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Outcome Scores in Pediatric Rheumatology

  • Pediatric Rheumatology (S Ozen, Section Editor)
  • Published:
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Abstract

Purpose of Review

Providing a summary of the latest research on outcome measures in juvenile idiopathic arthritis, childhood -onset systemic lupus erythematosus, and juvenile dermatomyositis.

Recent Findings

A rational management of patients with pediatric rheumatic diseases requires the regular assessment of the level of disease activity and damage, as well as the evaluation of therapeutic response through validated and standardized outcome measures. Ideally, such tools should be simple, feasible, and easily applicable in routine care. Recently, there has been a great deal of effort to refine existing tools and devise novel outcome measures, aiming to address the various aspects of disease impact and to improve the reliability of research studies and clinical trials.

Summary

The newest outcome tools in pediatric rheumatology have markedly enlarged the spectrum of health domains assessable in a standardized way, thus increasing the reliability of evaluation of clinical response and fostering future clinical trials.

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References

  1. Brunner HI, Ravelli A. Develo** outcome measures for paediatric rheumatic diseases. Best Pract Res Clin Rheumatol. 2009;23:609–24.

    Article  PubMed  Google Scholar 

  2. Luca N, Feldman B. Health outcomes of pediatric rheumatic diseases. Best Pr Res Clin Rheumatol. 2014;28:331–50.

    Article  Google Scholar 

  3. Filocamo G, Consolaro A, Ferrari C, Ravelli A. Introducing new tools for assessment of parent- and child-reported outcomes in paediatric rheumatology practice: a work in progress. Clin Exp Rheumatol. 2013;31:964–8.

    PubMed  Google Scholar 

  4. Filocamo G, Consolaro A, Schiappapietra B, Ruperto N, Pistorio A, Solari N, et al. Parent and child acceptable symptom state in juvenile idiopathic arthritis. J Rheumatol. 2012;39:856–63.

    Article  PubMed  Google Scholar 

  5. Silva CA, Avcin T, Brunner HI. Taxonomy for systemic lupus erythematosus with onset before adulthood. Arthritis Care Res. 2012;64:1787–93.

    Article  Google Scholar 

  6. Ravelli A, Martini A. Juvenile idiopathic arthritis. Lancet (London, England). 2007;369:767–78.

    Article  CAS  Google Scholar 

  7. Viola S, Felici E, Magni-Manzoni S, Pistorio A, Buoncompagni A, Ruperto N, et al. Development and validation of a clinical index for assessment of long-term damage in juvenile idiopathic arthritis. Arthritis Rheum. 2005;52:2092–102.

    Article  PubMed  Google Scholar 

  8. Brunner HI, Giannini EH. Health-related quality of life in children with rheumatic diseases. Curr Opin Rheumatol. 2003;15:602–12.

    Article  PubMed  Google Scholar 

  9. Duffy C. Measurement of health status, functional sta¬tus, and quality of life in children with juvenile idiopathic arthritis: clinical science for the pediatrician. Pediatr Clin N Am. 2005;52:359–72.

    Article  Google Scholar 

  10. Wallace CA, Giannini EH, Huang B, Itert L, Ruperto N. American College of Rheumatology provisional criteria for defining clinical inactive disease in select categories of juvenile idiopathic arthritis. Arthritis Care Res. 2011;63:929–36.

    Article  Google Scholar 

  11. Ruperto N, Ravelli A, Pistorio A, Malattia C, Cavuto S, Gado-West L, et al. Cross-cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries. Review of the general methodology. Clin Exp Rheumatol. 2001;19:S1–9.

    CAS  PubMed  Google Scholar 

  12. Giannini EH. Redundancy of conventional articular response variables used in juvenile chronic arthritis clinical trials. Ann Rheum Dis. 1996;55.

  13. Giannini EH, Ruperto N, Ravelli A, Lovell DJ, Felson DT, Martini A. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum. 1997;40:1202–9.

    Article  CAS  PubMed  Google Scholar 

  14. Ruperto N, Murray KJ, Gerloni V, Wulffraat N, de Oliveira SKF, Falcini F, et al. A randomized trial of parenteral methotrexate comparing an intermediate dose with a higher dose in children with juvenile idiopathic arthritis who failed to respond to standard doses of methotrexate. Arthritis Rheum. 2004;50:2191–201.

    Article  CAS  PubMed  Google Scholar 

  15. Lovell DJ, Giannini EH, Reiff A, Cawkwell GD, Silverman ED, Nocton JJ, et al. Etanercept in children with polyarticular juvenile rheumatoid arthritis. N Engl J Med. 2000;342:763–9.

    Article  CAS  PubMed  Google Scholar 

  16. Ruperto N, Lovell DJ, Quartier P, Paz E, Rubio-Pérez N, Silva C a, et al. Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trial. Lancet. 2008;372:383–91.

    Article  CAS  PubMed  Google Scholar 

  17. Lovell DJ, Ruperto N, Goodman S, Reiff A, Jung L, Jarosova K, et al. Adalimumab with or without methotrexate in juvenile rheumatoid arthritis. N Engl J Med. 2008;359:810–20.

    Article  CAS  PubMed  Google Scholar 

  18. Ruperto N, Lovell DJ, Cuttica R, Wilkinson N, Woo P, Espada G, 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.

    Article  CAS  PubMed  Google Scholar 

  19. De Benedetti F, Brunner HI, Ruperto N, Kenwright A, Wright S, Calvo I, et al. Randomized trial of tocilizumab in systemic juvenile idiopathic arthritis. N Engl J Med. 2012;367:2385–95.

    Article  PubMed  CAS  Google Scholar 

  20. Ruperto N, Brunner HI, Quartier P, Constantin T, Wulffraat N, Horneff G, et al. Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med. 2012;367:2396–406.

    Article  CAS  PubMed  Google Scholar 

  21. Brunner HI, Ruperto N, Zuber Z, Keane C, Harari O, Kenwright A, et al. Efficacy and safety of tocilizumab in patients with polyarticular-course juvenile idiopathic arthritis: Results from a phase 3, randomised, double-blind withdrawal trial. Ann Rheum Dis. 2015;74:1110–7.

    Article  CAS  PubMed  Google Scholar 

  22. Brunner HI, Ruperto N, Tzaribachev N, Horneff G, Chasnyk VG, Panaviene V, et al. Subcutaneous golimumab for children with active polyarticular-course juvenile idiopathic arthritis: results of a multicentre, double-blind, randomised-withdrawal trial. Ann Rheum Dis. 2018 Jan;77(1):21–9.

    Article  CAS  PubMed  Google Scholar 

  23. Ruperto N, Martini A. Current and future perspectives in the management of juvenile idiopathic arthritis. Lancet Child Adolesc Health. 2018;2:360–70.

    Article  PubMed  Google Scholar 

  24. Morgan EM, Munro JE, Horonjeff J, Horgan B, Shea B, Feldman BM, et al. Establishing an updated core domain set for studies in juvenile idiopathic arthritis: A report from the OMERACT 2018 JIA workshop. J Rheumatol. 2019;46:1006–13.

    Article  PubMed  Google Scholar 

  25. Ruperto N, Martini A. Networking in paediatrics: the example of the Paediatric Rheumatology International Trials Organisation (PRINTO). Arch Dis Child. 2011;96:596–601.

    Article  PubMed  Google Scholar 

  26. Wallace CA, Ruperto N, Giannini E, Arthritis C. Preliminary Criteria for Clinical Remission for Select Categories of Juvenile Idiopathic Arthritis re er d. J Rheumatol. 2004;31:2290–4.

    PubMed  Google Scholar 

  27. Ravelli A, Consolaro A, Horneff G, Laxer RM, Lovell DJ, Wulffraat NM, et al. Treating juvenile idiopathic arthritis to target: recommendations of an international task force. Ann Rheum Dis. 2018;77:819–28.

    PubMed  Google Scholar 

  28. Consolaro A, Ruperto N, Bazso A, Pistorio A, Magni-Manzoni S, Filocamo G, et al. Development and validation of a composite disease activity score for juvenile idiopathic arthritis. Arthritis Rheum. 2009;61:658–66.

    Article  PubMed  Google Scholar 

  29. Consolaro A, Bracciolini G, Ruperto N, Pistorio A, Magni-Manzoni S, Malattia C, et al. Remission, minimal disease activity, and acceptable symptom state in juvenile idiopathic arthritis: Defining criteria based on the juvenile arthritis disease activity score. Arthritis Rheum. 2012;64:2366–74.

    Article  PubMed  Google Scholar 

  30. Nordal EB, Zak M, Aalto K, Berntson L, Fasth A, Herlin T, et al. Validity and predictive ability of the juvenile arthritis disease activity score based on CRP versus ESR in a Nordic population-based setting. Ann Rheum Dis. 2012;71:1122–7.

    Article  CAS  PubMed  Google Scholar 

  31. McErlane F, Beresford MW, Baildam EM, Chieng SEA, Davidson JE, Foster HE, et al. Validity of a three-variable Juvenile Arthritis Disease Activity Score in children with new-onset juvenile idiopathic arthritis. Ann Rheum Dis. 2013;72:1983–8.

    Article  PubMed  Google Scholar 

  32. Swart JF, van Dijkhuizen EHP, Wulffraat NM, de Roock S. Clinical Juvenile Arthritis Disease Activity Score proves to be a useful tool in treat-to-target therapy in juvenile idiopathic arthritis. Ann Rheum Dis. 2018;77:336–42.

    Article  CAS  PubMed  Google Scholar 

  33. Tibaldi J, Pistorio A, Aldera E, Puzone L, El Miedany Y, Pal P, et al. Development and initial validation of a composite disease activity score for systemic juvenile idiopathic arthritis. Rheumatology (Oxford). 2020;59:3505–14.

    Article  CAS  Google Scholar 

  34. Consolaro A, Ruperto N, Bracciolini G, Frisina A, Gallo MC, Pistorio A, et al. Defining criteria for high disease activity in juvenile idiopathic arthritis based on the Juvenile Arthritis Disease Activity Score. Ann Rheum Dis. 2014;73:1380–3.

    Article  PubMed  Google Scholar 

  35. Consolaro A, Calandra S, Robbiano C, Ravelli A. Treating juvenile idiopathic arthritis according to JADAS-based targets. Ann Paediatr Rheumatol. 2014;3:4.

    Article  Google Scholar 

  36. Consolaro A, Schiappapietra B, Dalprà S, Calandra S, Martini A, Ravelli A. Optimisation of disease assessments in juvenile idiopathic arthritis. Clin Exp Rheumatol. 2014;32:S126–30.

    Google Scholar 

  37. Consolaro A, Verazza S, Gallo M, Bracciolini G, Negro G, Frisina A. Development and validation of the cutoff values for disease activity states of the three-item version of the Juvenile Arthritis Disease Activity Score. Arthritis Rheum. 2013;65:S121–2.

    Google Scholar 

  38. Magni-Manzoni S, Ruperto N, Pistorio A, Sala E, Solari N, Palmisani E, et al. Development and validation of a preliminary definition of minimal disease activity in patients with juvenile idiopathic arthritis. Arthritis Rheum. 2008;59:1120–7.

    Article  PubMed  Google Scholar 

  39. Backström M, Tynjälä P, Ylijoki H, Aalto K, Kärki J, Pohjankoski H, et al. Finding specific 10-joint Juvenile Arthritis Disease Activity Score (JADAS10) and clinical JADAS10 cut-off values for disease activity levels in non-systemic juvenile idiopathic arthritis: A Finnish multicentre study. Rheumatol (United Kingdom). 2016;55:615–23.

    Google Scholar 

  40. Beukelman T, Patkar NM, Saag KG, Tolleson-Rinehart S, Cron RQ, DeWitt EM, et al. 2011 American College of Rheumatology Recommendations for the treatment of juvenile idiopathic arthritis: initiation and safety monitoring of therapeutic agents for the treatment of arthritis and systemic features. Arthritis Care Res. 2011;63:465–82.

    Article  Google Scholar 

  41. Weiss PF, Colbert RA, **ao R, Feudtner C, Beukelman T, Dewitt EM, et al. Development and retrospective validation of the juvenile spondyloarthritis disease activity index. Arthritis Care Res. 2014;66:1775–82.

    Article  Google Scholar 

  42. Swart J, Giancane G, Horneff G, Magnusson B, Hofer M, Alexeeva E, et al. Pharmacovigilance in juvenile idiopathic arthritis patients treated with biologic or synthetic drugs: combined data of more than 15,000 patients from Pharmachild and national registries. Arthritis Res Ther. 2018;20:1–11.

    Article  CAS  Google Scholar 

  43. Stichweh D, Arce E, Pascual V. Update on pediatric systemic lupus erythematosus. Curr Opin Rheumatol. 2004;16:577–87.

    Article  CAS  PubMed  Google Scholar 

  44. Hiraki L, Feldman C, Liu J, Alarcón G, Fischer M, Winkelmayer W, et al. Prevalence, incidence, and demographics of systemic lupus erythematosus and lupus nephritis from 2000 to 2004 among children in the US Medicaid beneficiary population. Arthritis Rheum. 2012;64:2669–76.

    Article  PubMed  PubMed Central  Google Scholar 

  45. van Vollenhoven R, Mosca M, Bertsias G, Isenberg D, Kuhn A, Lerstrøm K, et al. Treat-to-target in systemic lupus erythematosus: recommendations from an international task force. Ann Rheum Dis. 2004;73:958–67.

    Article  Google Scholar 

  46. Ruperto N, Ravelli A, Murray K, Lovell D, Andersson-Gare B, Feldman B, et al. Preliminary core sets of measures for disease activity and damage assessment in juvenile systemic lupus erythematosus and juvenile dermatomyositis. Rheumatol. 2003;42:1452–9.

    Article  Google Scholar 

  47. Ruperto N, Ravelli A, Cuttica R, Espada G, Ozen S, Porras O, et al. The Pediatric Rheumatology International Trials Organization criteria for the evaluation of response to therapy in juvenile systemic lupus erythematosus: prospective validation of the disease activity core set. Arthritis Rheum. 2005;52:2854–64.

    Article  PubMed  Google Scholar 

  48. Brunner HI, Abud-Mendoza C, Viola DO, Calvo Penades I, Levy D, Anton J, et al. Safety and efficacy of intravenous belimumab in children with systemic lupus erythematosus: Results from a randomised, placebo-controlled trial. Ann Rheum Dis. 2020;79:1340–8.

    Article  CAS  PubMed  Google Scholar 

  49. Brunner HI, Martini A, Lovell DJ, Ruperto N. Clinical trials in children and adolescents with systemic lupus erythematosus: methodological aspects, regulatory landscape and future opportunities. Ann Rheum Dis. 2019;78:162–70.

    Article  PubMed  Google Scholar 

  50. Mina R, Klein-Gitelman M, Ravelli A, Beresford M, Avcin T, Espada G, et al. Inactive disease and remission in childhood-onset systemic lupus erythematosus. Arthritis Care Res. 2012;64:683–93.

    Article  CAS  Google Scholar 

  51. Brunner H, Holland M, Beresford M, Ardoin S, Appenzeller S, Silva C, et al. American College of Rheumatology provisional criteria for global flares in childhood-onset systemic lupus erythematosus. Arthritis Care Res. 2018;70:813–22.

    Article  Google Scholar 

  52. Brunner H, Holland M, Beresford M, Ardoin S, Appenzeller S, Silva C, et al. American College of Rheumatology provisional criteria for clinically relevant improvement in children and adolescents with childhood-onset systemic lupus erythematosus. Arthritis Care Res. 2019;71:579–90.

    Article  Google Scholar 

  53. Symmons D, Coppock J, Bacon P, Bresnihan B, Isenberg D, Maddison P, et al. Development and assessment of a computerized index of clinical disease activity in systemic lupus erythematosus. Members of the British Isles Lupus Assessment Group (BILAG). Q J Med. 1988;69:927–37.

    CAS  PubMed  Google Scholar 

  54. Yee C, Cresswell L, Farewell V, Rahman A, Teh L, Griffiths B, et al. Numerical scoring for the BILAG-2004 index. Rheumatol. 2010;49:1665–9.

    Article  Google Scholar 

  55. Pope J. The revised BILAG Index with numerical scoring in systemic lupus erythematosus: added value with some limitations. Rheumatol. 2010;49:1616–7.

    Article  Google Scholar 

  56. Navarra SV, Guzmán RM, Gallacher AE, Hall S, Levy RA, Jimenez RE, et al. Efficacy and safety of belimumab in patients with active systemic lupus erythematosus: A randomised, placebo-controlled, phase 3 trial. Lancet. 2011;377:721–31.

    Article  CAS  PubMed  Google Scholar 

  57. Furie R, Rovin BH, Houssiau F, Malvar A, Teng YKO, Contreras G, et al. Two-Year, Randomized, Controlled Trial of Belimumab in Lupus Nephritis. N Engl J Med. 2020;383:1117–28.

    Article  CAS  PubMed  Google Scholar 

  58. Furie R, Petri M, Zamani O, Cervera R, Wallace DJ, Tegzová D, et al. 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;63:3918–30.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  59. Vitali C, Bencivelli W, Isenberg D, Smolen J, Snaith M, Sciuto M, et al. Disease activity in systemic lupus erythematosus: report of the Consensus Study Group of the European Workshop for Rheumatology Research. I. A descriptive analysis of 704 European lupus patients. European Consensus Study Group for Disease Activity in SLE. Clin Exp Rheumatol. 1992;10:527–39.

    CAS  PubMed  Google Scholar 

  60. Brunner H, Silverman E, Bombardier C, Feldman B. European Consensus Lupus Activity Measurement is sensitive to change in disease activity in childhood-onset systemic lupus erythematosus. Arthritis Rheum. 2003;49:335–41.

    Article  PubMed  Google Scholar 

  61. Schned E, Glickstein S, Doyle M. Derivation of the SLEDAI. Arthritis Rheum. 2003;36:877–8.

    Article  Google Scholar 

  62. Brunner H, Feldman B, Bombardier C, Silverman E. Sensitivity of the Systemic Lupus Erythematosus Disease Activity Index, British Isles Lupus Assessment Group Index, and Systemic Lupus Activity Measure in the evaluation of clinical change in childhood-onset systemic lupus erythematosus. Arthritis Rheum. 1999;42:1354–60.

    Article  CAS  PubMed  Google Scholar 

  63. Gladman D, Ibañez D, Urowitz M. Systemic lupus erythematosus disease activity index 2000. J Rheumatol. 2002;29:288–91.

    PubMed  Google Scholar 

  64. Guzmán J, Cardiel M, Arce-Salinas A, Sánchez-Guerrero J, Alarcón-Segovia D. Measurement of disease activity in systemic lupus erythematosus. Prospective validation of 3 clinical indices. J Rheumatol. 1992;19:1551–8.

    PubMed  Google Scholar 

  65. Lattanzi B, Consolaro A, Solari N, Ruperto N, Martini A, Ravelli A. Measures of disease activity and damage in pediatric systemic lupus erythematosus: British Isles Lupus Assessment Group (BILAG), European Consensus Lupus Activity Measurement (ECLAM), Systemic Lupus Activity Measure (SLAM), Systemic Lupus Erythematosus Di. Arthritis Care Res. 2011;63:S112–7.

    Article  Google Scholar 

  66. Kushner CJ, Tarazi M, Gaffney RG, Feng R, Ardalan K, Brandling-Bennett HA, et al. Evaluation of the reliability and validity of the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) in paediatric cutaneous lupus among paediatric dermatologists and rheumatologists. Br J Dermatol. 2019;180:165–71.

    Article  CAS  PubMed  Google Scholar 

  67. Gladman D, Ginzler E, Goldsmith C, Fortin P, Liang M, Urowitz M, et al. The development and initial validation of the Systemic Lupus International Collaborating Clinics/American College of Rheumatology damage index for systemic lupus erythematosus. Arthritis Rheum. 1996;39:363–9.

    Article  CAS  PubMed  Google Scholar 

  68. Gladman D, Urowitz M, Goldsmith C, Fortin P, Ginzler E, Gordon C, et al. The reliability of the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index in patients with systemic lupus erythematosus. Arthritis Rheum. 1997;40:809–13.

    Article  CAS  PubMed  Google Scholar 

  69. Ravelli A, Ruperto N, Martini A. Outcome in juvenile onset systemic lupus erythematosus. Curr Opin Rheumatol. 2005;17:568–73.

    Article  PubMed  Google Scholar 

  70. Gutiérrez-Suárez R, Ruperto N, Gastaldi R, Pistorio A, Felici E, Burgos-Vargas R, et al. A proposal for a pediatric version of the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index based on the analysis of 1,015 patients with juvenile-onset systemic lupus erythematosus. Arthritis Rheum. 2006;54:2989–96.

    Article  PubMed  Google Scholar 

  71. Holland M, Beresford M, Feldman B, Huggins J, Norambuena X, Silva C, et al. Measuring Disease Damage and Its Severity in Childhood-Onset Systemic Lupus Erythematosus. Arthritis Care Res. 2018;70:1621–9.

    Article  Google Scholar 

  72. Feldman B, Rider L, Reed A, Pachman L. Juvenile dermatomyositis and other idiopathic inflammatory myopathies of childhood. Lancet. 2008;371:2201–12.

    Article  PubMed  Google Scholar 

  73. Symmons D, Sills J, Davis S. The incidence of juvenile dermatomyositis: results from a nation-wide study. Br J Rheumatol. 1995;34:732–6.

    Article  CAS  PubMed  Google Scholar 

  74. Mendez E, Lipton R, Ramsey-Goldman R, Roettcher P, Bowyer S, Dyer A, et al. US incidence of juvenile dermatomyositis, 1995-1998: results from the National Institute of Arthritis and Musculoskeletal and Skin Diseases Registry. Arthritis Rheum. 2003;49:300–5.

    Article  PubMed  Google Scholar 

  75. Ravelli A, Trail L, Ferrari C, Ruperto N, Pistorio A, Pilkington C, et al. Long-term outcome and prognostic factors of juvenile dermatomyositis: a multinational, multicenter study of 490 patients. Arthritis Care Res. 2010;62:63–72. https://doi.org/10.1002/acr.20015.

    Article  Google Scholar 

  76. Sanner H, Kirkhus E, Merckoll E, Tollisen A, Røisland M, Lie B, et al. Long-term muscular outcome and predisposing and prognostic factors in juvenile dermatomyositis: a case-control study. Arthritis Care Res. 2010;62:1103–11.

    Article  Google Scholar 

  77. Rosina S, Varnier GC, Mazzoni M, Lanni S, Malattia C, Ravelli A. Innovative research design to meet the challenges of clinical trials for juvenile dermatomyositis. Curr Rheumatol Rep. 2018;20.

  78. Ruperto N, Ravelli A, Pistorio A, Ferriani V, Calvo I, Ganser G, et al. The provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism Disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: a prospective va. Arthritis Rheum. 2008;59:4–13.

    Article  PubMed  Google Scholar 

  79. Lovell D, Lindsley C, Rennebohm R, Ballinger S, Bowyer S, Giannini E, et al. Development of validated disease activity and damage indices for the juvenile idiopathic inflammatory myopathies. II. The Childhood Myositis Assessment Scale (CMAS): a quantitative tool for the evaluation of muscle function. Arthritis Rheum. 1999;42:2213–9.

    Article  CAS  PubMed  Google Scholar 

  80. Rider L, Feldman B, Perez M, Rennebohm R, Lindsley C, Zemel L, et al. Development of validated disease activity and damage indices for the juvenile idiopathic inflammatory myopathies: I. Physician, parent, and patient global assessments. Juvenile Dermatomyositis Disease Activity Collaborative Study Group. Arthritis Rheum. 1997;40:1976–83.

    Article  CAS  PubMed  Google Scholar 

  81. Bode R, Klein-Gitelman M, Miller M, Lechman T, Pachman LM. Disease activity score for children with juvenile dermatomyositis: reliability and validity evidence. Arthritis Rheum. 2003;49:7–15.

    Article  PubMed  Google Scholar 

  82. Singh G, Athreya B, Fries J, Goldsmith D. Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis Rheum. 1994;37:1761–9.

    Article  CAS  PubMed  Google Scholar 

  83. Landgraf J, Abetz L, Ware J. The CHQ User’s Manual. First Edit. Boston: The Health InstituteNew England Medical Center; 1996.

    Google Scholar 

  84. Ruperto N, Ravelli A, Pistorio A, Ferriani V, Calvo I, Ganser G, et al. The Provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: A prospective validation study. Arthritis Care Res. 2008;59:4–13.

    Article  Google Scholar 

  85. Ruperto N, Pistorio A, Ravelli A, Rider L, Pilkington C, Oliveira S, et al. The Paediatric Rheumatology International Trials Organisation provisional criteria for the evaluation of response to therapy in juvenile dermatomyositis. Arthritis Care Res. 2010;62:1533–41.

    Article  Google Scholar 

  86. Ruperto N, Pistorio A, Oliveira S, Zulian F, Cuttica R, Ravelli A, et al. Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: A randomised trial. Lancet. 2016;387:671–8.

    Article  PubMed  Google Scholar 

  87. McCann LJ, Pilkington CA, Huber AM, Ravelli A, Appelbe D, Kirkham JJ, et al. Development of a consensus core dataset in juvenile dermatomyositis for clinical use to inform research. Ann Rheum Dis. 2018;77:241–50.

    Article  PubMed  Google Scholar 

  88. Almeida B, Campanilho-Marques R, Arnold K, Pilkington C, Wedderburn L, Nistala K, et al. Analysis of published criteria for clinically inactive disease in a large juvenile dermatomyositis cohort shows that skin disease is underestimated. Arthritis Rheum. 2015;67:2495–502.

    Article  Google Scholar 

  89. Rider L, Werth V, Huber A, Alexanderson H, Rao A, Ruperto N, et al. Measures for adult and juvenile dermatomyositis, polymyositis, and inclusion body myositis. Arthritis Care Res. 2011;63:S118–57.

    Article  Google Scholar 

  90. Escolar D, Henricson E, Mayhew J, Florence J, Leshner R, Patel K, et al. Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children. Muscle Nerve. 2001;24:787–93.

    Article  CAS  PubMed  Google Scholar 

  91. Harris-Love M, Shrader J, Koziol D, Pahlajani N, Jain M, Smith M, et al. Distribution and severity of weakness among patients with polymyositis, dermatomyositis and juvenile dermatomyositis. Rheumatol. 2009;48:134–9.

    Article  CAS  Google Scholar 

  92. Rider L, Koziol D, Giannini E, Jain M, Smith M, Whitney-Mahoney K, et al. Validation of manual muscle testing and a subset of eight muscles for adult and juvenile idiopathic inflammatory myopathies. Arthritis Care Res. 2010;62:465–72.

    Article  Google Scholar 

  93. Rennebohm R, Jones K, Huber A, Ballinger S, Bowyer S, Feldman B, et al. Normal scores for nine maneuvers of the Childhood Myositis Assessment Scale. Arthritis Rheum. 2009;51:365–70.

    Article  Google Scholar 

  94. Varnier GC, Rosina S, Ferrari C, Pistorio A, Consolaro A, Bovis F, et al. Development and testing of a hybrid measure of muscle strength in juvenile dermatomyositis for use in routine care. Arthritis Care Res. 2018;70:1312–9.

    Article  Google Scholar 

  95. Campanilho-Marques R, Almeida B, Deakin C, Arnold K, Gallot N, de Iorio M, et al. Comparison of the utility and validity of three scoring tools to measure skin involvement in patients with juvenile dermatomyositis. Arthritis Care Res. 2016;68:1514–21.

    Article  Google Scholar 

  96. Huber A, Dugan E, Lachenbruch P, Feldman B, Perez M, Zemel L, et al. The Cutaneous Assessment Tool: development and reliability in juvenile idiopathic inflammatory myopathy. Rheumatology (Oxford). 2007;46:1606–11.

    Article  CAS  Google Scholar 

  97. Huber A, Lachenbruch P, Dugan E, Miller F, Rider L, Group. JDDACS. Alternative scoring of the Cutaneous Assessment Tool in juvenile dermatomyositis: results using abbreviated formats. Arthritis Rheum. 2008;59:352–6.

    Article  PubMed  PubMed Central  Google Scholar 

  98. Anyanwu C, Fiorentino D, Chung L, Dzuong C, Wang Y, Okawa J, et al. Validation of the Cutaneous Dermatomyositis Disease Area and Severity Index: characterizing disease severity and assessing responsiveness to clinical change. Br J Dermatol. 2015;173:969–74.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  99. Tiao J, Feng R, Berger E, Brandsema J, Coughlin C, Khan N, et al. Evaluation of the reliability of the Cutaneous Dermatomyositis Disease Area and Severity Index and the Cutaneous Assessment Tool-Binary Method in juvenile dermatomyositis among paediatric dermatologists, rheumatologists and neurologists. Br J Dermatol. 2017;177:1086–92.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  100. Sultan S, Allen E, Oddis C, Kiely P, Cooper R, Lundberg I, et al. Reliability and validity of the myositis disease activity assessment tool. Arthritis Rheum. 2008;58:3593–9.

    Article  PubMed  Google Scholar 

  101. Hay E, Bacon P, Gordon C, Isenberg D, Maddison P, Snaith M, et al. The BILAG index: a reliable and valid instrument for measuring clinical disease activity in systemic lupus erythematosus. Q J Med. 1993;86:447–58.

    CAS  PubMed  Google Scholar 

  102. Rosina S, Consolaro A, Van Dijkhuizen P, Pistorio A, Varnier GC, Bovis F, et al. Development and validation of a composite disease activity score for measurement of muscle and skin involvement in juvenile dermatomyositis. Rheumatol (United Kingdom). 2019;58:1196–205.

    Google Scholar 

  103. Miller F, Rider L, Chung Y, Cooper R, Danko K, Farewell V, et al. Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies. Rheumatology (Oxford). 2001;40:1262–73.

    Article  CAS  Google Scholar 

  104. Stoll T, Seifert B, Isenberg D. Damage Index is valid, and renal and pulmonary organ scores are predictors of severe outcome in patients with systemic lupus erythematosus. Br J Rheumatol. 1996;35:248–54.

    Article  CAS  PubMed  Google Scholar 

  105. Rahman P, Gladman D, Urowitz M, Hallett D, Tam L. Early damage as measured by the SLICC/ACR damage index is a predictor of mortality in systemic lupus erythematosus. Lupus. 2001;10:93–6.

    Article  CAS  PubMed  Google Scholar 

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Acknowledgements

Note for the use of the tools cited in the manuscript.

For the use of each tool cited in the manuscript, readers should refer to the developer/copyright holders.

For JIA, please refer to reference [28].

For JDM, please refer to references [76,77,78].

For SLE, please refer to references [44,45,46].

Data Availability (Data Transparency)

The authors confirm that the data supporting the findings of this study are available within the articles reported in the bibliography

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Not applicable

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Authors and Affiliations

  1. Clinica Pediatrica e Reumatologia, IRCCS Istituto Giannina Gaslini, Genoa, Italy

    Gabriella Giancane, Silvia Rosina, Alessandro Consolaro & Nicolino Ruperto

  2. Dipartimento di Neuroscienze, Riabilitazione, Oftalmologia, Genetica e Scienze Materno-Infantili (DiNOGMI), Università degli Studi di Genova, Genoa, Italy

    Gabriella Giancane & Alessandro Consolaro

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  1. Gabriella Giancane
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  2. Silvia Rosina
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  4. Nicolino Ruperto
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Contributions

All authors contributed to literature review, manuscript drafting, critical revision of the paper, and final approval of the version submitted.

Corresponding author

Correspondence to Nicolino Ruperto.

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Conflict of Interest

Gabriella Giancane has no conflicts of interest to declare.

Silvia Rosina has no conflicts of interest to declare.

Alessandro Consolaro reports Investigator initiated research grant from Pfizer and speaker’s bureaus from Abbvie.

Nicolino Ruperto has received honoraria for consultancies or speaker bureaus (< 10.000 USD each) from the following pharmaceutical companies in the past 3 years: Ablynx, Astrazeneca-Medimmune, Bayer, Biogen, Boehringer, Bristol Myers and Squibb, Celgene, Eli-Lilly, EMD Serono, Glaxo Smith and Kline, Hoffmann-La Roche,Janssen, Merck, Novartis, Pfizer, R-Pharma, Sinergie, Sobi and UCB.

The IRCCS Istituto Giannina Gaslini (IGG), where NR works as full-time public employee has received contributions (> 10.000 USD each) from the following industries in the last 3 years: BMS, Eli-Lilly, GlaxoSmithKline, F Hoffmann-La Roche, Janssen, Novartis, Pfizer, Sobi. This funding has been reinvested for the research activities of the hospital in a fully independent manner, without any commitment with third parties.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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Giancane, G., Rosina, S., Consolaro, A. et al. Outcome Scores in Pediatric Rheumatology. Curr Rheumatol Rep 23, 23 (2021). https://doi.org/10.1007/s11926-021-00988-x

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