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Long-Term Cholic Acid Therapy in Zellweger Spectrum Disorders.
Case Reports in Gastroenterology 2018 May
Zellweger spectrum disorders (ZSDs), a subgroup of peroxisomal biogenesis disorders, have a generalized defect in peroxisome function. Liver disease in ZSDs has been linked to accumulation of C27 -bile acid intermediates due to the lack of peroxisomal β-oxidation of these intermediates to form primary C24 -bile acids. Oral treatment with primary bile acid, cholic acid (CA), inhibits formation of hepatotoxic C27 -bile acids by restoring normal physiologic feedback inhibition on bile acid synthesis. We present the long-term CA treatment and liver-related outcomes for 3 pediatric patients with ZSDs who have received CA treatment for ≥15 years. Ongoing CA treatment was associated with stabilized liver function, as shown by serum biochemistries and liver histopathology, and no treatment-related adverse effects were observed. All 3 patients have attended regular school with classroom accommodations and attained a good quality of life. Our patient outcomes suggest that early and ongoing CA therapy may sustain liver function in patients with ZSDs.
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