Sitosterolemia (Phytosterolemia) Medication

Updated: Jul 07, 2022
  • Author: Robert D Steiner, MD, FAAP, FACMG; Chief Editor: Luis O Rohena, MD, PhD, FAAP, FACMG  more...
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Medication Summary

The goals of pharmacotherapy are to reduce morbidity and prevent complications. Ezetimibe improves VLDL and HDL subfraction distribution, thereby reducing the atherogenic lipid profile, thus providing potential clinical benefit in sitosterolemia even beyond cholesterol and plant sterol reduction. [41]


Bile acid-binding resins

Class Summary

These agents are used as lipid-regulating drugs to modify blood lipid concentrations. They are used in the management of hyperlipidemias and for the reduction of cardiovascular risk. They lower cholesterol by combining with bile acids in the gastrointestinal tract, thus preventing their reabsorption. This leads to increased cholesterol oxidation to replace the lost bile acids and increased hepatocyte low-density lipoprotein (LDL)-receptor synthesis, which results in reduced LDL-cholesterol levels.

Reductions of approximately 45% in cholesterol and plant sterols have been achieved with administration of bile acid-binding resins. Three of five Chinese patients received cholestyramine with a very good response initially, but all discontinued therapy due to poor compliance. [5]

Cholestyramine (Prevalite, Questran)

Bile acid sequestrant shown to lower plasma sterol levels in sitosterolemia. Dosage is, in part, determined by clinical and biochemical response. Pediatric doses up to 12 g/d have been used in sitosterolemia.

Start with low dose; administer PO as slurry in water, juice, or milk before meals; chewable bars are also available.


Antilipemic Agent, 2-Azetidinone

Class Summary

These agents inhibit dietary cholesterol absorption from the small intestine.

Ezetimibe (Zetia)

First in new class of cholesterol-lowering agents that inhibits intestinal absorption of cholesterol. Targets the sterol transporter, Niemann-Pick C1-Like 1 (NPC1L1), which is involved in the intestinal uptake of cholesterol and phytosterols.