Indication of Ellura (Pentosan Polysulfate Sodium)
Pentosan polysulfate sodium (Ellura) is the only FDA-approved oral agent for the treatment of interstitial cystitis/bladder pain syndrome (IC/BPS), specifically for the relief of bladder pain or discomfort associated with this condition. 1
Mechanism of Action and Efficacy
- Pentosan polysulfate sodium (PPS) is believed to repair damaged glycosaminoglycan (GAG) layers lining the urothelium and may provide an anti-inflammatory effect in patients with IC/BPS 2
- PPS has demonstrated efficacy in relieving bladder pain and discomfort in a proportion of patients with IC/BPS, though results from clinical trials have been mixed 1, 3
- Evidence suggests that earlier initiation of PPS treatment (within 6 months of IC/BPS diagnosis) may be associated with greater improvement in patient symptoms 4
- Long-term studies indicate that patients who respond positively to PPS appear to maintain this response over time 5
Clinical Evidence and Recommendations
- PPS has been extensively studied for IC/BPS and has an evidence strength rating of Grade B according to the American Urological Association (AUA) guidelines 1
- Some clinical trials have shown PPS to be significantly more effective than placebo in providing overall symptom improvement for IC/BPS patients 6
- Other studies have shown contradictory results, with some trials reporting no differences in symptom improvement compared to placebo 1, 3
- The AUA panel recommends discussing the benefits and risks of PPS with patients before initiating or continuing treatment 1
Important Safety Considerations
- In 2020, the FDA approved a new warning label for PPS due to concerns about retinal pigmentary maculopathy associated with its use 1
- Symptoms of this maculopathy may include difficulty reading, slow adjustment to low light environments, and blurred vision 1
- The risk of maculopathy appears to be related to the cumulative amount of PPS exposure 1
- Current recommendations for patients considering or using PPS include:
- Obtaining a detailed ophthalmologic history prior to starting treatment 1
- Conducting a comprehensive baseline retinal examination for patients with preexisting ophthalmologic conditions 1
- Performing a retinal examination within six months of initiating treatment and periodically during continued treatment 1
- Reevaluating the risks and benefits of continuing treatment if pigmentary changes in the retina develop 1
Dosing and Administration
- The standard dosing of PPS is 100 mg three times daily 5, 6
- Treatment may need to be continued for extended periods, as some symptoms may take 5 months or longer to show improvement 5
- Common adverse events include reversible alopecia, diarrhea, nausea, headache, and rash, with an overall adverse event frequency of less than 4% in long-term studies 5
Treatment Context
- IC/BPS treatment typically requires a multi-modal approach, with PPS being one component of therapy 1
- PPS is considered a second-line oral medication for IC/BPS according to AUA guidelines, alongside other options such as amitriptyline, cimetidine, and hydroxyzine 1
- Pain management alone is typically not sufficient treatment for IC/BPS; addressing underlying bladder-related symptoms is essential 1
Clinical Pitfalls to Avoid
- Failing to monitor for visual symptoms or conduct appropriate ophthalmologic examinations in patients on long-term PPS therapy 1
- Not recognizing that symptom improvement with PPS may take several months to become apparent 5
- Using PPS as monotherapy rather than as part of a comprehensive treatment approach for IC/BPS 1