Note: Some physicians now urge Elmiron users to undergo vision screening for maculopathy (eye damage) at least once a year. Those with evidence of retinal damage or atrophy should discuss stopping Elmiron therapy with their doctor. Consumers should not alter their medication or dosage without first seeking professional medical advice.
Elmiron (generic name: pentosan polysulfate sodium) is a prescription drug used to treat a disease called interstitial cystitis (IC). IC is a chronic, painful bladder condition. Many people with IC rely on Elmiron to treat their symptoms.
However, Elmiron may damage the eye (retina), potentially leading to blindness. Elmiron users may be concerned about this side effect and how to respond to it.
What Pentosan Polysulfate Sodium?
Pentosan polysulfate sodium (PPS) is the active ingredient in Elmiron.
Pentosan polysulfate sodium belongs to the heparinoid class of molecules. This means it has properties similar to heparin, an anti-clotting drug (anticoagulant). PPS is a scentless white powder. PPS is also a weak anticoagulant (blood thinner), but it is not used to treat clotting-related disorders.
Interesting Fact: PPS isn't just prescribed to humans. Veterinarians use an injectable form of PPS to treat osteoarthritis in animals.
General Elmiron Usage Information
Indication: for the relief of bladder pain/discomfort from interstitial cystitis (IC)
Dosage: 100 mg capsule, three times per day
Manufacturer: Ortho - McNeil - Janssen Pharmaceuticals, Inc.
Generic: none available
Typical Cost: ~$900 per 90-day supply
It may take three to six months for symptoms to improve. The manufacturer has not determined the value or risk of taking the drug for more than six months in patients who do not achieve pain relief by that point.
What Does Elmiron Treat?
Elmiron treats interstitial cystitis (IC). IC is a rare but debilitating bladder condition. It affects approximately 1 million people in the United States. The disease mainly affects women.
IC causes a number of symptoms that can vary in frequency and severity.
How Does Elmiron Help Treat Interstitial Cystitis?
How Elmiron works to treat IC is officially unknown. However, some publications have popularized one theory about its efficacy. Researchers claim PPS (Elmiron's active ingredient) may reinforce defective portions of the bladder's lining.
Per the theory above, PPS may supplement the bladder's natural lining. As the drug accumulates, PPS may fill in for bladder-lining molecules that are missing or damaged.
Interesting Fact: Scientific studies confirm that PPS does build up in the bladder over a period of months.
Once PPS reaches a certain level, the bladder lining may function better. This may explain how Elmiron reduces pain and other symptoms of IC.
Interstitial Cystitis Symptoms Elmiron Treats
- Bladder, pelvic and/or abdominal pain
- Frequent urination
- Waking up in the night to urinate (nocturia)
- Insomnia and fatigue (resulting from nocturia)
- A feeling of urgency to urinate
How Do People Get Interstitial Cystitis?
Doctors and researchers have yet to determine a clear cause for IC. However, several studies have suggested IC may arise due to flaws in the protective lining of the bladder.
Potential IC Cause: Protective Coating Dysfunction
A healthy bladder creates a layer of protective molecules between urine and bladder tissue. These molecules have structures similar to PPS (Elmiron's active ingredient).
Researchers believe this layer may be damaged or otherwise dysfunctional in IC patients. Without this protective layer intact, it may be possible for harmful substances to reach and irritate the bladder wall and muscle cells.
If harmful substances in urine can reach bladder tissue underneath the protective layer, this may cause pain and urinary frequency and urgency. In other words, damage to the bladder's protective lining may cause IC.
This may explain Elmiron's efficacy in treating IC, given its ability to build up in the bladder over time.
Does Elmiron Cure Interstitial Cystitis?
No, Elmiron does not cure interstitial cystitis (IC). It does improve certain symptoms of IC. According to pre-approval clinical studies, approximately 38% of patients showed greater than 50% pain relief. This level of pain relief was only achieved after taking the drug for at least three months.
There is currently no known cure for IC, but research is ongoing.
Elmiron Side Effects
The most serious adverse event reported for Elmiron is eye damage. The specific form of eye damage Elmiron causes is called pigmentary maculopathy. This side effect is not referenced in the common side effects section of the drug label. However, you can find information about this side effect in the Warnings and Contraindications section below.
1 to 4% of Elmiron patients reported the following side effects in a pre-approval trial:
- Hair loss (alopecia)
- Stomach pain
- Liver function changes
In a post-approval trial, 6.3% of patients reported rectal bleeding (hemorrhage). This occurred while taking the standard 100-mg dose of Elmiron three times a day. Another group of patients took 900 mg of the drug per day. In this higher dose group, 15% of patients reported rectal bleeding.
Any patient who experiences a bleeding event while taking Elmiron should seek medical attention immediately.
Long-Term Elmiron Side Effects
The five most common Elmiron side effects reported to the FDA between 1997 and 2020 are:
- Hair loss (alopecia)
According to scientific data, Elmiron-associated hair loss is reversible.
The same cannot be said of Elmiron-associated maculopathy, or eye damage. In one study, Elmiron patients with maculopathy did not experience reversal of eye damage after stopping Elmiron therapy. Given the condition may be irreversible, maculopathy appears to be the most serious long-term side effect of Elmiron.
Side Effects of Stopping Elmiron Therapy
Between 1997 and 2020, the FDA received four reports of withdrawal syndrome from patients who took Elmiron. According to these reports, patients experienced the following symptoms:
- Hair loss
- Macular degeneration
- Visual impairment
- Weight loss
The relationship between these adverse effects and Elmiron therapy is unclear at this time.
Pigmentary changes in the retina, reported in the literature as pigmentary maculopathy, have been identified with long-term use of ElmironElmiron 100 mg Capsules
Warnings and Contraindications
The Elmiron drug label did not list any official warnings until very recently. In June 2020, Janssen Pharmaceuticals added a single warning for retinal pigmentary changes.
The label claims the visual consequences of these pigment abnormalities are unknown. Most cases of Elmiron vision loss occur after three or more years of use.
These pigmentary changes may come with the following symptoms:
- Difficulty reading
- Trouble adjusting to low light
- Blurred vision
Elmiron users are instructed to have a baseline retinal examination before or shortly after starting the drug. The manufacturer also recommends regular follow-up screenings with a healthcare professional.
Prescribers are urged to exercise caution when considering Elmiron for patients with:
- Increased risk of bleeding for any reason
- Previous heparin-related complications
- Hepatic insufficiency
Patients should be aware that Elmiron does have a weak anti-clotting effect. This may prolong bleeding time.
Elmiron has only one contraindication. Elmiron should not be used in patients who have a known hypersensitivity to it or any similar drugs.
Notable Elmiron Drug Interactions
There are currently no other pill-based drugs approved to treat the same symptoms as Elmiron. Some patients have reported using over-the-counter drugs like Cystex and Azo, but they have not proven very effective.
Physicians have used other therapies in an off-label manner to treat IC, including:
- Amitriptyline (an antidepressant)
- Cyclosporine A (an immune system-modulating drug)
- Adalimumab (an immune system-modulating drug)
- Dimethyl sulfoxide (DMSO)
Note: None of the therapies listed above are approved for treatment of IC. Some of them must be administered directly to the bladder. Patients should discuss potential Elmiron alternatives with a licensed physician.
A 2018 study found a link between Elmiron and a new form of macular disease. Some patients reported vision loss, such as difficulty reading and adapting to low-light situations.
A subsequent study found that symptoms did not improve after discontinuing the drug. Researchers believe this condition may involve permanent vision loss.
As a result, some Elmiron users have filed lawsuits against Elmiron manufacturers. These lawsuits claim manufacturers knew the drug could damage vision and did not warn patients.
Interesting Fact: Some physicians have mistaken Elmiron eye damage for age-related macular degeneration or pattern dystrophy.