title: Assessing disease progression using a composite endpoint.
abstract: Scleroderma patients usually have serious medical events in several organ systems and it is desirable to have a composite index that accounts for disease activity in these organ systems. We show how one may use a composite 'time to event' analysis for evaluating such patients and more generally for patients suffering from a chronic disease. The composite 'time to event' analysis requires a composite endpoint with a Kaplan-Meier type analysis. As an illustration, we use data from a clinical trial for scleroderma patients and present sensitivity analysis where one or more of the organ involvement definition criteria are modified. In addition, we propose desirability functions to monitor patients' disease improvement when the outcomes are all continuous. This method offers several possible advantages over existing methods for measuring patients' improvement.
authors.affiliation: Department of Biostatistics, University of California at Los Angeles, CA, USA. wkwong@ucla.edu
authors.forename: Weng Kee
authors.initials: WK
authors.lastname: Wong
authors.forename: Daniel E
authors.initials: DE
authors.lastname: Furst
authors.forename: Philip J
authors.initials: PJ
authors.lastname: Clements
authors.forename: Jim B
authors.initials: JB
authors.lastname: Streisand
mesh_terms: D018501:Antirheumatic Agents; D003627:Data Interpretation, Statistical; D018450:Disease Progression; D023381:Endpoint Determination / Q000379:methods / Q000706:statistics & numerical data*; D006801:Humans; D053208:Kaplan-Meier Estimate; D017063:Outcome Assessment, Health Care; D010396:Penicillamine; D016032:Randomized Controlled Trials as Topic; D018401:Sample Size; D045743:Scleroderma, Diffuse / Q000188:drug therapy / Q000503:physiopathology*; D012680:Sensitivity and Specificity; D014481:United States
chemical_list: D018501:Antirheumatic Agents; D010396:Penicillamine