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Relative Responsiveness of Physician/Assessor-Derived and Patient-Derived Core Set Measures in Rheumatoid Arthritis Trials TUHINA NEOGI, HUI XIE, and DAVID T. FELSON
ABSTRACT. Methods. We used data from 9 RA trials [anti-tumor necrosis factor-a (TNF-a) and disease modifying antirheumatic drug (DMARD)] in which CSM had been assessed, conducted from the early 1990s to present, with a total of 2969 patients. We grouped the CSM as CPD (pain, patient global assessment, function) and CMD [tender joint count (TJC), swollen joint count (SJC), physician global, inflammatory marker]. Using bootstrap simulation, we estimated the sample size that would be required to distinguish active treatment from placebo with the Wilcoxon rank-sum test in the clinical trials for the outcomes of percentage change of each individual CSM, of the Disease Activity Score (DAS), and average percentage change of the CMD or of the CPD. Results. Comparing the performance of individual CSM relative to one another, the physician and patient global assessments and TJC would require the lowest sample sizes to distinguish active treatment from placebo, while use of the SJC, inflammatory marker, and function would require the highest. The CMD performed similarly to the DAS, requiring similar sample sizes, while the CPD would require 1.7 times greater sample size to distinguish treatment from placebo. The results were similar across DMARD and anti-TNF-a trials. Conclusion. Because of their demonstrated sensitivity to change, composite measures assessing RA outcomes in clinical trials should continue to include physician/assessor-derived core set measure assessments. (First Release April 15 2008; J Rheumatol 2008;35:757-62) Key Indexing Terms:
CLINICAL TRIALS From the Clinical Epidemiology Research and Training Unit, Boston University School of Medicine, Boston, Massachusetts; and the Department of Biostatistics, University of Illinois at Chicago, School of Public Health, Chicago, Illinois, USA. Supported by NIH AR47785 and a grant to reevaluate response criteria from the American College of Rheumatology. Dr. Neogi was supported by the Abbott Scholar Award in Rheumatology and the Arthritis Foundation Postdoctoral Fellowship Award. She is currently supported by the ACR-REF ASP Junior Career Development Award in Geriatric Medicine, Arthritis Foundation Arthritis Investigator Award, and NIH 1K23AR055127. T. Neogi, MD, FRCPC, Clinical Epidemiology Research and Training Unit, Boston University School of Medicine; H. Xie, PhD, Department of Biostatistics, University of Illinois at Chicago, School of Public Health; D.T. Felson, MD, MPH, Clinical Epidemiology Research and Training Unit, Boston University School of Medicine, Boston. Address reprint requests to Dr. T. Neogi, Boston University School of Medicine, Clinical Epidemiology Unit, Suite X-200, 650 Albany Street, Boston, MA 02118. Accepted for publication December 27, 2007. |
