Successful therapy of interstitial cystitis* with pentosan polysulfate¹
Parsons CL & Mulholland SG. J Urol 1987; 138: 513-516.
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• A randomised, double-blind, placebo-controlled study in patients (n= 75) experiencing symptoms of pain, urgency, frequency and/or nocturia for at least 1-year
• All patients had to have cystoscopic proof of lesions (Hunner lesions and/or glomerulations)
• elmiron^® or placebo was administered for a minimum of 4 months and some patients entered an extension study for a further 14 months
• Efficacy was evaluated based on the patient reported improvement on four typical symptoms

Significantly greater responses were seen with elmiron^® vs placebo across all subjective parameters

• Patients who responded to treatment showed improvement at 5-10 weeks and continued to improve during several months
• Long-term relief of symptoms was demonstrated in 24/25 patients treated for more than 18 months during the open-label study extension period
• When elmiron^® treatment was stopped, 80% of the patients found their symptoms reappeared within 3-12 weeks

Pentosan polysulfate sodium for therapy of interstitial cystitis^2*
Mulholland SG et al. Urology 1990; 35: 552-558.
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• A randomised, double-blind, placebo-controlled study in patients (n=110) who had been symptomatic for more than one year and failed to respond to previous therapy
• elmiron^® dose of 100mg, 3 times daily or placebo was administered for 3 months
• Primary endpoints:
  • 6-point patient-reported improvement
  • ≥50% overall improvement reported by patients

Pain was the first symptom to improve, representing a decrease in inflammatory response

• 18 months after study completion 44% of patients chose to continue with elmiron^®
• All adverse events were minor, with a higher incidence seen in the placebo group, but the difference was not significant

A quantitatively controlled method to study prospectively interstitial cystitis^* and demonstrate the efficacy of pentosan polysulfate³
Parsons CL et al. J Urol 1993; 150: 845-848
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• A randomised, double-blind, placebo-controlled study in patients (n= 148)
• elmiron^® dose of 100mg, 3 times daily or placebo was administered for 3 months
• Primary efficacy evaluation were:
  • patients’ self-evaluation of overall improvement
  • investigators’ evaluation of overall improvement changes in pain and urgency scale
  • From a 3 day bladder diary: change in nocturia, change in frequency and average voided volume

Improvements in symptoms were noted by both patients and healthcare professionals with elmiron^®

Significant improvements in pain and urgency were evident with elmiron^® use

A pilot clinical trial of oral pentosan polysulfate and oral hydroxyzine in patients with interstitial cystitis^4*
Sant GR et al. J Urol 2003; 170: 810-815.
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• A randomised, double-blind, placebo-controlled study in patients (n= 121) reporting at least moderate pain and frequency for a minimum of 6 months
• Four study treatment arms
  • elmiron^® 100mg, 3 times daily
  • elmiron^® 100mg, 3 times daily + hydroxyzine 25-50mg, once daily
  • hydroxyzine titrated to 25-50mg, once daily
  • placebo
• Primary endpoint:
  • Patient-reported Global Response Assessment (GRA) after 24 weeks of treatment

Global Response Assessment: patient reported perception of their change in condition

• Measured patient perception of overall change in their condition, grading pain, urgency, frequency, nocturia
• 7-point centred scale: worse, moderately worse, slightly worse, no change, slightly improved, moderately improved or markedly improved
• Treatment responder: moderately or markedly improved

Global Response Assessment results

• There were no statistically significant differences between the main treatments
  in symptom changes over time for any of the secondary end points
• Hydroxyzine (19/61, 31%) vs no hydroxyzine (12/60, 20%), (p=0.26)
• elmiron^® treatment (20/59, 34%) vs no elmiron^® (11/62, 18%), (p=0.064)
• Best response rate was in combination of elmiron^® and hydroxyzine (12/30, 40%)
  followed by elmiron^® alone (8/29, 28%) and hydroxyzine alone (7/31, 23%)
• The response rate on the placebo treatment was 13% (4/31)
• A trend for better efficacy was observed for the primary endpoint with elmiron^®
  (either alone or in combination with hydroxyzine) compared to those patients not receiving
  elmiron^®, but who might receive hydroxyzine (11/62, 18%), (p=0.064)
• The majority of adverse events were minor, and not specifically related to elmiron^® or hydroxyzine

Efficacy of pentosan polysulfate for the treatment of interstitial cystitis/bladder pain syndrome: results of a systematic review of randomized controlled trials⁵
van Ophoven A et al. Curr Med Res Opin 2019; 35(9): 1495-1503.
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• Systematic review of PubMed/ Medline and the Cochrane Library in June 2018 using the free-text search control terms “interstitial cystitis” or “bladder pain syndrome” and “clinical trial”
• Also a search for additional potentially unpublished studies on ClinicalTrials.gov
• Only randomized, placebo-controlled clinical trials with sufficient information to estimate at least one relevant effect size measure to compare the efficacy of oral elmiron^® versus placebo were included
• To be included, studies had to:
  • Evaluate the therapeutic effect of oral elmiron^®
  • Be placebo-controlled clinical trials
  • Involve patients with a clinical and cystoscopically-verified diagnosis of BPS
    with bladder lesions (ESSIC class 2-3)
  • Provide sufficient information to estimate at least one relevant effect size
    measure to compare the efficacy of elmiron^® vs placebo

Evidence acquisition

Efficacy of elmiron^® in patients with cystoscopic findings (Hunner’s lesions and/or
glomerulations)

• Four (of the six) studies identified limited enrolment to patients diagnosed based on cystoscopic
  examinations
  • Parsons & Mulholland 1987¹
  • Mulholland et al. 1990²
  • Parsons et al. 1993³
  • Sant et al. 2003⁴
• This patient population represents the patient population for whom elmiron^® is currently indicated in the EU⁵
  i.e. for the treatment of bladder pain syndrome characterized by either glomerulations or Hunner’s lesions in
  adults with moderate to severe pain, urgency and frequency of micturition⁶
• Duration of these four studies ranged from 3 to 6 months
• Patients consistently reported improvements of their BPS symptoms with elmiron^® across the four clinical studies

Pooled responder rates per treatment arm

• A 2-fold higher clinical improvement in pain, urgency and global response assessment has been seen with elmiron^® versus placebo