What is pilocarpine?

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What is Pilocarpine?

Pilocarpine is a cholinergic parasympathomimetic agent that binds to muscarinic receptors to stimulate secretion from exocrine glands, particularly salivary, lacrimal, and sweat glands. 1

Mechanism of Action

  • Pilocarpine exerts its effects through muscarinic receptor activation, predominantly targeting M3 receptors, which triggers increased secretion from multiple glandular systems including salivary, lacrimal, sweat, gastric, pancreatic, and intestinal glands 1

  • The drug increases salivary flow 2-3 fold compared to placebo, with effects maintained throughout 12 weeks of treatment 2

  • When administered orally, pilocarpine produces onset of action at 20 minutes, peak effect at 1 hour, and duration of 3-5 hours 1

Clinical Applications

Primary Indications

  • Sjögren's Syndrome: Licensed worldwide for treatment of dry mouth (xerostomia) and dry eye symptoms in patients with Sjögren's syndrome 3

  • Radiation-Induced Xerostomia: Approved for treating dry mouth following radiation therapy for head and neck cancers 1, 4

  • Glaucoma: When applied topically to the eye, it causes miosis and lowers intraocular pressure 1, 5

Efficacy Profile

  • Pilocarpine shows greater improvement in dry mouth symptoms than dry eye symptoms at the standard 20 mg/day dosage 2

  • In head and neck cancer patients, 63% showed increased whole saliva flow with 5 mg dosing and 90% with 10 mg dosing after the first dose 1

  • For Sjögren's syndrome, approximately 50% of patients experience symptom improvement compared to 25% with placebo 4

Dosing and Administration

  • Standard dosing: 5 mg four times daily (20 mg/day total) for moderate glandular dysfunction 2

  • Dose escalation: Can be increased to 30 mg/day (7.5 mg four times daily) for enhanced ocular symptom relief 6

  • Available as oral tablets in 5 mg and 7.5 mg strengths 1

Adverse Effects

  • Most common: Excessive sweating occurs in over 40% of patients 2

  • Other parasympathomimetic effects: Urinary frequency, flushing, rhinitis, nausea, and chills 6

  • Discontinuation rate: Approximately 2% of patients stop treatment due to side effects, primarily sweating 2

  • Serious toxicity: Overdose can cause bradycardia, hypotension, and muscarinic symptoms, treatable with atropine 7

Contraindications and Precautions

  • Use with caution in patients with asthma, cardiac arrhythmia, iridocyclitis, and closed-angle glaucoma due to parasympathomimetic effects 4

  • Pilocarpine may cause paradoxical cardiovascular effects including both hypertension and hypotension, as well as both bradycardia and tachycardia 1

Comparison to Alternatives

  • Cevimeline: Another muscarinic agonist that may have fewer systemic adverse effects than pilocarpine but is not as widely available worldwide 2, 3

  • Cost consideration: In some markets, pilocarpine oral formulations cost significantly more than off-label use of pilocarpine eye drops 4

References

Guideline

Pilocarpine Therapy for Sjögren's Syndrome

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

Pilocarpine toxicity and the treatment of xerostomia.

The Journal of emergency medicine, 2004

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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