Progressive Familial Intrahepatic Cholestasis Medication

Updated: Oct 04, 2021
  • Author: Andrew J Wehrman, MD; Chief Editor: Carmen Cuffari, MD  more...
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Medication Summary

For information on most of the medications used to treat progressive familial intrahepatic cholestasis (PFIC), including antipruritic therapy and fat-soluble vitamins, see the Medscape Drugs and Diseases article Cholestasis.


Gallstone-solubilizing agents

Class Summary

Ursodeoxycholic acid (ursodiol), a naturally occurring bile acid present in small quantities in human bile, suppresses liver synthesis, suppresses secretion of cholesterol, and inhibits intestinal cholesterol absorption.

Ursodiol (Actigall, URSO)

Also called ursodeoxycholic acid. Shown to promote bile flow in cholestatic conditions associated with patent extrahepatic biliary system. Decreases cholesterol content of bile and decreases likelihood of sludging and bile stones. Hydrophilic bile acid thought to act by decreasing overall toxicity of bile acid pool.


Bile-acid binding agents

Class Summary

These agents are FDA approved for pruritus caused by biliary stasis.

Cholestyramine (Prevalite, Questran)

Forms a nonabsorbable complex with bile acids in the intestine, which in turn inhibits enterohepatic reuptake of intestinal bile salts. Take other medications at least 1 h before or 4-6 h after cholestyramine.

Not to be administered in dry powder form. Mix with plenty of water or applesauce.

May use as adjunct in primary hypercholesterolemia.


Hepatic enzyme inducers

Class Summary

These agents are used to induce activity of hepatic enzymes, thus enhancing bilirubin excretion, which may improve function in some patients with cholestasis. An antipruritic effect is noticed with reduction of serum bilirubin.

Phenobarbital (Luminal)

Mainly used as an anticonvulsant that interferes with transmission of impulses from thalamus to cortex of brain, causing an imbalance in central inhibitory and facilitatory mechanisms. Used in cholestasis to induce CYP450 system in treatment of neonatal hyperbilirubinemia and to lower bilirubin in chronic cholestasis.

Rifampin (Rifadin, Rimactane)

Inhibits RNA synthesis in bacteria by binding to beta subunit of DNA-dependent RNA polymerase, which in turn blocks RNA transcription.



Class Summary

Fat-soluble vitamins A, D, E, and K should be administered as individual supplements to ensure proper absorption.

Phytonadione (AquaMEPHYTON)

Vitamin K is a fat-soluble vitamin absorbed by the gut and stored in the liver. Necessary for function of clotting factors in coagulation cascade. Used to replace essential vitamins not obtained in sufficient quantities in diet or to further supplement levels.

Vitamin E (Liqui E)

Protects polyunsaturated fatty acids in membranes from attack by free radicals and protects RBCs against hemolysis.

Vitamin A (Aquasol A)

Needed for bone development, growth, visual adaptation to darkness, and testicular and ovarian function and as a cofactor in many biochemical processes.

Ergocalciferol (Calciferol, Drisdol)

Vitamin D stimulates absorption of calcium and phosphate from small intestine and promotes release of calcium from bone into blood; PO solution is 8000 U/mL (200 mcg/mL, 40 U/mcg).


Ileal Bile Acid Transport Inhibitors

Class Summary

Inhibitors of ileal bile acid transport (IBAT) act locally in the distal ileum to decrease reuptake of bile acids and increase clearance of bile acids through the colon, thereby reducing bile acid serum concentration.

Odevixibat (Bylvay)

Indicated for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 3 months and older.