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. 2000 Dec;59(12):966–974. doi: 10.1136/ard.59.12.966

Responsiveness of the core set, response criteria, and utilities in early rheumatoid arthritis

A Verhoeven 1, M Boers 1, S van der Linden 1
PMCID: PMC1753042  PMID: 11087700

Abstract

OBJECTIVE—Validation of responsiveness and discriminative power of the World Health Organisation/International League of Associations for Rheumatology (WHO/ILAR) core set, the American College of Rheumatology (ACR), and European League for Rheumatology (EULAR) criteria for improvement/response, and other single and combined measures (indices) in a trial in patients with early rheumatoid arthritis (RA).
METHODS—Ranking of measures by response (standardised response means and effect sizes) and between-group discrimination (unpaired t test and χ2 values) at two time points in the COBRA study. This study included 155 patients with early RA randomly allocated to two treatment groups with distinct levels of expected response: combined treatment, high response; sulfasalazine treatment, moderate response.
RESULTS—At week 16, standardised response means of core set measures ranged between 0.8 and 3.5 for combined treatment and between 0.4 and 1.2 for sulfasalazine treatment (95% confidence interval ±0.25). Performance of patient oriented measures (for example, pain, global assessment) was best when the questions were focused on the disease. The most responsive single measure was the patient's assessment of change in disease activity, at 3.5. Patient utility, a generic health status measure, was moderately (rating scale) to poorly (standard gamble) responsive. Response means of most indices (combined measures) exceeded 2.0, the simple count of core set measures improved by 20% was most responsive at 4.1. Discrimination performance yielded similar but not identical results: best discrimination between treatment groups was achieved by the EULAR response and ACR improvement criteria (at 20% and other percentage levels), the pooled index, and the disease activity score (DAS), but also by the Health Assessment Questionnaire (HAQ) and grip strength.
CONCLUSIONS—Responsiveness and discrimination between levels of response are not identical concepts, and need separate study. The WHO/ILAR core set comprises responsive measures that discriminate well between different levels of response in early RA. However, the performance of patient oriented measures is highly dependent on their format. The excellent performance of indices such as the ACR improvement and EULAR response criteria confirms that they are the preferred primary end point in RA clinical trials.



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Figure 1  .

Figure 1  

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Comparison of responsiveness and discrimination performance of end points. The order of the measures evaluated corresponds with that of table 3. Dark bars: standardised response means of the combination group at 16 (black) and 28 weeks (dark grey). Light bars: standardised response means of the sulfasalazine group at 16 (light grey) and 28 weeks (white). Dots: unpaired t values of the between-group comparison at 16 (black) and 28 weeks (white). pL = points Likert; MACTAR = McMaster Toronto Arthritis patient preference questionnaire; DAS = disease activity score; AIMS = Arthritis Impact Measurement Scale; HAQ = Health Assessment Questionnaire; VAS = visual analogue scale; ESR = erythrocyte sedimentation rate; RS = rating scale; CRP = C reactive protein; SG = standard gamble.

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Selected References

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