pentosan polysulfate sodium
Regulatory sources consulted
Approved indications
- Bladder pain syndrome in adults with glomerulations or Hunner lesions, moderate or severe pain, urgency, and urinary frequency.
Contraindications
Absolute
- Hypersensitivity or active bleeding; menstruation is not a contraindication.
Clinical warnings
- Major warning · Rare pigmentary maculopathy has been reported; it may be irreversible and progress even after stopping. Perform a baseline retinal examination within 6 months of starting and periodic follow-up; consider stopping for reading difficulty or prolonged dark adaptation. — FDA/DailyMed, set_id f0ba651e-3d8a-11df-8fbe-119855d89593
- Major warning · It is a weak anticoagulant. Assess bleeding before procedures and monitor closely in significant renal or liver impairment. — CIMA/AEMPS, ficha técnica 1171189001
Drug interactions
- HighAnticoagulants, heparins, thrombolytics, antiplatelets, or NSAIDs
Mechanism: Effects on coagulation may increase bleeding risk.
Recommendation: Assess for bleeding and adjust treatment if needed.
CIMA/AEMPS, ficha técnica 1171189001https://cima.aemps.es/cima/dochtml/ft/1171189001/FT_1171189001.html
Adverse events
Common (≥1%)
Headache · Dizziness · Nausea or diarrhea · Abdominal pain or distension · Rectal bleeding · Alopecia or peripheral edema
Rare but serious
Pigmentary maculopathy · Bleeding or coagulation disorder · Thrombocytopenia
Pregnancy and lactation
Not recommended during pregnancy and should not be used while breastfeeding.
Recent literature (PubMed)
Osteoarthritis (OA) is the most prevalent arthritis-type and is a major contributor to chronic joint pain, impaired physical function, and limited mobility. By the end of 2020, a total of 595 million, equal to 7·6% of the global population, had OA; this figure is expected to rise exponentially by 2050. Even while the disorder's intricate pathophysiology is starting to appear intelligible, we are yet to have a cure for the disorder. OA is typically managed with traditional palliative measures, such as topical and systemic analgesics, including non-steroidal anti-inflammatory drugs, therapeutic exercise, and braces. Sometimes, intra-articular glucocorticoids, viscosupplementation, or regenerative interventions provide short-term pain relief and functional improvement; some may require arthroplasty. Researchers continue their efforts to unveil a new therapeutic target to be effective in OA that modifies symptoms and arrests disease progression as well. In the present literature review, insights into new therapeutic strategies in OA, for example, liposome-based dexamethasone, microspore-based triamcinolone, nerve growth factor antagonist, anti-ADAMTS-5 (A Disintegrin And Metalloproteinase Thrombospoidin Motifs - 5), pentosan polysulfate sodium, allogeneic stem cells, C-C chemokine receptor type-4 (CCR4) ligand 17 inhibitor, Wnt-signaling inhibitor, and anti-obesity medications are provided.
Pentosan polysulfate sodium (PPS), a semisynthetic sulfated polysaccharide, is the only FDA-approved oral therapy for interstitial cystitis. Recent studies have described a progressive, vision-threatening macular condition associated with long-term PPS use. We reviewed all publications concerning PPS maculopathy to consolidate known clinical features and to evaluate the strength of this association. Current literature supports a strong dose-dependent association between PPS exposure and a progressive maculopathy impacting the retinal pigment epithelium (RPE) and RPE-photoreceptor interface that may worsen even after drug cessation. Initial symptoms may include prolonged dark adaptation and difficulty reading with relative visual acuity preservation. Fundus examination often shows macular pigment clumps corresponding to lesions of focal RPE thickening. Fundus autofluorescence most clearly depicts the condition, with a distinctive pattern of hypo- and hyperautofluorescent spots in the posterior pole that sometimes extends to the retinal periphery. Many cases also show a characteristic peripapillary hypoautofluorescent halo. Near infrared reflectance may aid in early detection. RPE atrophy, cystoid macular edema, and macular neovascularization may also occur, potentially resulting in loss of central acuity. This newly described association implies significant public health risk. Ophthalmologists should screen PPS users with multimodal retinal imaging, and prescribers should minimize dose and duration of PPS use.
Interstitial cystitis/bladder pain syndrome (IC/BPS) is defined as persistent or chronic discomfort perceived to be related to the urinary bladder accompanied by urinary urgency or frequency. Pharmacotherapies used to treat IC/BPS include oral and intravesical agents. Oral therapies include amitriptyline, hydroxyzine, cyclosporine A, and pentosan polysulfate sodium (PPS), although the recent finding of pigmented maculopathy with chronic PPS is very concerning and must be discussed with patients, many of whom will choose to either come off this medicine or not even start it. Certolizumab pegol is a pharmacologic therapy that is currently in clinical development for treatment of IC/BPS symptoms.
Pentosan polysulfate sodium (PPS) maculopathy is a recently described condition of macular toxicity. We report the prevalence (15 %), the clinical characteristics, and the multimodal imaging (MMI) features of PPS maculopathy based on a final prospective analysis of a cohort of 154 subjects and highlight the association with inflammatory bowel disease (IBD). In addition, we summarize the most recent and relevant literature on PPS maculopathy. This study and other recent analyses provide solid evidence of the toxic effects of PPS on the retina and choroid and refute the concept that the maculopathy is an association of the underlying disease, interstitial cystitis. Moreover, this study and others raise a major concern that PPS may also be toxic to the bowel. Vigilant surveillance of patients receiving this potentially harmful drug is critical. We review the recommended screening guidelines, the pathophysiology and MMI spectrum of disease, the differential diagnosis including MMI clues to an accurate diagnosis, and the progression of retinopathy that can take place despite discontinuation of the drug, emphasizing the critical importance of early detection of toxicity.
For women with recurrent urinary tract infection (UTI), previous U101 study has shown that pentosan polysulfate sodium (PPS) monotherapy for 16 weeks significantly reduced UTI episodes in the treatment group throughout the trial period. In this follow-up study, we aimed to assess whether the effects of PPS would last after completion of the trial to prevent recurrent UTIs. Conducted from 2018 to 2019, the U101 study was a multicenter, prospective, phase 2a, randomized trial, enrolling women with recurrent UTI to study the effects of a 16-week oral PPS monotherapy. After approximately two years, the follow-up was conducted by phone interview, obtaining data including self-reported UTI events, quality of life questionnaire, and adverse events. The primary endpoint of follow-up study was UTI recurrence-free survival and the secondary endpoints were quality of life and adverse events. Approximately two years after completion of the trial, the rate of recurrent UTI was 25% (3 of the 12 patients) in the PPS group and 85.7% (12 of the 14 patients) in the control group. Over the entire follow-up period, the UTI recurrence-free survival was significantly better in the PPS group than in the control group (log-rank test p < 0.001). The quality of life at two years was significantly improved in the PPS when compared to the control group (91.7 vs. 77.5, p < 0.001). No late adverse event was observed after cessation of the treatment. In this study, sixteen weeks of PPS monotherapy in women with recurrent UTI significantly reduced the numbers of recurrent UTI episodes during the 2-year follow-up.