A 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)

Trials

Volumn 15, Issue 1, 2014, Pages

  Ramanan, Athimalaipet V ^a   Dick, Andrew D ^b   Benton, Diana ^a   Compeyrot Lacassagne, Sandrine ^c   Dawoud, Dalia ^e   Hardwick, Ben ^d   Hickey, Helen ^d   Hughes, Dyfrig ^e   Jones, Ashley ^d   Woo, Patricia ^c   Edelsten, Clive ^c   Beresford, Michael W ^d  

^a UNIVERSITY HOSPITALS BRISTOL NHS FOUNDATION TRUST   (United Kingdom)

^b BRISTOL EYE HOSPITAL   (United Kingdom)

^c GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^d UNIVERSITY OF LIVERPOOL   (United Kingdom)

^e BANGOR UNIVERSITY   (United Kingdom)

Author keywords

Adalimumab; Juvenile idiopathic arthritis; Methotrexate; Ophthalmology; Paediatric; Rheumatology; Safety; Uveitis

Indexed keywords

ADALIMUMAB; METHOTREXATE; PLACEBO;

ADOLESCENT; ADULT; ARTICLE; CHILD; CLINICAL EFFECTIVENESS; CONTROLLED STUDY; COST EFFECTIVENESS ANALYSIS; DISEASE ASSOCIATION; DRUG SAFETY; FEMALE; HUMAN; JUVENILE RHEUMATOID ARTHRITIS; MAJOR CLINICAL STUDY; MALE; PRESCHOOL CHILD; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD; UNSPECIFIED SIDE EFFECT; UVEITIS; YOUNG ADULT;

ADOLESCENT; ANTI-INFLAMMATORY AGENTS; ANTIBODIES, MONOCLONAL, HUMANIZED; ARTHRITIS, JUVENILE; CHILD; CHILD, PRESCHOOL; CLINICAL PROTOCOLS; COST-BENEFIT ANALYSIS; DRUG COSTS; DRUG THERAPY, COMBINATION; HUMANS; LONDON; METHOTREXATE; RESEARCH DESIGN; TIME FACTORS; TREATMENT OUTCOME; UVEITIS;

EID: 84892161005     PISSN: None     EISSN: 17456215     Source Type: Journal    
DOI: 10.1186/1745-6215-15-14     Document Type: Article

References (63)

1. Edelsten C, Reddy MA, Stanford MR, Graham EM. Visual loss associated with pediatric uveitis in English primary and referral centers. Am J Ophthalmol 2003, 135:676-680. 10.1016/S0002-9394(02)02148-7, 12719076.
2. Smith JA, Mackensen F, Sen HN, Leigh JF, Watkins AS, Pyatetsky D, Tessler HH, Nussenblatt RB, Rosenbaum JT, Reed GF, Vitale S, Smith JR, Goldstein DA. Epidemiology and course of disease in childhood uveitis. Ophthalmology 2009, 116:1544-1551.e1. 10.1016/j.ophtha.2009.05.002, 2937251, 19651312.
3. Kanski JJ. Uveitis in juvenile chronic arthritis: incidence, clinical features and prognosis. Eye (Lond) 1988, 2:641-645. 10.1038/eye.1988.118, 3256502.
4. Kotaniemi K, Kautiainen H, Karma A, Aho K. Occurrence of uveitis in recently diagnosed juvenile chronic arthritis: a prospective study. Ophthalmology 2001, 108:2071-2075. 10.1016/S0161-6420(01)00773-4, 11713082.
5. Saurenmann RK, Levin AV, Feldman BM, Rose JB, Laxer RM, Schneider R, Silverman ED. Prevalence, risk factors, and outcome of uveitis in juvenile idiopathic arthritis: a long-term followup study. Arthritis Rheum 2007, 56:647-657. 10.1002/art.22381, 17265500.
6. Chia A, Lee V, Graham EM, Edelsten C. Factors related to severe uveitis at diagnosis in children with juvenile idiopathic arthritis in a screening program. Am J Ophthalmol 2003, 135:757-762. 10.1016/S0002-9394(03)00225-3, 12788113.
7. Holland GN, Denove CS, Yu F. Chronic anterior uveitis in children: clinical characteristics and complications. Am J Ophthalmol 2009, 147:667-678.e5. 10.1016/j.ajo.2008.11.009, 19195641.
8. Woreta F, Thorne JE, Jabs DA, Kedhar SR, Dunn JP. Risk factors for ocular complications and poor visual acuity at presentation among patients with uveitis associated with juvenile idiopathic arthritis. Am J Ophthalmol 2007, 143:647-655. 10.1016/j.ajo.2006.11.025, 1892578, 17224116.
9. Edelsten C, Lee V, Bentley CR, Kanski JJ, Graham EM. An evaluation of baseline risk factors predicting severity in juvenile idiopathic arthritis associated uveitis and other chronic anterior uveitis in early childhood. Br J Ophthalmol 2002, 86:51-56. 10.1136/bjo.86.1.51, 1770968, 11801504.
10. Jabs DA, Nussenblatt RB, Rosenbaum JT. Standardization of Uveitis Nomenclature (SUN) Working Group Standardization of Uveitis Nomenclature for reporting clinical data: results of the First International Workshop. Am J Ophthalmol 2005, 140:509-516. Standardization of Uveitis Nomenclature (SUN) Working Group.
11. Thorne JE, Woreta F, Kedhar SR, Dunn JP, Jabs DA. Juvenile idiopathic arthritis-associated uveitis: incidence of ocular complications and visual acuity loss. Am J Ophthalmol 2007, 143:840-846. 10.1016/j.ajo.2007.01.033, 17362866.
12. Wolf MD, Lichter PR, Ragsdale CG. Prognostic factors in the uveitis of juvenile rheumatoid arthritis. Ophthalmology 1987, 94:1242-1248. 10.1016/S0161-6420(87)80007-6, 3684202.
13. de Boer J, Wulffraat N, Rothova A. Visual loss in uveitis of childhood. Br J Ophthalmol 2003, 87:879-884. 10.1136/bjo.87.7.879, 1771761, 12812891.
14. Takken T, van der Net JJ, Helders PPJM. Methotrexate for treating juvenile idiopathic arthritis. Cochrane Database Syst Rev 2001, 4. CD003129.
15. Beresford MW, Baildam EM. New advances in the management of juvenile idiopathic arthritis-1: non-biological therapy. Arch Dis Child Educ Pract Ed 2009, 94:144-150. 10.1136/adc.2008.144576, 19770494.
16. Foeldvari I, Wierk A. Methotrexate is an effective treatment for chronic uveitis associated with juvenile idiopathic arthritis. J Rheumatol 2005, 32:362-365.
17. Weiss AH, Wallace CA, Sherry DD. Methotrexate for resistant chronic uveitis in children with juvenile rheumatoid arthritis. J Pediatr 1998, 133:266-268. 10.1016/S0022-3476(98)70232-X, 9709718.
18. Yu EN, Meniconi ME, Tufail F, Baltatzis S, Foster CS. Outcomes of treatment with immunomodulatory therapy in patients with corticosteroid-resistant juvenile idiopathic arthritis-associated chronic iridocyclitis. Ocul Immunol Inflamm 2005, 13:353-360. 10.1080/09273940590951061, 16419420.
19. Sharma SM, Dick AD, Ramanan AV. Non-infectious pediatric uveitis: An update on immunomodulatory management. Paediatr Drugs 2009, 11:229-241. 10.2165/00148581-200911040-00002, 19566107.
20. Kalinina Ayuso V, van de Winkel EL, Rothova A, de Boer JH. Relapse rate of uveitis post-methotrexate treatment in juvenile idiopathic arthritis. Am J Ophthalmol 2011, 151:217-222. 10.1016/j.ajo.2010.08.021, 21145533.
21. Doycheva D, Deuter C, Stuebiger N, Biester S, Zierhut M. Mycophenolate mofetil in the treatment of uveitis in children. Br J Ophthalmol 2007, 91:180-184. 10.1136/bjo.2006.094698, 1857605, 16825275.
22. Kilmartin DJ, Forrester JV, Dick AD. Cyclosporin A therapy in refractory non-infectious childhood uveitis. Br J Ophthalmol 1998, 82:737-742. 10.1136/bjo.82.7.737, 1722678, 9924362.
23. Dick AD, Forrester JV, Liversidge J, Cope AP. The role of tumour necrosis factor (TNF-α) in experimental autoimmune uveoretinitis (EAU). Prog Retin Eye Res 2004, 23:617-637. 10.1016/j.preteyeres.2004.06.005, 15388077.
24. Imrie FR, Dick AD. Biologics in the treatment of uveitis. Curr Opin Ophthalmol 2007, 18:481-486. 10.1097/ICU.0b013e3282f03d42, 18163000.
25. Smith JR, Hart PH, Coster DJ, Williams KA. Mice deficient in tumor necrosis factor receptors p55 and p75, interleukin-4, or inducible nitric oxide synthase are susceptible to endotoxin-induced uveitis. Invest Ophthalmol Vis Sci 1998, 39:658-661.
26. Koizumi K, Poulaki V, Doehmen S, Welsandt G, Radetzky S, Lappas A, Kociok N, Kirchhof B, Joussen AM. Contribution of TNF-α to leukocyte adhesion, vascular leakage, and apoptotic cell death in endotoxin-induced uveitis in vivo. Invest Ophthalmol Vis Sci 2003, 44:2184-2191. 10.1167/iovs.02-0589, 12714660.
27. Diaz-Llopis M, García-Delpech S, Salom D, Udaondo P, Bosch-Morell F, Quijada A, Romero FJ, Amselem L. High-dose infliximab prophylaxis in endotoxin-induced uveitis. J Ocul Pharmacol Ther 2007, 23:343-350. 10.1089/jop.2006.0148, 17803432.
28. 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. 10.1136/bjo.2006.103721, 1857691, 17035274.
29. Foeldvari I, Nielsen S, Kümmerle-Deschner J, Espada G, Horneff G, Bica B, Olivieri AN, Wierk A, Saurenmann RK. Tumor necrosis factor-α blocker in treatment of juvenile idiopathic arthritis-associated uveitis refractory to second-line agents: results of a multinational survey. J Rheumatol 2007, 34:1146-1150.
30. Gallagher M, Quinones K, Cervantes-Castañeda RA, Yilmaz T, Foster CS. Biological response modifier therapy for refractory childhood uveitis. Br J Ophthalmol 2007, 91:1341-1344. 10.1136/bjo.2007.124081, 2000999, 17556427.
31. Saurenmann RK, Levin AV, Rose JB, Parker S, Rabinovitch T, Tyrrell PN, Feldman BM, Laxer RM, Schneider R, Silverman ED. Tumour necrosis factor α inhibitors in the treatment of childhood uveitis. Rheumatology (Oxford) 2006, 45:982-989. 10.1093/rheumatology/kel030, 16461435.
32. Sharma SM, Ramanan AV, Riley P, Dick AD. Use of infliximab in juvenile onset rheumatological disease-associated refractory uveitis: efficacy in joint and ocular disease. Ann Rheum Dis 2007, 66:840-841. 10.1136/ard.2006.065441, 1954647, 17170050.
33. Tynjälä P, Kotaniemi K, Lindahl P, Latva K, Aalto K, Honkanen V, Lahdenne P. Adalimumab in juvenile idiopathic arthritis-associated chronic anterior uveitis. Rheumatology (Oxford) 2008, 47:339-344.
34. Schmeling H, Horneff G. Etanercept and uveitis in patients with juvenile idiopathic arthritis. Rheumatology (Oxford) 2005, 44:1008-1011. 10.1093/rheumatology/keh658, 15855187.
35. Smith JA, Thompson DJ, Whitcup SM, Suhler E, Clarke G, Smith S, Robinson M, Kim J, Barron KS. A randomized, placebo-controlled, double-masked clinical trial of etanercept for the treatment of uveitis associated with juvenile idiopathic arthritis. Arthritis Rheum 2005, 53:18-23. 10.1002/art.20904, 15696578.
36. Hale S, Lightman S. Anti-TNF therapies in the management of acute and chronic uveitis. Cytokine 2006, 33:231-237. 10.1016/j.cyto.2005.12.012, 16542849.
37. Saurenmann RK, Levin AV, Feldman BM, Laxer RM, Schneider R, Silverman ED. Risk of new-onset uveitis in patients with juvenile idiopathic arthritis treated with anti-TNFα agents. J Pediatr 2006, 149:833-836. 10.1016/j.jpeds.2006.08.044, 17137902.
38. Taban M, Dupps WJ, Mandell B, Perez VL. Etanercept (Enbrel)-associated inflammatory eye disease: case report and review of the literature. Ocul Immunol Inflamm 2006, 14:145-150. 10.1080/09273940600659393, 16766397.
39. Lim LL, Fraunfelder FW, Rosenbaum JT. Do tumor necrosis factor inhibitors cause uveitis? A registry-based study. Arthritis Rheum 2007, 56:3248-3252. 10.1002/art.22918, 17907169.
40. Keystone EC, Kavanaugh AF, Sharp JT, Tannenbaum H, Hua Y, Teoh LS, Fischkoff SA, Chartash EK. Radiographic, clinical, and functional outcomes of treatment with adalimumab (a human anti-tumor necrosis factor monoclonal antibody) in patients with active rheumatoid arthritis receiving concomitant methotrexate therapy: a randomized, placebo-controlled, 52-week trial. Arthritis Rheum 2004, 50:1400-1411. 10.1002/art.20217, 15146409.
41. 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. Pediatric Rheumatology Collaborative Study Group; Pediatric Rheumatology International Trials Organisation Adalimumab with or without methotrexate in juvenile rheumatoid arthritis. N Engl J Med 2008, 359:810-820. 10.1056/NEJMoa0706290, 18716298, Pediatric Rheumatology Collaborative Study Group; Pediatric Rheumatology International Trials Organisation.
42. Vazquez-Cobian LB, Flynn T, Lehman TJ. Adalimumab therapy for childhood uveitis. J Pediatr 2006, 149:572-575. 10.1016/j.jpeds.2006.04.058, 17011337.
43. US Food and Drug Administration (FDA) Information for Healthcare Professionals: Tumor Necrosis Factor (TNF) Blockers (marketed as Remicade, Enbrel, Humira, Cimzia, and Simponi) FDA Alert (4 August 2009). Available at http://www.fda.gov/Drugs/DrugSafety/PostmarketDrugSafetyInformationforPatientsandProviders/DrugSafetyInformationforHeathcareProfessionals/ucm174474.htm (accessed 12 December 2013), US Food and Drug Administration (FDA).
44. Bernatsky S, Rosenberg A, Oen KG, Ramsey-Goldman R, St Pierre Y, Turnbull E, Clarke AE. Malignancy in juvenile idiopathic arthritis [abstract]. Arthritis Rheum 2009, 60(Suppl 10):250. 10.1002/art.25333.
45. McCroskery P, Wallace C, Lovell D, Stryker S, Chernyukhin N, Blosch C, Zack DJ. Summary of worldwide pediatric malignancies reported after exposure to etanercept [abstract]. Ann Rheum Dis 2010, 69(Suppl 3):627.
46. Harrison M, Cummins C, Horneff G, Southwood T, Mines D. Cancer risk in juvenile arthritis patients exposed to etanercept: results from three observational cohorts [abstract]. Ann Rheum Dis 2010, 69(Suppl 3):147.
47. Caspersen S, Elkjaer M, Riis L, Pedersen N, Mortensen C, Jess T, Sarto P, Hansen TS, Wewer V, Bendtsen F, Moesgaard F, Munkholm P. Danish Crohn Colitis Database Infliximab for inflammatory bowel disease in Denmark 1999-2005: clinical outcome and follow-up evaluation of malignancy and mortality. Clin Gastroenterol Hepatol 2008, 6:1212-1217. 10.1016/j.cgh.2008.05.010, 18848503, Danish Crohn Colitis Database.
48. de Vries HS, van Oijen MG, de Jong DJ. Serious events with infliximab in patients with inflammatory bowel disease: a 9-year cohort study in the Netherlands. Drug Saf 2008, 31:1135-1144.
49. Kempen JH, Daniel E, Dunn JP, Foster CS, Gangaputra S, Hanish A, Helzlsouer KJ, Jabs DA, Kaçmaz RO, Levy-Clarke GA, Liesegang TL, Newcomb CW, Nussenblatt RB, Pujari SS, Rosenbaum JT, Suhler EB, Thorne JE. Overall and cancer related mortality among patients with ocular inflammation treated with immunosuppressive drugs: retrospective cohort study. BMJ 2009, 339:b2480. 10.1136/bmj.b2480, 2714688, 19578087.
50. Nam JL, Winthrop KL, van Vollenhoven RF, Pavelka K, Valesini G, Hensor EMA, Worthy G, Landewé R, Smolen JS, Emery P, Buch MH. Current evidence for the management of rheumatoid arthritis with biological disease-modifying antirheumatic drugs: a systematic literature review informing the EULAR recommendations for the management of RA. Ann Rheum Dis 2010, 69:976-986. 10.1136/ard.2009.126573, 20447957.
51. 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.
52. Oliveira S, Ravelli A, Pistorio A, Castell E, Malattia C, Prieur AM, Saad-Magalhães C, Murray KJ, Bae SC, Joos R, Foeldvari I, Duarte-Salazar C, Wulffraat N, Lahdenne P, Dolezalova P, de Inocencio J, Kanakoudi-Tsakalidou F, Hofer M, Nikishina I, Ozdogan H, Hashkes PJ, Landgraf JM, Martini A, Ruperto N. Pediatric Rheumatology International Trials Organization (PRINTO) Proxy-reported health-related quality of life of patients with juvenile idiopathic arthritis: the Pediatric Rheumatology International Trials Organization multinational quality of life cohort study. Arthritis Rheum 2007, 57:35-43. 10.1002/art.22473, 17266064, Pediatric Rheumatology International Trials Organization (PRINTO).
53. Wallace CA, Ruperto N, Giannini E. Childhood Arthritis and Rheumatology Research Alliance; Pediatric Rheumatology International Trials Organization; Pediatric Rheumatology Collaborative Study Group Preliminary criteria for clinical remission for select categories of juvenile idiopathic arthritis. J Rheumatol 2004, 31:2290-2294. Childhood Arthritis and Rheumatology Research Alliance; Pediatric Rheumatology International Trials Organization; Pediatric Rheumatology Collaborative Study Group.
54. Magni-Manzoni S, Ruperto N, Pistorio A, Sala E, Solari N, Palmisani E, Cugno C, Bozzola E, Martini A, Ravelli A. Development and validation of a preliminary definition of minimal disease activity in patients with juvenile idiopathic arthritis. Arthritis Rheum 2008, 59:1120-1127. 10.1002/art.23916, 18668599.
55. Ilowite NT. Update on biologics in juvenile idiopathic arthritis. Curr Opin Rheumatol 2008, 20:613-618. 10.1097/BOR.0b013e3283060778, 18698187.
56. 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, et al. A randomized, placebo-controlled trial of infliximab plus methotrexate for the treatment of polyarticular-course juvenile rheumatoid arthritis. Arthritis Rheum 2007, 56:3096-3106. 10.1002/art.22838, 17763439.
57. Department of Health Reference Costs: 2011-12 Policy Paper (8 November 2012). Available at https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/213060/2011-12-reference-costs-publication.pdf (accessed 12 December 2013), Department of Health.
58. Curtis L. Unit Costs of Health & Social Care 2012 2012, Kent, UK: University of Kent, Personal Social Services Research Unit, Available at http://kar.kent.ac.uk/32408/1/full-with-covers.pdf (accessed 12 December 2013).
59. Glick HA, Doshi JA, Sonnad SS, Polsky D. Economic Evaluation in Clinical Trials (Handbooks in Health Economic Evaluation) 2007, Oxford: Oxford University Press.
60. Briggs A, Sculpher M, Claxton K. Decision Modelling for Health Economic Evaluation (Handbooks in Health Economic Evaluation) 2006, Oxford: Oxford University Press.
61. Kotaniemi K, Arkela-Kautiainen M, Haapasaari J, Leirisalo-Repo M. Uveitis in young adults with juvenile idiopathic arthritis: a clinical evaluation of 123 patients. Ann Rheum Dis 2005, 64:871-874. 10.1136/ard.2004.026955, 1755508, 15897307.
62. Carvounis PE, Herman DC, Cha S, Burke JP. Incidence and outcomes of uveitis in juvenile rheumatoid arthritis, a synthesis of the literature. Graefes Arch Clin Exp Ophthalmol 2006, 244:281-290. 10.1007/s00417-005-0087-3, 16228217.
63. National Institute for Health and Care Excellence (NICE) Guide to the Methods of Technology Appraisal (Process and Methods Guides) 2013, London: NICE, Available at http://www.nice.org.uk/media/D45/1E/GuideToMethodsTechnologyAppraisal2013.pdf (accessed 12 December 2013), National Institute for Health and Care Excellence (NICE).