Developing international consensus on measures of improvement for patients with myositis

Abstract

We discuss methods of developing consensus in measuring improvement in myositis. We consider selecting candidate variables, reliability and validity, percentage improvement/worsening rules, rules based on CART and logistic regression. We discuss criteria for determining an acceptable rule that include both numerical measures and physician acceptance.

Get full access to this article

View all access options for this article.

References

Breiman L. , Friedman JH , Olshen RA , Stone CJ Classification and regression trees. Wadsworth International Group, 1984.

Dunham RB 1998. Nominal Group Technique: A User's Guide Available on internet at http://instruction.bus.wisc.edu/obdemo/readings/ngt.html or via Google.

FDA Guidance for Industry: Patient Reported Outcomes: Use in Medial Product Development to Support Claims in Approved Labels or Advertising. March 2004 (website: www.fda.gov/cder/regulatory/default.htm )

Felson DT , Anderson JJ , Boers M. , Bombardier C. , Chernoff M. , Fried B. , Furst D. , Goldsmith C. , Kieszak S. , Lightfoot R. , Paulus H. , Tugwell P. , Weinblatt M. , Widmark R. , Williams HJ , Wolfe, F. The American College of Rheumatology preliminary core set of disease activity measures for rheumatoid arthritis clinical trials. The Committee on Outcome Measures in Rheumatoid Arthritis Clinical Trials. Arthritis Rheum 1993; 36: 729—40.

Felson DT , Anderson JJ , Boers M. , Bombardier C. , Chernoff M. , Fried B. , Furst D. , Goldsmith C. , Katz LM , Lightfoot, Jr. R. , Paulus H. , Strand V. , Tugwell P. , Weinblatt M. , Williams HJ , Wolfe F. , Kieszak S. American College of Rheumatology Preliminary definition of improvement in rheumatoid arthritis. Arthritis Rheum 1995 ; 38: 727—35.

Isenberg DA , Allen E. , Farewell V. , Ehrenstein MR , Hanna MG , Lundberg IE , Oddis C. , Pilkington C. , Plotz P. , Scott D. , Vencovsky J. , Cooper R. , Rider L. , Miller F. 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: 49—54.

Miller FW , Rider LG , Chung YL , Cooper R. , Danko K. , Farewell V. , Lundberg I. , Morrison C. , Oakley L. , Oakley I. , Pilkington C. , Vencovsky J. , Vincent K. , Scott DL , Isenberg DA Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies. Rheumatology 2001; 40: 1262—73.

Miller FW , Leitman SF , Cronin ME , Hicks JE , Leff RL , Wesley R. , Fraser DD , Dalakas M. , Plotz PH Controlled trial of plasma exchange and leukapheresis in polymyositis and dermatomyositis. N Engl J Med 1992; 326(21): 1380—4.

Rider LG , Miller FW Idiopathic inflammatory muscle disease: clinical aspects. Bailliere's Clin Rheumatol 2000; 14: 37—54.

10.

Rider LG , Giannini EH , Harris-Love M. , Joe G. , Isenberg D. , Pilkington C. , Lachenbruch PA , Miller FW Defining clinical improvement in adult and juvenile myositis. J Rheumatol 2003; 30: 603—17.

11.

Rider LG Outcome assessment in the adult and juvenile idiopathic inflammatory myopathies . Rheum Dis Clin North Am 2002; 28: 935—77.

12.

Rider LG , Giannini EH , Brunner HI , Ruperto N. , James-Newton L. , Reed AM , Lachenbruch PA , Miller FW , for the International Myositis Assessment and Clinical Studies Group (IMACS) 6. International Consensus on Outcome Measures for Patients with Idiopathic Inflammatory Myopathies. Preliminary Definitions of Improvement for Adult and Juvenile Myositis. Arthritis Rheum 2004; 50: 2281—90.