Background/Purpose: To date, there are no approved treatments for cutaneous lupus erythematosus (CLE), a disease known to significantly burden a patient’s quality of life (QoL). Clinical trials are important for the advancement of new treatments for CLE and outcome measures should reflect clinically meaningful improvement in disease activity and its effect on QoL. Currently, trials use an efficacy measure of ≥50% improvement in disease activity, defined by the Cutaneous Lupus Disease Area and Severity Index score for activity (CLASI-A), in patients with an initial CLASI-A of ≥10. However, the degree of improvement in disease activity needed to predict a meaningful impact on QoL, an important variable in the design and interpretation of future clinical trials, has not been defined.

Methods:  This study included 126 patients seen at the Hospital of the University of Pennsylvania who participate in a longitudinal research database. Patients with mild, moderate or severe initial CLASI-A were analyzed separately and using a linear regression model, we calculated the percent change and difference needed in CLASI-A to have an important impact on QoL, defined as a 9.38-point and a 7.37-point improvement in the Emotions and Symptoms subscales of Skindex-29, respectively.

Results: In patients with an initial CLASI-A ≥8, a decrease by 42.1% or 7-points and a decrease by 31.0% or 5-points in disease activity is associated with a meaningful impact in the Emotions and the Symptoms subscales, respectively. For both subscales, patients with increasingly severe initial disease required a smaller percent change in CLASI-A to predict a meaningful change in QoL.

Conclusion: We find that using a CLASI-A ≥8 for trial entry allows for the inclusion of patients with milder disease for whom improvement of CLASI-A by ≥50% results in a meaningful impact on QoL, as determined by the Emotions and Symptoms subscales of Skindex-29. In patients with CLASI-A ≥8, a decrease in activity by seven and five-points is not only a clinically significant improvement but also indicates a meaningful impact on the Emotions and Symptoms subscales, respectively. For trials enrolling a larger proportion of patients with severe disease activity, or CLASI-A ≥20, we recommend stratifying patients by disease severity, as a smaller magnitude of percent change in disease activity predicts meaningful improvement in QoL. Our findings establish appropriate trial endpoints by determining clinically significant change in disease activity associated with meaningful changes in patients’ QoL.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/assessing-meaningful-changes-in-disease-activity-as-clinical-trial-efficacy-measures-for-cutaneous-lupus-erythematosus/