Pentosan Polysulfate Maculopathy

Excerpt

Pentosan polysulfate (PPS) is a semisynthetic pentasaccharide heparinoid with anticoagulant properties. It was initially used in the 1950s as a thrombolytic due to the ability of the molecule to bind the glycocalyx of circulating red blood cells. PPS is the only medication approved by the United States Food and Drug Administration to treat interstitial cystitis (IC).

IC is characterized by bladder pain (suprapubic, pelvic, urethral, vaginal, or perineal) caused by filling and relieved by emptying with petechial bladder mucosal hemorrhages on endoscopy and decreased bladder compliance on urodynamics. This disease is very common, affecting over 1 million Americans, the vast majority of whom are female. In the bladder, PPS is postulated to bind to the urothelium and replace disrupted glycosaminoglycans to protect the urothelium. Less frequently, PPS is used for other indications, including irritable bowel syndrome, pelvic pain syndrome, and inner bladder wall cracks. The recommended dosage for PPS is 100 mg, 3 times a day.

Twenty-two years after its approval as a second-line agent for interstitial cystitis, a 6-patient case series described a progressive maculopathy associated with long-term use of the drug, an association that researchers have demonstrated and characterized multiple times.