Working Core Outcome Set Aimed at Improving Cutaneous Lupus Erythematosus Clinical Trials

A newly developed working core outcome set to improve cutaneous lupus erythematosus (CLE) clinical trials is the starting point for further refinement and evaluation of outcome measure quality, according to a study published in Lupus Science and Medicine.

Previous CLE clinical trials have used a variety of outcome measures with little continuity, which has led to confusion in interpreting results and comparing efficacy across studies. The lack of validated CLE outcome measures has delayed the development of new medications.

In the current study, researchers sought to establish a preliminary set of core outcomes for use in upcoming CLE trials.

The researchers conducted an extensive literature search of online databases and ClinicalTrials.gov (including randomized control trials involving CLE and systemic lupus erythematosus [SLE]), and a scoping review of qualitative studies to identify potential domains and outcome measures. A qualitative synthesis by a steering committee classified domains into core, import but optional, and research agenda categories. Instruments for each core domain were identified from the literature and appraised using consensus-based standards for the selection of health measurement instruments (COSMIN) methodology.

Six core domains were proposed including skin-specific disease activity, investigator global assessment of disease activity, skin-specific disease damage, symptoms (itch, pain, and photosensitivity), health-related quality of life, and patient global assessment of disease activity.

The researchers recommended the Cutaneous Lupus Erythematosus Disease Area and Severity Index-Activity (CLASI-A) and CLASI-Damage (CLASI-D) for skin-specific disease activity and damage domains, respectively, and the Cutaneous Lupus Activity Investigator’s Global Assessment (CLA-IGA) for the investigator global assessment of disease activity domain. Several suitable instruments were identified for symptoms and health-related quality of life domains, but there was no clear superior instrument due to lack of validation data. For the patient global assessment disease activity domain, the available instruments were poorly defined and not validated for CLE.

Limitations of the study included the use of expert opinion to develop the core outcome set rather than consensus activity and a lack of validation studies for instruments to assess CLE domains (except for CLASI).

The researchers concluded, “Developed based on extensive literature review and expert opinion, this [core outcome set] should not be viewed as restrictive or unchangeable but rather as a timely starting point that will likely continue to be refined. Importantly, the establishment and uptake of a CLE [core outcome set] will enable improved design of CLE clinical studies and better synthesis of trial data.”

Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.

Reference

Guo LN, Perez-Chada LM, Borucki R, Nambudiri VE, Werth VP, Merola JF. Development of a working core outcome set for cutaneous lupus erythematosus: a practical approach to an urgent unmet need. Lup Sci Med. Published online December 27, 2021. doi:10.1136/lupus-2021-000529