Treatment of Pruritus With Maralixibat in Early-Stage Chronic Graft Dysfunction of a Child With Alagille Syndrome.

Alagille syndrome (ALGS) is a rare, autosomal dominant disorder characterized by cholestasis and progressive liver disease that can lead to liver transplantation or death. Liver transplant recipients with ALGS can face chronic graft dysfunction and refractory pruritus that significantly impairs quality of life. We present the case of a 16-year-old male patient with ALGS who developed severe pruritus 13 years after receiving a liver transplant. Despite conventional therapies, his symptoms persisted, accompanied by elevated serum bile acids. Treatment with maralixibat, an ileal bile acid transporter inhibitor, resulted in complete resolution of pruritus within two months as well as a substantial reduction in serum bile acids. No adverse events were reported. This case offers real-world evidence of the effectiveness of maralixibat in managing cholestatic pruritus in a patient with ALGS and early-stage chronic graft dysfunction, highlighting its potential as a therapeutic option in this challenging patient population.