The American College of Rheumatology Provisional Composite Response Index for Clinical Trials in Early Diffuse Cutaneous Systemic Sclerosis

Arthritis and Rheumatology

Volumn 68, Issue 2, 2016, Pages 299-311

  Khanna, Dinesh ^a   Berrocal, Veronica J ^a   Giannini, Edward H ^b   Seibold, James R ^c   Merkel, Peter A ^d   Mayes, Maureen D ^e   Baron, Murray ^f   Clements, Philip J ^g   Steen, Virginia ^h   Assassi, Shervin ^e   Schiopu, Elena ^a   Phillips, Kristine ^a   Simms, Robert W ⁱ   Allanore, Yannick ^h   Denton, Christopher P ^j   Distler, Oliver ^k   Johnson, Sindhu R ^l   Matucci Cerinic, Marco ^m   Pope, Janet E ⁿ   Proudman, Susanna M ^o   Siegel, Jeffrey ^p   Wong, Weng Kee ^g   Wells, Athol U ^q   Furst, Daniel E ^g   more..

^a UNIVERSITY OF MICHIGAN HEALTH SYSTEM   (United States)

^b CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^c Scleroderma Research Consultants   (United States)

^d UNIVERSITY OF PENNSYLVANIA   (United States)

^e UNIVERSITY OF TEXAS   (United States)

^f MCGILL UNIVERSITY   (Canada)

^g UNIVERSITY OF CALIFORNIA   (United States)

^h UNIVERSITÉ PARIS DESCARTES   (France)

ⁱ BOSTON UNIVERSITY   (United States)

^j ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

^k UNIVERSITY HOSPITAL ZURICH   (Switzerland)

^l UNIVERSITY OF TORONTO   (Canada)

^m CAREGGI UNIVERSITY HOSPITAL   (Italy)

ⁿ WESTERN UNIVERSITY   (Canada)

^o UNIVERSITY OF ADELAIDE   (Australia)

^p GENENTECH INC   (United States)

^q ROYAL BROMPTON HOSPITAL   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

METHOTREXATE; ANTIRHEUMATIC AGENT;

ADULT; ARTICLE; CLINICAL ASSESSMENT TOOL; COMPOSITE RESPONSE INDEX IN SYSTEMIC SCLEROSIS; CONSENSUS; CONTROLLED STUDY; DIFFUSE CUTANEOUS SYSTEMIC SCLEROSIS; FACE VALIDITY; FEMALE; FOLLOW UP; FORCED VITAL CAPACITY; HEALTH ASSESSMENT QUESTIONNAIRE; HUMAN; MAJOR CLINICAL STUDY; MALE; PATIENT CODING; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SCLERODERMA; SKINFOLD THICKNESS; SYSTEMIC SCLEROSIS; COHORT ANALYSIS; CONSENSUS DEVELOPMENT; LONGITUDINAL STUDY; MEDICAL SOCIETY; MIDDLE AGED; OUTCOME ASSESSMENT; RANDOMIZED CONTROLLED TRIAL (TOPIC); REPRODUCIBILITY; RHEUMATOLOGY; SCLERODERMA, DIFFUSE; STATISTICAL MODEL;

ADULT; ANTIRHEUMATIC AGENTS; COHORT STUDIES; FEMALE; HUMANS; LOGISTIC MODELS; LONGITUDINAL STUDIES; MALE; METHOTREXATE; MIDDLE AGED; OUTCOME ASSESSMENT (HEALTH CARE); RANDOMIZED CONTROLLED TRIALS AS TOPIC; REPRODUCIBILITY OF RESULTS; RHEUMATOLOGY; SCLERODERMA, DIFFUSE; SOCIETIES, MEDICAL;

EID: 84956807922     PISSN: 23265191     EISSN: 23265205     Source Type: Journal    
DOI: 10.1002/art.39501     Document Type: Article

References (48)

1. Ioannidis JP, Vlachoyiannopoulos PG, Haidich AB, Medsger TA Jr, Lucas M, Michet CJ, et al., Mortality in systemic sclerosis: an international meta-analysis of individual patient data. Am J Med 2005; 118: 2-10.
2. Elhai M, Meune C, Avouac J, Kahan A, Allanore Y., Trends in mortality in patients with systemic sclerosis over 40 years: a systematic review and meta-analysis of cohort studies. Rheumatology (Oxford) 2012; 51: 1017-26.
3. Khanna D, Kowal-Bielecka O, Khanna PP, Lapinska A, Asch SM, Wenger N, et al., Quality indicator set for systemic sclerosis. Clin Exp Rheumatol 2011; 29 Suppl 65: 33-9.
4. Khanna D, Distler O, Avouac J, Behrens F, Clements PJ, Denton C, et al, for the Investigators in CRISS and EPOSS. Measures of response in clinical trials of systemic sclerosis: the Combined Response Index for Systemic Sclerosis (CRISS) and Outcome Measures in Pulmonary Arterial Hypertension related to Systemic Sclerosis (EPOSS). J Rheumatol 2009; 36: 2356-61.
5. Khanna D, Lovell DJ, Giannini E, Clements PJ, Merkel PA, Seibold JR, et al., Development of a provisional core set of response measures for clinical trials of systemic sclerosis. Ann Rheum Dis 2008; 67: 703-9.
6. Khanna D, Merkel PA., Outcome measures in systemic sclerosis: an update on instruments and current research. Curr Rheumatol Rep 2007; 9: 151-7.
7. Chung L, Denton CP, Distler O, Furst DE, Khanna D, Merkel PA., Clinical trial design in scleroderma: where are we and where do we go next? Clin Exp Rheumatol 2012; 30 Suppl 71: 97-102.
8. Abraham DJ, Varga J., Scleroderma: from cell and molecular mechanisms to disease models. Trends Immunol 2005; 26: 587-95.
9. Nagaraja V, Denton CP, Khanna D., Old medications and new targeted therapies in systemic sclerosis. Rheumatology (Oxford) 2015; 54: 1944-53.
10. Clements P, Lachenbruch P, Seibold J, White B, Weiner S, Martin R, et al., Inter and intraobserver variability of total skin thickness score (modified Rodnan TSS) in systemic sclerosis. J Rheumatol 1995; 22: 1281-5.
11. Van der Heijde DM, van 't Hof MA, van Riel PL, Theunisse LA, Lubberts EW, van Leeuwen MA, 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: 916-20.
12. Paulus HE, Egger MJ, Ward JR, Williams HJ, and the Cooperative Systematic Studies of Rheumatic Diseases Group. Analysis of improvement in individual rheumatoid arthritis patients treated with disease-modifying antirheumatic drugs, based on the findings in patients treated with placebo. Arthritis Rheum 1990; 33: 477-84.
13. Felson DT, Anderson JJ, Boers M, Bombardier C, Furst D, Goldsmith C, et al., American College of Rheumatology preliminary definition of improvement in rheumatoid arthritis. Arthritis Rheum 1995; 38: 727-35.
14. Nair R, Aggarwal R, Khanna D., Methods of formal consensus in classification/diagnostic criteria and guideline development. Semin Arthritis Rheum 2011; 41: 95-105.
15. Classification and Response Criteria Subcommittee of the American College of Rheumatology Committee on Quality Measures. Development of classification and response criteria for rheumatic diseases. Arthritis Rheum 2006; 55: 348-52.
16. Merkel PA, Silliman NP, Clements PJ, Denton CP, Furst DE, Mayes MD, et al, for the Scleroderma Clinical Trials Consortium. Patterns and predictors of change in outcome measures in clinical trials in scleroderma: an individual patient meta-analysis of 629 subjects with diffuse cutaneous systemic sclerosis. Arthritis Rheum 2012; 64: 3420-9.
17. Wiese AB, Berrocal VJ, Furst DE, Seibold JR, Merkel PA, Mayes MD, et al., Correlates and responsiveness to change of measures of skin and musculoskeletal disease in early diffuse systemic sclerosis. Arthritis Care Res (Hoboken) 2014; 66: 1731-9.
18. Hays RD, Hadorn D., Responsiveness to change: an aspect of validity, not a separate dimension. Qual Life Res 1992; 1: 73-5.
19. Cohen J., The analysis of variance and covariance. In: Statistical power analysis for the behavioral sciences. 2nd ed. Hillsdale (NJ): Lawrence Erlbaum Associates; 1988. p. 273-406.
20. Davies EH, Surtees R, DeVile C, Schoon I, Vellodi A., A severity scoring tool to assess the neurological features of neuronopathic Gaucher disease. J Inherit Metab Dis 2007; 30: 768-82.
21. Fan X, Pope J, the Canadian Scleroderma Research Group, Baron M., What is the relationship between disease activity, severity and damage in a large Canadian systemic sclerosis cohort? Results from the Canadian Scleroderma Research Group (CSRG). Rheumatol Int 2009; 30: 1205-10.
22. Bassel M, Hudson M, Taillefer SS, Schieir O, Baron M, Thombs BD., Frequency and impact of symptoms experienced by patients with systemic sclerosis: results from a Canadian National Survey. Rheumatology (Oxford) 2011; 50: 762-67.
23. Suarez-Almazor ME, Kallen MA, Roundtree AK, Mayes M., Disease and symptom burden in systemic sclerosis: a patient perspective. J Rheumatol 2007; 34: 1718-26.
24. Stamm TA, Mattsson M, Mihai C, Stocker J, Binder A, Bauernfeind B, et al., Concepts of functioning and health important to people with systemic sclerosis: a qualitative study in four European countries. Ann Rheum Dis 2011; 70: 1074-9.
25. Ware JE Jr, Snow KK, Kosinski M, Gandek B., SF-36 health survey: manual and interpretation guide. Boston: The Health Institute, New England Medical Center; 1993.
26. Gneiting T, Raftery A., Strictly proper scoring rules. J Am Stat Assoc 2007; 102: 359-78.
27. Nagelkerke NG., A note on a general definition of the coefficient of determination. Biometrika 1991; 78: 691-2.
28. Pope JE, Bellamy N, Seibold JR, Baron M, Ellman M, Carette S, et al., A randomized, controlled trial of methotrexate versus placebo in early diffuse scleroderma. Arthritis Rheum 2001; 44: 1351-8.
29. Furst D, Khanna D, Matucci-Cerinic M, Clements P, Steen V, Pope J, et al., Systemic sclerosis: continuing progress in developing clinical measures of response. J Rheumatol 2007; 34: 1194-200.
30. Fries JF, Spitz P, Kraines RG, Holman HR., Measurement of patient outcome in arthritis. Arthritis Rheum 1980; 23: 137-45.
31. Merkel PA, Silliman NP, Denton CP, Furst DE, Khanna D, Emery P, et al, for the CAT-192 Research Group and the Scleroderma Clinical Trials Consortium. Validity, reliability, and feasibility of durometer measurements of scleroderma skin disease in a multicenter treatment trial. Arthritis Rheum 2008; 59: 699-705.
32. ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med 2002; 166: 111-7.
33. Merkel PA, Herlyn K, Martin RW, Anderson JJ, Mayes MD, Bell P, et al, for the Scleroderma Clinical Trials Consortium. Measuring disease activity and functional status in patients with scleroderma and Raynaud's phenomenon. Arthritis Rheum 2002; 46: 2410-20.
34. Khanna D, Clements PJ, Furst DE, Korn JH, Ellman M, Rothfield N, et al, for the Relaxin Investigators and the Scleroderma Clinical Trials Consortium. Recombinant human relaxin in the treatment of systemic sclerosis with diffuse cutaneous involvement: a randomized, double-blind, placebo-controlled trial. Arthritis Rheum 2009; 60: 1102-11.
35. Tashkin DP, Elashoff R, Clements PJ, Goldin J, Roth MD, Furst DE, et al., Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med 2006; 354: 2655-66.
36. Merkel PA, Clements PJ, Reveille JD, Suarez-Almazor ME, Valentini G, Furst DE., Current status of outcome measure development for clinical trials in systemic sclerosis: report from OMERACT 6. J Rheumatol 2003; 30: 1630-47.
37. Clements PJ, Hurwitz EL, Wong WK, Seibold JR, Mayes M, White B, et al., Skin thickness score as a predictor and correlate of outcome in systemic sclerosis: high-dose versus low-dose penicillamine trial. Arthritis Rheum 2000; 43: 2445-54.
38. Steen VD, Medsger TA Jr., Severe organ involvement in systemic sclerosis with diffuse scleroderma. Arthritis Rheum 2000; 43: 2437-44.
39. Amjadi S, Maranian P, Furst DE, Clements PJ, Wong WK, Postlethwaite AE, et al, for the Investigators of the D-Penicillamine, Human Recombinant Relaxin, and Oral Bovine Type I Collagen Clinical Trials. Course of the modified Rodnan skin thickness score in systemic sclerosis clinical trials: analysis of three large multicenter, double-blind, randomized controlled trials. Arthritis Rheum 2009; 60: 2490-8.
40. Van der Heijde DM, van 't Hof MA, van Riel PL, van Leeuwen MA, van Rijswijk MH, van de Putte LB., Validity of single variables and composite indices for measuring disease activity in rheumatoid arthritis. Ann Rheum Dis 1992; 51: 177-81.
41. Luijten KM, Tekstra J, Bijlsma JW, Bijl M., The Systemic Lupus Erythematosus Responder Index (SRI); a new SLE disease activity assessment. Autoimmun Rev 2012; 11: 326-9.
42. Symmons DP, Coppock JS, Bacon PA, Bresnihan B, Isenberg DA, Maddison P, et al, and Members of the British Isles Lupus Assessment Group (BILAG). Development and assessment of a computerized index of clinical disease activity in systemic lupus erythematosus. Q J Med 1988; 69: 927-37.
43. Bombardier C, Gladman DD, Urowitz MB, Caron D, Chang DH, and the Committee on Prognosis Studies in SLE. Derivation of the SLEDAI: a disease activity index for lupus patients. Arthritis Rheum 1992; 35: 630-40.
44. Valentini G, D'Angelo S, Della RA, Bencivelli W, Bombardieri S., European Scleroderma Study Group to define disease activity criteria for systemic sclerosis. IV. Assessment of skin thickening by modified Rodnan skin score. Ann Rheum Dis 2003; 62: 904-5.
45. Medsger TA Jr, Silman AJ, Steen VD, Black CM, Akesson A, Bacon PA, et al., A disease severity scale for systemic sclerosis: development and testing. J Rheumatol 1999; 26: 2159-67.
46. 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.
47. Steen VD, Mayes MD, Merkel PA., Assessment of kidney involvement. Clin Exp Rheumatol 2003; 21 Suppl 29: 29-31.
48. Hoeper MM, Bogaard HJ, Condliffe R, Frantz R, Khanna D, Kurzyna M, et al., Definitions and diagnosis of pulmonary hypertension. J Am Coll Cardiol 2013; 62: 42-50.