Long-Term Cholic Acid Therapy in Zellweger Spectrum Disorders

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.Case Rep Gastroenterol 2018;12:360 –372
Source: Case Reports in Gastroenterology - Category: Gastroenterology Source Type: research