The ACR20 and defining a threshold for response in rheumatic diseases: too much of a good thing

Arthritis Res Ther. 2014 Jan 3;16(1):101. doi: 10.1186/ar4428.

Abstract

In the past 20 years great progress has been made in the development of multidimensional outcome measures (such as the Disease Activity Score and ACR20) to evaluate treatments in rheumatoid arthritis, a process disseminated throughout rheumatic diseases. These outcome measures have standardized the assessment of outcomes in trials, making it possible to evaluate and compare the efficacy of treatments. The methodologic advances have included the selection of pre-existing outcome measures that detected change in a sensitive fashion (in rheumatoid arthritis, this was the Core Set Measures). These measures were then combined into a single multidimensional outcome measure and such outcome measures have been widely adopted in trials and endorsed by the American College of Rheumatology (ACR) and the European League Against Rheumatism (EULAR) and regulatory agencies. The secular improvement in treatment for patients with rheumatoid arthritis has been facilitated in part by these major methodologic advancements. The one element of this effort that has not optimized measurement of outcomes nor made it easier to detect the effect of treatments is the dichotomization of continuous measures of response, creating responders and non-responder definitions (for example, ACR20 responders; EULAR good responders). Dichotomizing response sacrifices statistical power and eliminates variability in response. Future methodologic work will need to focus on improving multidimensional outcome measurement without arbitrarily characterizing some patients as responders while labeling others as non-responders.

MeSH terms

  • Antirheumatic Agents / therapeutic use
  • Humans
  • Outcome Assessment, Health Care / standards*
  • Rheumatic Diseases / therapy*
  • Rheumatology / methods
  • Rheumatology / standards*
  • Severity of Illness Index*
  • Treatment Outcome

Substances

  • Antirheumatic Agents