What is Elmiron (pentosan polysulfate) used for in interstitial cystitis?

Elmiron (Pentosan Polysulfate) in Interstitial Cystitis

Elmiron (pentosan polysulfate sodium) is the only FDA-approved oral medication specifically indicated for the relief of bladder pain or discomfort associated with interstitial cystitis/bladder pain syndrome (IC/BPS). 1

Mechanism and Efficacy

• Pentosan polysulfate is a heparin-like, sulfated polysaccharide that is believed to repair damaged glycosaminoglycan (GAG) layers lining the urothelium and may provide an anti-inflammatory effect in patients with IC/BPS 2
• The American Urological Association (AUA) classifies pentosan polysulfate as a second-line oral treatment option with Grade B evidence strength 3
• Clinical studies show mixed results - some trials report significant improvement in pain, urgency, and frequency compared to placebo, while others show no difference in symptom improvement 3, 4
• Meta-analysis data indicates pentosan polysulfate is more efficacious than placebo for pain (NNT=7), urgency (NNT=7.5), and frequency (NNT=6), but not significantly different for nocturia 4

Dosing and Administration

• The standard dosage is 100 mg taken orally three times daily, with water at least 1 hour before meals or 2 hours after meals 1
• Treatment should be continuous as it is not a pain medication like aspirin or acetaminophen that provides immediate relief 1
• Patients should be informed that IC/BPS is typically a chronic disorder requiring ongoing management 5

Important Safety Considerations

• Clinicians must counsel patients considering pentosan polysulfate on the potential risk for macular damage and vision-related injuries 3

• The FDA approved a new warning label for pentosan polysulfate in June 2020 due to reports of a unique retinal pigmentary maculopathy associated with its use 3

• Safety recommendations include:

  • Obtaining a detailed ophthalmologic history prior to starting treatment 3
  • Conducting a comprehensive baseline retinal examination for patients with preexisting ophthalmologic conditions 3
  • Performing retinal examinations within six months of initiating treatment and periodically during continued treatment 3
  • Reevaluating risks and benefits if pigmentary changes develop in the retina, as these changes may be irreversible 3

• Pentosan polysulfate has weak anticoagulant properties that may increase bleeding risk 1

• Patients should inform their doctor before undergoing surgery or if taking anticoagulant therapy such as warfarin, heparin, high doses of aspirin, or anti-inflammatory drugs 1

Treatment Approach

• The 2022 AUA guidelines recommend an individualized approach to IC/BPS treatment rather than a strict tiered approach 3
• Pentosan polysulfate should be considered after first-line behavioral and non-pharmacologic approaches have been implemented 3
• A multi-modal approach combining pentosan polysulfate with other therapies is likely to be most effective for symptom control 3, 5
• Common side effects include hair loss, diarrhea, nausea, blood in stool, headache, rash, upset stomach, abnormal liver function tests, dizziness, and bruising 1

Clinical Outcomes

• Early studies showed significant improvement in pain, urgency, frequency, and nocturia in 20 of 24 patients within 4-8 weeks of treatment initiation 6
• Long-term studies indicate that patients who respond to pentosan polysulfate continue to experience progressive improvement with time 6
• The benefits and risks of pentosan polysulfate should be thoroughly discussed with patients before initiating or continuing treatment 3