Best Practice & Research Clinical Rheumatology
Volume 23, Issue 5 , Pages 609-624 , October 2009

Developing outcome measures for paediatric rheumatic diseases

  • Hermine I. Brunner, MD MSc

      Affiliations

    • Cincinnati Children's Hospital Medical Center, University of Cincinnati, William Rowe Division of Rheumatology, E 4010, 3333 Burnet Avenue, Cincinnati, OH 45229-3039, USA
    • Corresponding Author InformationCorresponding author. Tel.: +1 513 636 7467; Fax: +1 513 636 5990.
  • ,
  • Angelo Ravelli, MD

      Affiliations

    • Dipartimento di Scienze Pediatriche G. De Toni, Università degli Studi di Genova and Istituto di Ricovero e Cura a Carattere Scientifico G. Gaslini, Genova, Italy

References 

  1. Dalkey NC, Brown BB, Cochran S. The Delphi method an experimental study of group opinion. [microform:] Santa Monica: Calif: Rand Corp; 1969;
  2. Delbecq AL. “Matrix organization”; a conceptual guide to organizational variation. Madison: University of Wisconsin, Bureau of Business Research and Service; 1969;
  3. Bombardier C, Gladmann DD, Urowitz MB. Committee on prognosis studies in systemic lupus erythematosus. The derivation of the SLEDAI: a disease activity index for lupus patients. Arthritis Rheum. 1992;35:630–640
  4. Giannini EH, Ruperto N, Ravelli A, et al. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum. 1997 Jul;40(7):1202–1209
  5. Rider LG, Giannini EH, Brunner HI, et al. International consensus on preliminary definitions of improvement in adult and juvenile myositis. Arthritis Rheum. 2004 Jul;50(7):2281–2290
  6. Wallace CA, Ruperto N, Giannini E. Preliminary criteria for clinical remission for select categories of juvenile idiopathic arthritis. J Rheumatol. 2004 Nov;31(11):2290–2294
  7. Tafeit E, Moller R, Sudi K, Reibnegger G. ROC and CART analysis of subcutaneous adipose tissue topography (SAT-Top) in type-2 diabetic women and healthy females. Am J Human Biol. 2000 May;12(3):388–394
  8. Lai JS, Cook K, Stone A, et al. Classical test theory and item response theory/Rasch model to assess differences between patient-reported fatigue using 7-day and 4-week recall periods. J Clin Epidemiol. 2009 Feb 10;
  9. Reise SP, Waller NG. Item response theory and clinical measurement. Annu Rev Clin Psychol. 2008 Oct 31;
  10. Safrit MJ, Cohen AS, Costa MG. Item response theory and the measurement of motor behavior. Res Q Exerc Sport. 1989 Dec;60(4):325–335
  11. Rasch G. An item analysis which takes individual differences into account. Br J Math Stat Psychol. 1966;19(1):49–57
  12. Brunner HI, Feldman BM, Urowitz MB, Gladman DD. Item weightings for the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Disease Damage Index using Rasch analysis do not lead to an important improvement. J Rheumatol. 2003 Feb;30(2):292–297
  13. Taylor WJ, McPherson KM. Using Rasch analysis to compare the psychometric properties of the short form 36 physical function score and the health assessment questionnaire disability index in patients with psoriatic arthritis and rheumatoid arthritis. Arthritis Rheum. 2007 Jun 15;57(5):723–729
  14. Wolfe F, Hawley DJ, Goldenberg DL, et al. The assessment of functional impairment in fibromyalgia (FM): Rasch analyses of 5 functional scales and the development of the FM health assessment questionnaire. J Rheumatol. 2000 Aug;27(8):1989–1999
  15. Wolfe F, Kong SX. Rasch analysis of the Western Ontario MacMaster questionnaire (WOMAC) in 2205 patients with osteoarthritis, rheumatoid arthritis, and fibromyalgia. Ann Rheum Dis. 1999;58(9):563–568
  16. Pouchot J, Ecosse E, Coste J, Guillemin F. Validity of the childhood health assessment questionnaire is independent of age in juvenile idiopathic arthritis. Arthritis Rheum. 2004 Aug 15;51(4):519–526
  17. Streiner DL, Norman GR. Health measurement scales: a practical guide to their development and use. 4th edn.. Oxford; New York: Oxford University Press; 2008;
  18. Singh JA, Solomon DH, Dougados M, et al. Development of classification and response criteria for rheumatic diseases. Arthritis Rheum. 2006 Jun 15;55(3):348–352
  19. Lawshe CH. A quantitative approach to content validity. Personnel Psychol. 1975;28(4):563–575
  20. Wallace CA, Ravelli A, Huang B, Giannini EH. Preliminary validation of clinical remission criteria using the OMERACT filter for select categories of juvenile idiopathic arthritis. J Rheumatol. 2006 Apr;33(4):789–795
  21. Brunner HI, Feldman BM, Bombardier C, Silverman ED. 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(7):1354–1360
  22. Hanley JA. Receiver operating characteristic (ROC) methodology: the state of the art. Crit Rev Diagn Imaging. 1989;29(3):307–335
  23. Hanley JA, Negassa A, Edwardes MD, Forrester JE. Statistical analysis of correlated data using generalized estimating equations: an orientation. Am J Epidemiol. 2003 Feb 15;157(4):364–375
  24. Liang MH, Socher SA, Larson MG, Schur PH. Reliability and validity of six systems for the clinical assessment of disease activity in systemic lupus erythematosus. Arthritis Rheum. 1989;32(9):1107–1118
  25. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989 Dec;10(4):407–415
  26. Beaton DE, Boers M, Wells GA. Many faces of the minimal clinically important difference (MCID): a literature review and directions for future research. Curr Opin Rheumatol. 2002 Mar;14(2):109–114
  27. Moretti C, Viola S, Pistorio A, et al. Relative responsiveness of condition specific and generic health status measures in juvenile idiopathic arthritis. Ann Rheum Dis. 2005 Feb;64(2):257–261
  28. Ruperto N, Ravelli A, Migliavacca D, et al. Responsiveness of clinical measures in children with oligoarticular juvenile chronic arthritis. J Rheumatol. 1999 Aug;26(8):1827–1830
  29. Brunner HI, Klein-Gitelman MS, Miller MJ, et al. Minimal clinically important differences of the childhood health assessment questionnaire. J Rheumatol. 2005 Jan;32(1):150–161
  30. Feldman BM, Ayling-Campos A, Luy L, et al. Measuring disability in juvenile dermatomyositis: validity of the childhood health assessment questionnaire. J Rheumatol. 1995 Feb;22(2):326–331
  31. Wells G, Beaton D, Shea B, et al. Minimal clinically important differences: review of methods. J Rheumatol. 2001 Feb;28(2):406–412
  32. Ruperto N, Ravelli A, Pistorio A, 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 Rheum. 2008;59:4–13
  33. Ruperto N, Ravelli A, Oliveira S, et al. The Pediatric Rheumatology International Trials Organization/American College of Rheumatology provisional criteria for the evaluation of response to therapy in juvenile systemic lupus erythematosus: prospective validation of the definition of improvement. Arthritis Rheum. 2006;55:355–363
  34. Ruperto N, Ravelli A, Cuttica R, 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;52:2854–2864
  35. Lovell DJ, Giannini EH, Reiff A, et al. Etanercept in children with polyarticular juvenile rheumatoid arthritis. N Engl J Med. 2000;342:763–769
  36. Ruperto N, Lovell DJ, Woo P, et al. A randomized trial of infliximab plus methotrexate for the treatment of polyarticular course juvenile rheumatoid arthritis. Arthritis Rheum. 2007;56:3096–3106
  37. Yokota S, Miyamae T, Imagawa T, et al. Therapeutic efficacy of humanized recombinant anti-interleukin-6 receptor antibody in children with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum. 2005;52:818–825
  38. 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;372:383–391
  39. Lovell DJ, Ruperto N, Goodman S, et al. Adalimumab with or without methotrexate in juvenile rheumatoid arthritis. N Engl J Med. 2008;359:810–820
  40. van der Heijde DMFM, van't Hof M, van Riel PLCM, et al. Judging disease activity in clinical practice in rheumatoid arthritis: first step in the development of a disease activity score. Ann Rheum Dis. 1990;49:916–920
  41. Prevoo ML, van't Hof MA, Kuper HH, et al. Modified disease activity scores that include twenty-eight-joint counts. Development and validation in a prospective longitudinal study of patients with rheumatoid arthritis. Arthritis Rheum. 1995;38:44–48
  42. Smolen JS, Breedveld FC, Schiff MH, et al. A simplified disease activity index for rheumatoid arthritis for use in clinical practice. Rheumatology. 2003;42:244–257
  43. Aletaha D, Nell VP, Stamm T, et al. Acute phase reactants add little to composite disease activity indices for rheumatoid arthritis: validation of a clinical activity score. Arthritis Res Ther. 2005;7:R796–R806
  44. Consolaro A, Ruperto N, Bazso A, et al. Development and validation of a composite disease activity score for juvenile idiopathic arthritis. Arthritis Rheum. 2009;61:658–666
  45. Bazso A, Consolaro A, Ruperto N, et al. Development and testing of reduced joint counts in juvenile idiopathic arthritis. J Rheumatol. 2009;36:183–190
  46. Lam GKW, Petri M. Assessment in systemic lupus erythematosus. Clin Exp Rheumatol. 2005;23(Suppl. 39):S120–S132
  47. Isenberg D, Ramsey-Goldman R. Assessing patients with lupus: towards a drug responder index. Rheumatology. 1999;38:1045–1049
  48. Bode RK, Klein-Gitelman MS, Miller ML, et al. Disease activity score for children with juvenile dermatomyositis: reliability and validity evidence. Arthritis Rheum. 2003 Feb 15;49(1):7–15
  49. Rider LG, Giannini EH, Harris-Love M, et al. International Myositis Assessment and Clinical Studies Group. Defining Clinical Improvement in Adult and Juvenile Myositis. J Rheumatol 2003 Mar; 30(3):603–17
  50. Pilkington C. Clinical assessment in juvenile idiopathic inflammatory myopathies and the development of disease activity and damage tools. Curr Opin Rheumatol. 2004;16:673–677
  51. Wells GA, Boers M, Shea B, et al. Minimal disease activity for rheumatoid arthritis: a preliminary definition. J Rheumatol. 2005;32:2016–2024
  52. Magni-Manzoni S, Ruperto N, Pistorio A, et al. Development and validation of a preliminary definition of minimal disease activity in patients with juvenile idiopathic arthritis. Arthritis Rheum. 2008;59:1120–1127
  53. Consolaro A, Vitale R, Pistorio A, et al. Physicians' and parents' ratings of inactive disease are frequently discordant in juvenile idiopathic arthritis. J Rheumatol. 2007;34:1773–1776
  54. Tubach F, Ravaud P, Beaton D, et al. Minimal clinically important improvement and patient acceptable symptom state for subjective outcome measures in rheumatic disorders. J Rheumatol. 2007;34:1188–1193
  55. Filocamo G, Schiappapietra B, Consolaro A, et al. Development and initial validation of the parent acceptable symptom state in juvenile idiopathic arthritis (JIA). Arthritis Rheum. 2008;58(Suppl.):S714–S715
  56. Singh G, Athreya BH, Fries JF, Goldsmith DP. Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis Rheum. 1994;37:1761–1769
  57. Lam C, Young N, Marwaha J, et al. Revised versions of the Childhood Health Assessment Questionnaire (CHAQ) are more sensitive and suffer less from a ceiling effect. Arthritis Rheum. 2004;51:881–889
  58. Filocamo G, Sztajnbok F, Cespedes-Cruz A, et al. Development and validation of a new short and simple measure of physical function for juvenile idiopathic arthritis. Arthritis Rheum. 2007;57:913–920
  59. Iglesias MJ, Cuttica RJ, Herrera Calvo M, et al. Design and validation of a new scale to assess the functional ability in children with juvenile idiopathic arthritis (JIA). Clin Exp Rheumatol. 2006;24:713–718
  60. Lovell DJ, Lindsley CB, Rennebohm RM, 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;42:2213–2219
  61. Huber AM, Feldman BM, Rennebohm RM, et al. Validation and clinical significance of the childhood myositis assessment scale for assessment of muscle function in the juvenile idiopathic inflammatory myopathies. Arthritis Rheum. 2004;50:1595–1603
  62. Huber AM, Hicks JE, Lachenbruch PA, et al. Validation of the childhood health assessment questionnaire in the juvenile idiopathic myopathies. Juvenile Dermatomyositis Disease Activity Collaborative Study Group. J Rheumatol. 2001;28:1106–1111
  63. Meiorin S, Pistorio A, Ravelli A, et al. Validation of the childhood health assessment questionnaire in active juvenile systemic lupus erythematosus. Arthritis Rheum. 2008;59:1112–1119
  64. Viola S, Felici E, Magni-Manzoni S, et al. Development and validation of a clinical index for assessment of long-term damage in juvenile idiopathic arthritis. Arthritis Rheum. 2005;52:2092–2102
  65. Gladman DD, Ginzler E, Goldsmith CH, 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;39:363–369
  66. Gladman DD, Urowitz MB, Goldsmith CH, 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;40:809–813
  67. Gutiérrez-Suárez R, Ruperto N, Gastaldi 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;54:2989–2996
  68. Miller FW, Rider LG, Chung YL, et al. Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies. Rheumatology. 2001;40:1262–1273
  69. Isenberg DA, Allen E, Farewell V, et al. International consensus outcome measures for patients with idiopathic inflammatory myopathies. Development and initial validation of myositis activity and damage indices in patients with adult onset disease. Rheumatology. 2004;43(1):49–54
  70. Landgraf JM, Abetz L, Ware JEJ. The CHQ user's manual. 1st edn.. Boston, MA: The Health Institute, New England Medical Center; 1996;
  71. Duffy CM, Arsenault L, Duffy KN, et al. The juvenile arthritis quality of life questionnaire-development of a new responsive index for juvenile rheumatoid arthritis and juvenile spondyloarthritides. J Rheumatol. 1997;24:738–746
  72. Varni JW, Seid M, Smith Knight T, et al. The PedsQL in pediatric rheumatology: reliability, validity, and responsiveness of the pediatric quality of life inventory generic core scales and rheumatology module. Arthritis Rheum. 2002;46:714–725
  73. Moorthy LN, Peterson MG, Baratelli M, et al. Multicenter validation of a new quality of life measure in pediatric lupus. Arthritis Rheum. 2007;57:1165–1173
  74. Ravelli A. The time has come to include assessment of radiographic progression in juvenile idiopathic arthritis clinical trials. J Rheumatol. 2008;35:553–557
  75. Poznanski AK, Hernandez RJ, Guire KE, et al. Carpal length in children – A useful measurement in the diagnosis of rheumatoid arthritis and some congenital malformation syndromes. Radiology. 1978;129:661–668
  76. van Rossum MA, Zwinderman AH, Boers M, et al. Radiologic features in juvenile idiopathic arthritis: a first step in the development of a standardized assessment method. Arthritis Rheum. 2003;48:507–515
  77. van Rossum MA, Boers M, Zwinderman AH, et al. Development of a standardized method of assessment of radiographs and radiographic change in juvenile idiopathic arthritis: introduction of the Dijkstra composite score. Arthritis Rheum. 2005;52:2865–2872
  78. Ravelli A, Ioseliani M, Norambuena X, et al. Adapted versions of the Sharp-van der Heijde scoring method are reliable and valid for the assessment of radiographic progression in juvenile idiopathic arthritis. Arthritis Rheum. 2007;56:3087–3095
  79. Graham TB. Imaging in juvenile arthritis. Curr Opin Rheumatol. 2005;17:574–578
  80. Gardner-Medwin JM, Killeen OG, Ryder CA, et al. Magnetic resonance imaging identifies features in clinically unaffected knees predicting extension of arthritis in children with monoarthritis. J Rheumatol. 2006;33:2337–2343
  81. Malattia C, Damasio MB, Magnaguagno F, et al. Magnetic resonance imaging, ultrasonography, and conventional radiography in the assessment of bone erosions in juvenile idiopathic arthritis. Arthritis Rheum. 2008;59:1764–1772
  82. Weiss P, Arabshahi B, Johnson A, et al. High prevalence of temporomandibular joint arthritis at disease onset in children with juvenile idiopathic arthritis, as detected by magnetic resonance imaging but not by ultrasound. Arthritis Rheum. 2008;58:1189–1196
  83. Magni-Manzoni S, Epis O, Ravelli A, et al. A comparison of clinical versus ultrasound determined synovitis in juvenile idiopathic arthritis. Manuscript submitted.
  84. Burns J, Wright S, McCallister C, Rooney M. The correlation between clinical and ultrasonographic findings of ankle disease in JIA. Arthritis Rheum. 2008;58(Suppl.):S716
  85. McCarron M, Wray M, Pascoli L, et al. Knee disease in juvenile idiopathic arthritis: correlation between clinical and ultrasonographic findings. Arthritis Rheum. 2008;58(Suppl.):S717

 Dr. Brunner is supported by a grant funded NIAMS P60 AR047784; Methodology Core.

PII: S1521-6942(09)00073-4

doi: 10.1016/j.berh.2009.07.001

Best Practice & Research Clinical Rheumatology
Volume 23, Issue 5 , Pages 609-624 , October 2009