Pilocarpine Drops for Xerostomia in Patients With a Life-Limiting Condition or Frailty: A Double-Blind Placebo-Controlled Randomized Clinical Trial.

Authors

Annelot van der Meulen, Judith Gulikers, Maurice Theunissen, Sander van Kuijk, Merel Kimman, Bénédicte Coenegracht, Arianne Stoppelenburg, Yvette van der Linden, Marieke van den Beuken-van Everdingen

Published in

Journal of the American Medical Directors Association. Volume 27. Issue 10. Pages 106428. Aug 25, 2026. Epub Aug 25, 2026.

Abstract

To assess the effectiveness and acceptability of oral pilocarpine drops for the treatment of xerostomia in patients with a life-limiting condition and/or frailty.
Double-blind, placebo-controlled randomized clinical trial, comparing topical pilocarpine with placebo.
This randomized controlled trial was conducted from June 2023 to April 2025 at 2 hospitals and 3 nursing homes in the Netherlands, involving 92 adults with xerostomia [Numeric Rating Scale ≥5 (NRS, 0-10)] and a life-limiting condition and/or frailty.
Participants received 4 weeks of either topical pilocarpine or placebo drops. The primary outcome was the mean change in xerostomia severity at 4 weeks. One hundred twenty participants were required to detect a meaningful difference between groups. Secondary outcomes included proportion of responders, xerostomia severity, oral health-related quality of life global perceived effect, side effects, medication changes, adherence, and medical costs at baseline, 2, and 4 weeks. Regression analyses were performed.
Of 299 patients screened, 92 were randomized (placebo, n = 48, pilocarpine, n = 44). Linear regression showed no significant between-group difference at week 2 or week 4 [adjusted β = 0.35; (95% CI, -0.44 to 1.15) and β = -0.37; (95% CI, -1.31 to 0.58), respectively]. Both groups showed a significant decrease in mean NRS from baseline to week 2 (placebo, -1.4; pilocarpine, -1.5), but the NRS decreased further only for pilocarpine between week 2 and week 4 [-0.86 (95% CI, -1.56 to -0.15)]. Oral health-related quality-of-life score significantly improved for pilocarpine compared with placebo at 2 weeks [adjusted β = 2.8 (95% CI, 0.91, 4.65)]. Other secondary outcomes showed no significant between-group differences. Adverse effects were limited.
Topical pilocarpine did not significantly reduce xerostomia severity compared with placebo. However, only pilocarpine showed continuation of reduction in xerostomia severity over 4 weeks, with good tolerability. As the current study was underpowered, the potential clinical benefit of pilocarpine should be further investigated in adequately powered trials.

PMID:
42641502
Bibliographic data and abstract were imported from PubMed on 26 Aug 2026.

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