Does ursodiol (ursodeoxycholic acid) cause pancytopenia?

From the Guidelines

Ursodiol does not typically cause pancytopenia, and there is no strong evidence to suggest a link between ursodiol therapy and the development of pancytopenia. Pancytopenia, a condition characterized by a reduction in all three blood cell types (red blood cells, white blood cells, and platelets), is not a known or common side effect of ursodiol therapy 1. Ursodiol, also known as ursodeoxycholic acid (UDCA), is primarily used to treat certain liver conditions like primary biliary cholangitis and to dissolve gallstones. The most common side effects of ursodiol include gastrointestinal symptoms such as diarrhea, nausea, vomiting, and abdominal pain.

Key Points to Consider

• Ursodiol works by decreasing the production and increasing the elimination of cholesterol in the liver, reducing the toxicity of bile acids, and protecting liver cells from damage 1.
• If a patient taking ursodiol develops symptoms of pancytopenia (such as unusual fatigue, frequent infections, or easy bruising/bleeding), they should seek medical attention promptly as this would likely be due to another cause rather than the ursodiol itself.
• High-dose ursodiol treatment (> 20 mg/kg/day) was associated with a worse disease outcome and should therefore be avoided 1.
• The current evidence does not support the use of ursodiol as a cause of pancytopenia, and its benefits in treating liver conditions and preventing colorectal cancer in patients with primary sclerosing cholangitis (PSC) are well-established 1.

Clinical Implications

• Clinicians should be aware of the potential benefits and risks of ursodiol therapy and monitor patients closely for any adverse effects.
• Patients taking ursodiol should be educated on the signs and symptoms of pancytopenia and instructed to seek medical attention if they experience any unusual fatigue, frequent infections, or easy bruising/bleeding.
• The dosage of ursodiol should not exceed 20 mg/kg/day to minimize the risk of adverse effects 1.

From the Research

Ursodiol and Pancytopenia

• There is no direct evidence in the provided studies that ursodiol causes pancytopenia 2, 3, 4, 5, 6.
• The studies focus on the etiology and diagnosis of pancytopenia, as well as the treatment of primary biliary cirrhosis and parenteral nutrition-associated cholestasis with ursodiol.
• Pancytopenia is a condition characterized by a reduction in the number of red blood cells, white blood cells, and platelets in the blood, and its causes include vitamin deficiencies, drugs, and bone marrow failure syndrome 2, 3.
• Ursodiol is used to treat primary biliary cirrhosis and has been shown to improve liver function and reduce the risk of treatment failure 5.
• There is limited information on the use of ursodiol in patients with pancytopenia, and no studies have investigated its potential to cause pancytopenia 2, 3, 4, 5, 6.

Potential Side Effects of Ursodiol

• The studies do not mention pancytopenia as a potential side effect of ursodiol 5, 6.
• Ursodiol is generally considered safe and well-tolerated, with few reported side effects 5, 6.
• However, the lack of information on the potential for ursodiol to cause pancytopenia does not necessarily mean that it is safe in patients with this condition, and further studies are needed to fully understand its effects 2, 3, 4, 5, 6.