# Treatment of intractable pruritus with maralixibat in patients with Alagille syndrome before and after reversal of biliary diversion

## TL;DR

Maralixibat effectively treats severe itching in Alagille syndrome patients, even after a surgical procedure to reverse bile diversion.

## Contribution

Maralixibat is presented as a safe and effective treatment for cholestatic pruritus in ALGS patients before and after surgical biliary diversion reversal.

## Key findings

- Maralixibat was well-tolerated and improved cholestatic pruritus in two ALGS patients.
- Treatment with maralixibat was effective both before and after reversal of surgical biliary diversion.
- Maralixibat may serve as a pharmacological alternative to surgical options in ALGS patients.

## Abstract

Alagille syndrome (ALGS) is a rare, cholestatic, multisystemic disorder characterized by bile duct paucity. Cholestatic pruritus is a common, and often severe, symptom of ALGS and is the leading cause of liver transplantation. The treatment of cholestatic pruritus is challenging and involves medical and surgical options, such as surgical biliary diversion (SBD) for refractory cases. However, SBD is associated with medical/lifestyle challenges. Maralixibat, an ileal bile acid transporter inhibitor, is a recently approved treatment for cholestatic pruritus in patients with ALGS and is used as part of standard of care. We present cases of two patients with ALGS who initiated treatment with maralixibat: one before, with continuation after, reversal of SBD, and one after SBD reversal. In both cases, treatment with maralixibat was well‐tolerated and demonstrated marked improvements in cholestatic pruritus. This suggests that maralixibat is a pharmacological alternative for patients who would like to pursue reversal of SBD.

Cholestatic pruritus is the most common and severe symptom of Alagille syndrome (ALGS).Treatment for cholestatic pruritus involves medical and surgical options.However, historical medical treatments are used off‐label and are ineffective at controlling symptoms, and surgical options, such as surgical biliary diversion (SBD), are associated with medical and lifestyle challenges.Maralixibat was approved for the treatment of cholestatic pruritus in patients with ALGS ≥3 months of age in the United States in 2021 and has since become the standard of care.

Cholestatic pruritus is the most common and severe symptom of Alagille syndrome (ALGS).

Treatment for cholestatic pruritus involves medical and surgical options.

However, historical medical treatments are used off‐label and are ineffective at controlling symptoms, and surgical options, such as surgical biliary diversion (SBD), are associated with medical and lifestyle challenges.

Maralixibat was approved for the treatment of cholestatic pruritus in patients with ALGS ≥3 months of age in the United States in 2021 and has since become the standard of care.

Maralixibat treatment was safe and demonstrated improvements in cholestatic pruritus in a patient with ALGS with an existing SBD and in a patient with ALGS who had SBD reversal.Treatment with maralixibat may be utilized in patients with ALGS who have undergone SBD.

Maralixibat treatment was safe and demonstrated improvements in cholestatic pruritus in a patient with ALGS with an existing SBD and in a patient with ALGS who had SBD reversal.

Treatment with maralixibat may be utilized in patients with ALGS who have undergone SBD.