Efficacy of a bile acid transport inhibitor in the treatment of cholestatic pruritus in children with Alagille syndrome: a single center experience

Alagille syndrome is a rare hereditary disease characterized by cholestasis syndrome due to bile duct hypoplasia. One of the frequent manifestations of cholestasis syndrome in Alagille syndrome is severe pruritus, which significantly reduces the quality of life. Existing standard approaches to antipruritic therapy, including ursodeoxycholic acid, bile acid sequestrants, rifampicin, anxiolytics, etc., is not effective enough. Currently, bile acid transport inhibitors (IBAT), such as maralixibat, which prevents enterohepatic bile acids recirculation and, as a result, reduces it’s blood concentration, which leads to a decrease of pruritus, are actively studied and introduced into therapy.

Methods: the study included 12 patients with Alagille syndrome (age 53.83±48.23 months). All children had severe pruritus and > four-fold increase of serum bile acid. Cholestatic pruritus was assessed by patients’ parents using the IthRo scale (0–4 points).

Results: One patient did not continue participation in the study after the drug prescription, 11 patients received maralixibat for ≥12 weeks, 7 for > 48 weeks. By week 12,86%, had a decrease of blood serum bile acid >50%, and by week 48 >three-fold decrease in blood serum bile acid concentrations. Pruritus severity decreased by ≥2 points by week 4 of therapy in 9(82%) patients. By week 48, pruritus intensity did not exceed 1.17 points (95% CI 0.38–1.96) in 5 of 7 patients. One patient, failed to achieve the reduction in pruritus, but double dose of the maralixibat at week 36 resulted in a reduction in pruritus score to 1 point on the ItchRo scale by week 48.

Conclusions: this study demonstrates the high efficacy of bile acid transport inhibitor in the treatment of cholestatic pruritus in patients with Alagille syndrome.

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