Figure 1: Cumulative probability plots of individual 1-year radiographic progression scores in 135 rheumatoid arthritis patients who were participating in the Combinatietherapie Bij Reumatoide Artritis (COBRA) trial (67 patients in the monotherapy group (circles) and 68 patients in the combination therapy group (triangles)). Reprinted from [14] with permission of John Wiley & Sons, Inc.

Mentions: Organ damage and death may be considered as 'hard' outcome measures in rheumatology. Progression of joint damage is a well-established outcome for trials in inflammatory joint diseases. In RA, measurement of joint damage of the hands and feet was deemed appropriate if trials last 12 months or longer, but with the current effective medication it may also be appropriate to measure joint damage after just 3 or 6 months. The eventual progression of joint damage can be rated by applying a standard scoring system, such as the Sharp-vanderHeijde score applied to plain X-rays of the hands and feet in RA [13]. The outcome measure can be the progression of raw scores or the number of patients that progress more than the smallest detectable change - for example, all patients that progress more than 6 points (Figure 1) [14]. Using the proportion of patients with progression of joint damage in two (treatment) groups favors the calculation of relative risk as an effect measure. Relative risks are easy to combine in meta-analyses.

Fransen J, van Riel PL - Arthritis Res. Ther. (2009)

Bottom Line: Inflammatory rheumatic diseases are generally multifaceted disorders and, therefore, measurement of multiple outcomes is relevant to most of these diseases.Developments in outcome measures in the rheumatic diseases are promoted by the development of successful treatments.Outcome measurement will increasingly deal with measurement of low levels of disease activity and avoidance of disease consequences.

Affiliation: Radboud University Nijmegen Medical Centre, Department of Rheumatology, 6500HB Nijmegen, The Netherlands. j.fransen@reuma.umcn.nl

Abstract: Inflammatory rheumatic diseases are generally multifaceted disorders and, therefore, measurement of multiple outcomes is relevant to most of these diseases. Developments in outcome measures in the rheumatic diseases are promoted by the development of successful treatments. Outcome measurement will increasingly deal with measurement of low levels of disease activity and avoidance of disease consequences. It is an advantage for patient management and knowledge transfer if the same outcomes are used in practice and in trials. Continuous measures of change are generally the most powerful and, therefore, are preferred as primary outcomes in trials. For daily clinical practice, outcome measures should reflect the patients' state and have to be easily derivable. The objective of this review is to describe recent developments in outcome measures for inflammatory rheumatic diseases for trials and clinical practice, with an emphasis on rheumatoid arthritis.