Cholic Acid Brand Name– Cholbam
What is Cholic Acid
Cholic acid is an oral therapy for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects (SED) and for patients with peroxisomal disorders (PD), including Zellweger spectrum disorders.
Although the incidence of bile acid synthesis disorders is unknown, it is believed to represent 1 to 2% of childhood cholestatic disorders.
Most of the known defects cause progressive liver injury, usually accompanied by inflammation.
When diagnosed early, bile acid synthesis defects can be successfully treated with the addition of substitute bile acids.
Numerous different disorders with both clinical and biochemical heterogeneity linked to peroxisomal dysfunction have been identified.
Peroxisomes are cell organelles found in all human cells except erythrocytes. They are mainly involved in lipid metabolism, are involved in the oxidation of phytanic acid, and in the formation of bile acids from mevalonate via cholesterol.
Cholic acid is a primary bile acid produced by the liver where it is synthesized from cholesterol. In bile acid synthesis disorders, due to both bile synthesis disorders in the biosynthetic pathway and in peroxisomal disorders, deficiency of primary bile acids leads to unregulated accumulation of intermediate bile acids and cholestasis.
Cholic acid enhances bile flow and provides the physiologic feedback inhibition of bile acid synthesis. In a long-term trial followed by an extension trial, improvements were noted in both baseline liver function tests (LFTs) and growth.
An uncontrolled trial involving 50 patients with bile acid synthesis disorders treated over an 18-year period (plus a 21-month extension trial that included 21 of the original patients plus 12 new patients) showed a positive response in 64% of patients with evaluable data.
Twenty-nine patients with Zellweger spectrum disorders were also included (the extension trial included 10 of the original patients plus 2 new patients). A response was noted in 46% of patients.
Specific laboratory and clinical monitoring is required while receiving cholic acid to assess efficacy and the development of potential adverse drug reactions.
Cholic acid was approved by the FDA for patients 3 weeks of age and older in March 2015.
Indications
- bile acid synthesis disorders
- peroxisomal disorders
- Zellweger spectrum disorders
Side Effects
- abdominal pain
- cholelithiasis
- cholestasis
- diarrhea
- elevated hepatic enzymes
- gastroesophageal reflux
- hyperbilirubinemia
- infection
- jaundice
- malaise
- nausea
- peripheral neuropathy
Monitoring Parameters
- INR
- LFTs
- serum alkaline phosphatase
- serum bilirubin
Contraindications
- breast-feeding
- hepatic disease
- pregnancy
- requires an experienced clinician
Interactions
- Aluminum Hydroxide
- Aluminum Hydroxide; Magnesium Carbonate
- Aluminum Hydroxide; Magnesium Hydroxide
- Aluminum Hydroxide; Magnesium Hydroxide; Simethicone
- Aluminum Hydroxide; Magnesium Trisilicate
- Bile acid sequestrants
- Cholestyramine
- Colesevelam
- Colestipol
- Cyclosporine
