From the Guidelines
Ursodiol should not be used for the routine treatment of newly diagnosed primary sclerosing cholangitis (PSC) due to lack of evidence showing improvement in outcomes and potential harm with high doses.
Introduction to Ursodiol
Ursodiol, also known as ursodeoxycholic acid (UDCA), is a hydrophilic bile acid used to treat various cholestatic liver diseases. It works by improving bile flow and reducing the production of toxic bile acids, which can cause liver damage.
Dosage and Administration
The typical adult dosage of ursodiol for gallstone dissolution is 8-10 mg/kg/day, divided into two or three doses, usually taken with meals for better absorption. For primary biliary cholangitis, the standard dose is 13-15 mg/kg/day.
Efficacy in Primary Sclerosing Cholangitis (PSC)
Studies have shown that ursodiol improves liver biochemistry in patients with PSC, but there is no evidence that it improves outcomes such as death, liver transplantation, or development of complications 1. In fact, high doses of ursodiol (28-30 mg/kg/day) may be harmful, with increased rates of serious adverse events and primary endpoints 1.
Recommendations
Based on the available evidence, the use of ursodiol is not recommended for the routine treatment of newly diagnosed PSC (strength of recommendation: STRONG; quality of evidence: GOOD) 1. For patients already established on ursodiol therapy, there may be evidence of harm in patients taking high-dose ursodiol, and the decision to continue or stop treatment should be made on a case-by-case basis.
Side Effects and Contraindications
Ursodiol is generally well-tolerated, with minimal side effects, which may include diarrhea, nausea, or abdominal discomfort. However, it is contraindicated in patients with certain gallbladder conditions, such as acute cholecystitis or biliary tract obstruction, and effectiveness decreases with larger or calcified gallstones.
Key Points
- Ursodiol improves liver biochemistry in PSC patients, but does not improve outcomes.
- High doses of ursodiol may be harmful.
- Ursodiol is not recommended for routine treatment of newly diagnosed PSC.
- Patients already on ursodiol therapy should be evaluated on a case-by-case basis to determine the risks and benefits of continuing treatment.
From the FDA Drug Label
Ursodiol capsules are indicated for patients with radiolucent, noncalcified gallbladder stones < 20 mm in greatest diameter in whom elective cholecystectomy would be undertaken except for the presence of increased surgical risk due to systemic disease, advanced age, idiosyncratic reaction to general anesthesia, or for those patients who refuse surgery. Ursodiol capsules are indicated for the prevention of gallstone formation in obese patients experiencing rapid weight loss.
The main indications for Ursodiol are:
- Patients with radiolucent, noncalcified gallbladder stones < 20 mm in diameter who are at increased surgical risk or refuse surgery.
- Prevention of gallstone formation in obese patients experiencing rapid weight loss 2.
From the Research
Ursodiol Overview
- Ursodiol, also known as ursodeoxycholic acid, is a hydrophilic bile acid used to treat primary biliary cirrhosis (PBC) 3.
- It works by reducing the concentration and injury from relatively toxic bile acids 4.
Efficacy of Ursodiol
- Studies have shown that ursodiol improves liver function tests and prolongs survival in patients with PBC 3, 5.
- A multicenter, controlled trial found that ursodiol treatment resulted in significant improvements in serum levels of bilirubin, alkaline phosphatase, and other liver function tests 3.
- Long-term ursodiol therapy has been shown to slow the progression of PBC and reduce the need for liver transplantation 6.
Optimum Dose of Ursodiol
- The optimum dose of ursodiol has been found to be around 13.5 mg/kg/day, which is equivalent to 900 mg/day 5.
- A dose-response study found that higher doses of ursodiol (900 and 1200 mg/day) were more effective than lower doses (300 and 600 mg/day) in improving liver function tests 5.