Severe cholestasis and pruritus are often the most debilitating manifestations of ALGS and are a frequent indication for liver transplantation. Ileal bile acid transport inhibitors are a new class of medications that have recently been approved for treatment of cholestatic pruritus in ALGS. Orally administered and minimally absorbed, these drugs result in a medical interruption of the enterohepatic circulation, increasing excretion of bile acids in the stool. Maralixibat is currently FDA approved for treatment of children older than age three months and odevixibat for children older than age 12 months. Additional studies have shown that clinical response to maralixibat treatment is associated with improved event-free survival and quality of life. Additional therapies can include vitamin supplementation.
A multidisciplinary approach is recommended for patient care including hepatology, cardiology, and ophthalmology for the major clinical features, and in some cases nephrology.
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