Background and aims Hydroxychloroquine (HCQ) is used by the majority of patients who have incomplete lupus erythematosus (ILE), defined as positive ANA and 1–2 other criteria for SLE, although efficacy in this situation has never been proven in a rigorous clinical trial. The Study of anti-Malarials in Incomplete Lupus Erythematosus (SMILE) is a proposed placebo-controlled trial of HCQ in ILE designed to measure the effect of drug on progression to SLE. In order to judge trial feasibility, “mock recruitment” was performed.
Methods 45 patients seen in outpatient clinics of the SMILE investigators for ANA and musculoskeletal or cutaneous complaints were interviewed using a structured script explaining the need for the trial, potential risks and benefits of HCQ and the possible randomization to placebo. They were then asked questions to ascertain their understanding of the trial and their willingness to enrol.
Results 96% of the subjects were female; median age was 35 and median symptom duration 3 years. 13% were Hispanic and 13% were African American. 18% had a personal history of autoimmune disease other than lupus; 42% had a family history. Musculoskeletal and cutaneous symptoms were each in 60% of subjects. 73% of subjects were interested, and 64% were likely to enrol. The most common reason for disinterest was lack of time to participate (50%), risks of HCQ (25%) and possibility of getting placebo (19%).
Conclusions A placebo-controlled clinical trial is feasible when the standard of care is an approved drug. 50% more subjects need to be screened to have enough to enrol in the trial.
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