Hydroxychloroquine for Sjögren syndrome does not appear effective

Hydroxychloroquine did not improve symptoms of primary Sjögren syndrome during 24 weeks of treatment compared with placebo, according to a study.

Researchers randomly assigned 120 patients with primary Sjögren syndrome from 15 university hospitals in France: 56 received hydroxychloroquine (400 mg per day) and 64 received placebo for 24 weeks. Both groups received hydroxychloroquine between weeks 24 and 48.

Results appeared in the July 16 Journal of the American Medical Association.

At 24 weeks, the proportion of patients meeting the primary end point (defined as a 30% or greater reduction in 2 of 3 scores evaluating dryness, pain, and fatigue) was 17.9% (10 of 56) in the hydroxychloroquine group and 17.2% (11 of 64) in the placebo group (odds ratio, 1.01; 95% CI, 0.37 to 2.78; P=0.98). Looking at the symptoms individually, the researchers also failed to find clinically significant differences between the treatment and placebo groups, although pain appeared to be nonsignificantly improved by hydroxychloroquine.

Hydroxychloroquine had no efficacy in the subgroups of patients with anti-SSA autoantibodies, high immunoglobulin G levels, or systemic involvement. During the first 24 weeks, there were 2 serious adverse events in the hydroxychloroquine group and 3 in the placebo group. In the last 24 weeks, there were 3 serious adverse events in the hydroxychloroquine group and 4 in the placebo group.

The trial extends the negative results of the only previous controlled crossover trial of the treatment, which included 19 patients and suggested that previous open trials overestimated the therapeutic efficacy of hydroxychloroquine, the authors wrote.

“The absence of superiority of 6 months of hydroxychloroquine over placebo was consistent whatever the clinical variable evaluated,” they wrote. “We observed no difference with the 2 treatments in objective features of dryness, disease-related discomfort, or quality of life assessed by validated questionnaires.”