz J, Baldassano RN, Faubion WA, Jr, Colletti RB, Dubinsky M, Kierkus J, Rosh J, Wang Y, Huang B, Bittle B, Marshall M, Lazar A,. Safety and efficacy of adalimumab for moderate to severe Crohn's disease in children. Gastroenterology 2012; 143: 365-374.
62. Callen JP, Jackson JH,. Adalimumab effectively controlled recalcitrant generalized pustular psoriasis in an adolescent. J Dermatol Treat 2005; 16: 350-352.
63. Alvarez AC, Rodriguez-Nevado I, De Argila D, Rubio FP, Rovira I, Torrelo A, Zambrano A,. Recalcitrant pustular psoriasis successfully treated with adalimumab. Pediatr Dermatol 2011; 28: 195-197.
64. Luu M, Cordoro KM,. The evolving role of biologics in the treatment of pediatric psoriasis. Skin Ther Lett 2013; 18: 1-4.
65. A multicenter, randomized, double-dummy, double-blind study evaluating two doses of adalimumab versus methotrexate (MTX) in pediatric subjects with chronic plaque psoriasis.: ClinicalTrial.gov. 2013. Available at http://www.clinicaltrials.gov/ct2/show/NCT01251614 (last accessed 13 June 2013).
66. Horneff G, Fitter S, Foeldvari I, Minden K, Kuemmerle-Deschner J, Tzaribacev N, Thon A, Borte M, Ganser G, Trauzeddel R, Huppertz HI,. Double-blind, placebo-controlled randomized trial with adalimumab for treatment of juvenile onset ankylosing spondylitis (JoAS): significant short term improvement. Arthritis Res Ther 2012; 14: R230.
67. Eleftheriou D, Melo M, Marks SD, Tullus K, Sills J, Cleary G, Dolezalova P, Ozen S, Pilkington C, Woo P, Klein N, Dillon MJ, Brogan PA,. Biologic therapy in primary systemic vasculitis of the young. Rheumatol (Oxford) 2009; 48: 978-986.
68. De Cassan C, De Vroey B, Dussault C, Hachulla E, Buche S, Colombel JF,. Successful treatment with adalimumab in a familial case of gastrointestinal Behcet's disease. J Crohn's Colitis 2011; 5: 364-368.
69. Robinson AB, Gallentine WB, Rabinovich CE,. Pediatric neuro-Behcet's disease responsive to adalimumab. Pediatr Neurol 2010; 43: 291-293.
70. Ruperto N, Lovell DJ, Cuttica R, Wilkinson N, Woo P, Espada G, Wouters C, Silverman ED, Balogh Z, Henrickson M, Apaz MT, Baildam E, Fasth A, Gerloni V, Lahdenne P, Prieur AM, Ravelli A, Saurenmann RK, Gamir ML, Wulffraat N, Marodi L, Petty RE, Joos R, Zulian F, McCurdy D, Myones BL, Nagy K, Reuman P, Szer I, Travers S, Beutler A, Keenan G, Clark J, Visvanathan S, Fasanmade A, Raychaudhuri A, Mendelsohn A, Martini A, Giannini EH, Paediatric Rheumatology International Trials Organisation, Pediatric Rheumatology Collaborative Study Group. A randomized, placebo-controlled trial of infliximab plus methotrexate for the treatment of polyarticular-course juvenile rheumatoid arthritis. Arthritis Rheum 2007; 56: 3096-3106.
71. Kahn P, Weiss M, Imundo LF, Levy DM,. Favorable response to high-dose infliximab for refractory childhood uveitis. Ophthalmol 2006; 113: 860-864.
72. Uperto N, Lovell DJ, Cuttica R, Woo P, Meiorin S, Wouters C, Silverman ED, Balogh Z, Henrickson M, Davidson J, Foeldvari I, Imundo L, Simonini G, Oppermann J, Xu S, Shen YK, Visvanathan S, Fasanmade A, Mendelsohn A, Martini A, Giannini EH,. Long-term efficacy and safety of infliximab plus methotrexate for the treatment of polyarticular-course juvenile rheumatoid arthritis: findings from an open-label treatment extension. Ann Rheum Dis 2010; 69: 718-722.
73. Baldassano R, Braegger CP, Escher JC, DeWoody K, Hendricks DF, Keenan GF, Winter HS,. Infliximab (REMICADE) therapy in the treatment of pediatric Crohn's disease. Am J Gastroenterol 2003; 98: 833-838.
74. Cezard JP, Nouaili N, Talbotec C, Hugot JP, Gobert JG, Schmitz J, Mougenot JF, Alberti C, Goulet O,. A prospective study of the efficacy and tolerance of a chimeric antibody to tumor necrosis factors (remicade) in severe pediatric crohn disease. J Pediatr Gastroenterol Nutr 2003; 36: 632-636.
75. Hyams J, Crandall W, Kugathasan S, Griffiths A, Olson A, Johanns J, Liu G, Travers S, Heuschkel R, Markowitz J, Cohen S, Winter H, Veereman-Wauters G, Ferry G, Baldassano R, Group RS,. Induction and maintenance infliximab therapy for the treatment of moderate-to-severe Crohn's disease in children. Gastroenterology 2007; 132: 863-873.
76. Hyams JS, Lerer T, Griffiths A, Pfefferkorn M, Kugathasan S, Evans J, Otley A, Carvalho R, Mack D, Bousvaros A, Rosh J, Mamula P, Kay M, Crandall W, Oliva-Hemker M, Keljo D, LeLeiko N, Markowitz J, Pediatric Inflammatory Bowel Disease Collaborative Research Group. Long-term outcome of maintenance infliximab therapy in children with Crohn's disease. Inflamm Bowel Dis 2009; 15: 816-822.
77. Ruemmele FM, Lachaux A, Cezard JP, Morali A, Maurage C, Ginies JL, Viola S, Goulet O, Lamireau T, Scaillon M, Breton A, Sarles J,. Efficacy of infliximab in pediatric Crohn's disease: a randomized multicenter open-label trial comparing scheduled to on demand maintenance therapy. Inflamm Bowel Dis 2009; 15: 388-394.
78. Vermeire S, Noman M, Van Assche G, Baert F, D'Haens G, Rutgeerts P,. Effectiveness of concomitant immunosuppressive therapy in suppressing the formation of antibodies to infliximab in Crohn's disease. Gut 2007; 56: 1226-1231.
79. Crandall W, Hyams J, Kugathasan S, Griffiths A, Zrubek J, Olson A, Liu G, Heuschkel R, Markowitz J, Cohen S, Winter H, Veereman-Wauters G, Ferry G, Baldassano RN,. Infliximab therapy in children with concurrent perianal Crohn disease: observations from REACH. J Pediatr Gastroenterol Nutr 2009; 49: 183-190.
80. Gasparetto M, Corradin S, Vallortigara F, Cananzi M, Guariso G,. Infliximab and pediatric stricturing Crohn's disease: a possible alternative to surgery? Experience of seven cases. Acta Gastroenterol Belg 2012; 75: 58-60.
81. Walters TD, Gilman AR, Griffiths AM,. Linear growth improves during infliximab therapy in children with chronically active severe Crohn's disease. Inflamm Bowel Dis 2007; 13: 424-430.
82. Braun J, Deodhar A, Inman RD, van der Heijde D, Mack M, Xu S, Hsu B,. Golimumab administered subcutaneously every 4 weeks in ankylosing spondylitis: 104-week results of the GO-RAISE study. Ann Rheum Dis 2012; 71: 661-667.
83. Choy EH, Hazleman B, Smith M, Moss K, Lisi L, Scott DG, Patel J, Sopwith M, Isenberg DA,. Efficacy of a novel PEGylated humanized anti-TNF fragment (CDP870) in patients with rheumatoid arthritis: a phase II double-blinded, randomized, dose-escalating trial. Rheumatol (Oxford) 2002; 41: 1133-1137.
84. Kavanaugh A, McInnes IB, Mease PJ, Krueger GG, Gladman DD, van der Heijde D, Mudivarthy S, Xu W, Mack M, Xu Z, Beutler A,. Clinical efficacy, radiographic and safety findings through 2 years of golimumab treatment in patients with active psoriatic arthritis: results from a long-term extension of the randomised, placebo-controlled GO-REVEAL study. Ann Rheum Dis 2013; 72: 1777-1785.
85. Andborn WJ, Feagan BG, Marano C, Zhang H, Strauss R, Johanns J, Adedokun OJ, Guzzo C, Colombel JF, Reinisch W, Gibson PR, Collins J, Jarnerot G, Rutgeerts P,. Subcutaneous golimumab maintains clinical response in patients with moderate-to-severe ulcerative colitis. Gastroenterology 2013; 146: 96-109.
86. Sandborn WJ, Feagan BG, Stoinov S, Honiball PJ, Rutgeerts P, Mason D, Bloomfield R, Schreiber S,. Certolizumab pegol for the treatment of Crohn's disease. N Engl J Med 2007; 357: 228-238.
87. Fleischmann R, Vencovsky J, van Vollenhoven RF, Borenstein D, Box J, Coteur G, Goel N, Brezinschek HP, Innes A, Strand V,. Efficacy and safety of certolizumab pegol monotherapy every 4 weeks in patients with rheumatoid arthritis failing previous disease-modifying antirheumatic therapy: the FAST4WARD study. Ann Rheum Dis 2009; 68: 805-811.
88. A study of the safety and efficacy of CNTO 148 (Golimumab) in children with Juvenile Idiopathic Arthritis (JIA) and multiple joint involvement who have poor response to methotrexate (GO KIDS). 2013. Available at http://clinicaltrials.gov/show/NCT01230827 (last accessed 20 June 2013).
89. Pediatric Arthritis Study of Certolizumab Pegol (PASCAL) U.S.: U.S. National Institutes of Health. 2012. Available at http://clinicaltrials.gov/ct2/show/NCT01550003?term=certolizumab+AND+JIA&rank=1 (last accessed 20 June 2013).
90. Lowite N, Porras O, Reiff A, Rudge S, Punaro M, Martin A, Allen R, Harville T, Sun YN, Bevirt T, Aras G, Appleton B,. Anakinra in the treatment of polyarticular-course juvenile rheumatoid arthritis: safety and preliminary efficacy results of a randomized multicenter study. Clin Rheumatol 2009; 28: 129-137.
91. Quartier P, Allantaz F, Cimaz R, Pillet P, Messiaen C, Bardin C, Bossuyt X, Boutten A, Bienvenu J, Duquesne A, Richer O, Chaussabel D, Mogenet A, Banchereau J, Treluyer JM, Landais P, Pascual V,. 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: 747-754.
92. Nigrovic PA, Mannion M, Prince FH, Zeft A, Rabinovich CE, van Rossum MA, Cortis E, Pardeo M, Miettunen PM, Janow G, Birmingham J, Eggebeen A, Janssen E, Shulman AI, Son MB, Hong S, Jones K, Ilowite NT, Cron RQ, Higgins GC,. 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: 545-555.
93. Gattorno M, Piccini A, Lasiglie D, Tassi S, Brisca G, Carta S, Delfino L, Ferlito F, Pelagatti MA, Caroli F, Buoncompagni A, Viola S, Loy A, Sironi M, Vecchi A, Ravelli A, Martini A, Rubartelli A,. The pattern of response to anti-interleukin-1 treatment distinguishes two subsets of patients with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum 2008; 58: 1505-1515.
94. Zeft A, Hollister R, LaFleur B, Sampath P, Soep J, McNally B, Kunkel G, Schlesinger M, Bohnsack J,. Anakinra for systemic juvenile arthritis: the Rocky Mountain experience. J Clin Rheumatol 2009; 15: 161-164.
95. Lequerre T, Quartier P, Rosellini D, Alaoui F, De Bandt M, Mejjad O, Kone-Paut I, Michel M, Dernis E, Khellaf M, Limal N, Job-Deslandre C, Fautrel B, Le Loet X, Sibilia J,. 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 2008; 67: 302-308.
96. Caorsi R, Federici S, Gattorno M,. Biologic drugs in autoinflammatory syndromes. Autoimmun Rev 2012; 12: 81-86.
97. Lovell DJ, Giannini EH, Reiff AO, Kimura Y, Li S, Hashkes PJ, Wallace CA, Onel KB, Foell D, Wu R, Biedermann S, Hamilton JD, Radin AR,. Long-term safety and efficacy of rilonacept in patients with Systemic Juvenile Idiopathic Arthritis (sJIA). Arthritis Rheum 2013; 65: 2486-2496.
98. Quatrier P, Erguven M, Horneff G,. IL-1b Inhibition with Canakinumab in patients with systemic juvenile idiopathic arthritis: efficacy and safety outcomes from a single dose, placebo-controlled study. Ann Rheum Dis 2012; 71 (Suppl. 3): 705.
99. Ruperto N, Brunner HI, Quartier P, Constantin T, Wulffraat N, Horneff G, Brik R, McCann L, Kasapcopur O, Rutkowska-Sak L, Schneider R, Berkun Y, Calvo I, Erguven M, Goffin L, Hofer M, Kallinich T, Oliveira SK, Uziel Y, Viola S, Nistala K, Wouters C, Cimaz R, Ferrandiz MA, Flato B, Gamir ML, Kone-Paut I, Grom A, Magnusson B, Ozen S, Sztajnbok F, Lheritier K, Abrams K, Kim D, Martini A, Lovell DJ,. Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med 2012; 367: 2396-2406.
100. Food and Drugs Administration. Drug approvals and databases. 2014. Available at http://www.fda.gov (last accessed 26 March 2014).
101. Ruperto N, Quartier P, Wulffraat N, Woo P, Ravelli A, Mouy R, Bader-Meunier B, Vastert SJ, Noseda E, D'Ambrosio D, Lecot J, Chakraborty A, Martini A, Chioato A, Paediatric Rheumatology International Clinical Trials Organisation. A phase II, multicenter, open-label study evaluating dosing and preliminary safety and efficacy of canakinumab in systemic juvenile idiopathic arthritis with active systemic features. Arthritis Rheum 2012; 64: 557-567.
102. Kutukculer N, Caglayan S, Aydogdu F,. Study of pro-inflammatory (TNF-alpha, IL-1alpha, IL-6) and T-cell-derived (IL-2, IL-4) cytokines in plasma and synovial fluid of patients with juvenile chronic arthritis: correlations with clinical and laboratory parameters. Clin Rheumatol 1998; 17: 288-292.
103. De Benedetti F, Massa M, Pignatti P, Albani S, Novick D, Martini A,. Serum soluble interleukin 6 (IL-6) receptor and IL-6/soluble IL-6 receptor complex in systemic juvenile rheumatoid arthritis. J Clin Invest 1994; 93: 2114-2119.
104. Yokota S, Miyamae T, Imagawa T, Iwata N, Katakura S, Mori M, Woo P, Nishimoto N, Yoshizaki K, Kishimoto T,. Therapeutic efficacy of humanized recombinant anti-interleukin-6 receptor antibody in children with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum 2005; 52: 818-825.
105. De Benedetti F, Brunner HI, Ruperto N, Kenwright A, Wright S, Calvo I, Cuttica R, Ravelli A, Schneider R, Woo P, Wouters C, Xavier R, Zemel L, Baildam E, Burgos-Vargas R, Dolezalova P, Garay SM, Merino R, Joos R, Grom A, Wulffraat N, Zuber Z, Zulian F, Lovell D, Martini A,. Randomized trial of tocilizumab in systemic juvenile idiopathic arthritis. N Engl J Med 2012; 367: 2385-2395.
106. Yokota S, Imagawa T, Mori M, Miyamae T, Takei S, Iwata N, Umebayashi H, Murata T, Miyoshi M, Tomiita M, Nishimoto N, Kishimoto T,. Long-term treatment of systemic juvenile idiopathic arthritis with tocilizumab: results of an open-label extension study in Japan. Ann Rheum Dis 2013; 72: 627-628.
107. Zulian F, Balzarin M, Falcini F, Martini G, Alessio M, Cimaz R, Ciminoz L, Zannin ME,. Abatacept for severe anti-tumor necrosis factor alpha refractory juvenile idiopathic arthritis-related uveitis. Arthritis Care Res 2010; 62: 821-825.
108. Runner HI, Ruperto N, Zuber Z, Keane C, Harari O, Kenwright A, Cuttica R,. Efficacy and safety of tocilizumab in patients with polyarticular juvenile idiopathic arthritis: data from a phase 3 trial. Arthritis Rheum 2012; 64: S682.
109. Ruperto N, Lovell DJ, Quartier P, Paz E, Rubio-Perez N, Silva CA, Abud-Mendoza C, Burgos-Vargas R, Gerloni V, Melo-Gomes JA, Saad-Magalhaes C, Sztajnbok F, Goldenstein-Schainberg C, Scheinberg M, Penades IC, Fischbach M, Orozco J, Hashkes PJ, Hom C, Jung L, Lepore L, Oliveira S, Wallace CA, Sigal LH, Block AJ, Covucci A, Martini A, Giannini EH,. Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trial. Lancet 2008; 372: 383-391.
110. Ruperto N, Lovell DJ, Quartier P, Paz E, Rubio-Perez N, Silva CA, Abud-Mendoza C, Burgos-Vargas R, Gerloni V, Melo-Gomes JA, Saad-Magalhaes C, Chavez-Corrales J, Huemer C, Kivitz A, Blanco FJ, Foeldvari I, Hofer M, Horneff G, Huppertz HI, Job-Deslandre C, Loy A, Minden K, Punaro M, Nunez AF, Sigal LH, Block AJ, Nys M, Martini A, Giannini EH,. Long-term safety and efficacy of abatacept in children with juvenile idiopathic arthritis. Arthritis Rheum 2010; 62: 1792-1802.
111. Borker A, Choudhary N,. Rituximab. Indian Pediatr 2011; 48: 627-632.
112. Podolskaya A, Stadermann M, Pilkington C, Marks SD, Tullus K,. B cell depletion therapy for 19 patients with refractory systemic lupus erythematosus. Arch Dis Child 2008; 93: 401-406.
113. Willems M, Haddad E, Niaudet P, Kone-Paut I, Bensman A, Cochat P, Deschenes G, Fakhouri F, Leblanc T, Llanas B, Loirat C, Pillet P, Ranchin B, Salomon R, Ulinski T, Bader-Meunier B,. Rituximab therapy for childhood-onset systemic lupus erythematosus. J Pediatr 2006; 148: 623-627.
114. Alexeeva EI, Valieva SI, Bzarova TM, Semikina EL, Isaeva KB, Lisitsyn AO, Denisova RV, Chistyakova EG,. Efficacy and safety of repeat courses of rituximab treatment in patients with severe refractory juvenile idiopathic arthritis. Clin Rheumatol 2011; 30: 1163-1172.
115. Jansson AF, Sengler C, Kuemmerle-Deschner J, Gruhn B, Kranz AB, Lehmann H, Kleinert D, Pape L, Girschick HJ, Foeldvari I, Haffner D, Haas JP, Moebius D, Foell D, Peitz J, Grote V,. B cell depletion for autoimmune diseases in paediatric patients. Clin Rheumatol 2011; 30: 87-97.
116. Navarra SV, Guzman RM, Gallacher AE, Hall S, Levy RA, Jimenez RE, Li EK, Thomas M, Kim HY, Leon MG, Tanasescu C, Nasonov E, Lan JL, Pineda L, Zhong ZJ, Freimuth W, Petri MA,. Efficacy and safety of belimumab in patients with active systemic lupus erythematosus: a randomised, placebo-controlled, phase 3 trial. Lancet 2011; 377: 721-731.
117. Furie R, Petri M, Zamani O, Cervera R, Wallace DJ, Tegzova D, Sanchez-Guerrero J, Schwarting A, Merrill JT, Chatham WW, Stohl W, Ginzler EM, Hough DR, Zhong ZJ, Freimuth W, van Vollenhoven RF,. A phase III, randomized, placebo-controlled study of belimumab, a monoclonal antibody that inhibits B lymphocyte stimulator, in patients with systemic lupus erythematosus. Arthritis Rheum 2011; 63: 3918-3930.
118. Lehman TJ,. Should the Food and Drug Administration warning of malignancy in children receiving tumor necrosis factor alpha blockers change the way we treat children with juvenile idiopathic arthritis? Arthritis Rheum 2010; 62: 2183-2184.
119. Simard JF, Neovius M, Hagelberg S, Askling J,. Juvenile idiopathic arthritis and risk of cancer: a nationwide cohort study. Arthritis Rheum 2010; 62: 3776-3782.
120. Horneff G, Foeldvari I, Minden K, Moebius D, Hospach T,. Report on malignancies in the German juvenile idiopathic arthritis registry. Rheumatol (Oxford) 2011; 50: 230-236.
121. Cleary AG, McDowell H, Sills JA,. Polyarticular juvenile idiopathic arthritis treated with methotrexate complicated by the development of non-Hodgkin's lymphoma. Arch Disease Child 2002; 86: 47-49.
122. Swart JF, de Roock S, Wulffraat NM,. What are the immunological consequences of long-term use of biological therapies for juvenile idiopathic arthritis? Arthritis Res Ther 2013; 15: 213.
123. Lepore L, Marchetti F, Facchini S, Leone V, Ventura A,. Drug-induced systemic lupus erythematosus associated with etanercept therapy in a child with juvenile idiopathic arthritis. Clin Exp Rheumatol 2003; 21: 276-277.
124. Kanakoudi-Tsakalidou F, Tzimouli V, Pratsidou-Gertsi P, Chronopoulou E, Trachana M,. The significance of persistent newly developed autoantibodies in JIA patients under long-term anti-TNF treatment. Cytokine 2008; 42: 293-297.
125. Hashkes PJ, Uziel Y, Laxer RM,. The safety profile of biologic therapies for juvenile idiopathic arthritis. Nat Rev Rheumatol 2010; 6: 561-571.
126. Bout-Tabaku S, Rivas-Chacon R, Restrepo R,. Systemic lupus erythematosus in a patient treated with etanercept for polyarticular juvenile rheumatoid arthritis. J Rheumatol 2007; 34: 2503-2504.
127. Zannin ME, Birolo C, Gerloni VM, Miserocchi E, Pontikaki I, Paroli MP, Bracaglia C, Shardlow A, Parentin F, Cimaz R, Simonini G, Falcini F, Corona F, Viola S, De Marco R, Breda L, La Torre F, Vittadello F, Martini G, Zulian F,. Safety and efficacy of infliximab and adalimumab for refractory uveitis in juvenile idiopathic arthritis: 1-year followup data from the Italian Registry. J Rheumatol 2013; 40: 74-79.