RepurpSS-II: Hydroxychloroquine plus Leflunomide in Sjögren's Save

A small pilot, phase 2b trial has shown that the combination of leflunomide and hydroxychloroquine (LEF+HCQ) was effective and safe in treating patients with active Sjögren's disease.

 

The RepurpSS-II study was a randomised, placebo-controlled, double-blind, phase 2b trial from Utrecht, The Netherlands, that enrolled adults with Sjögren's and active disease (ESSDAI of at least 5). Patients were randomized to receive placebo or leflunomide (20 mg) and hydroxychloroquine (400 mg) orally, once a day. The week 24 primary endpoint was the change in ESSDAI between groups.

 

A total of 46 SS patients were enrolled and treated with LEF+HCQ (n=21) or placebo (n=25). The mean difference in ESSDAI between groups at 24 weeks was –4.135 (95% CI –6·558 to –1·709, p=0·0012). 

 

Adverse events were similar between groups; gastrointestinal discomfort was most common (six [29%] of 21 patients in the leflunomide–hydroxychloroquine group and four [16%] of 25 in the placebo group). There were no deaths during the study.

 

These affordable and widely available treatment option merits further investigation.