Successful Treatment of Dry Mouth and Dry Eye Symptoms in Sjögren's Syndrome Patients With Oral Pilocarpine: A Randomized, Placebo-Controlled, Dose-Adjustment Study

Abstract

Background: : Sjögren's syndrome is characterized by the presence of xerostomia and/or xerophthalmia. Pilocarpine, a muscarinic cholinergic agonist, has been proven to be efficacious in treating radiation-induced xerostomia (up to 30 mg/day) and symptoms of dry mouth in Sjögren's patients (up to 20 mg/day).

Objective: : To compare the safety and efficacy of oral pilocarpine (dose-adjusted) versus placebo in the treatment of dry eye and dry mouth symptoms in Sjögren's syndrome at 6 and 12 weeks.

Methods: : In this 11-center, 256-patient placebo-controlled study, the safety and efficacy of oral pilocarpine (20 mg to 30 mg daily) for relief of Sjögren's-related dry mouth and dry eye symptoms was assessed. Changes in symptoms and salivary flow were measured over 12 weeks.

Results: : Compared with placebo, salivary flow was significantly increased in the pilocarpine group (P</= 0.0001) after the first dose and throughout the study. Significant improvement in patients' global assessment of dry mouth (P</= 0.0001) with relief in 5 of 7 separate oral symptoms (P</= 0.02) was reported by the treated patients throughout study. Minimal differences in 3 of 8 ocular symptoms were noted at 6 weeks (5-mg dose), but at 12 weeks (5- to 7.5-mg dose), the pilocarpine group demonstrated both significant improvement in global assessment of dry eyes (P</= 0.0001) and relief in 6 of 8 related symptoms (P</= 0.04). The drug was well tolerated at both doses. The most common pilocarpine-related side effects were sweating, urinary frequency, flushing, and chills.

Conclusions: : Significant relief in dry mouth symptoms was noted at 20 mg/day, and significant relief in ocular symptoms, including lower artificial tear requirement, was noted after the dose was increased to 30 mg/day.