Ileal bile acid transport (IBAT) blockers improved clinical outcomes across a variety of parameters for patients with Alagille syndrome (ALGS), researchers reported in the Journal of Clinical Medicine. 

Chronic cholestasis is a main characteristic of ALGS, but ALGS is often described as a multisystemic disease because it adversely affects other organs, such as the heart, kidney, and lungs. Chronic cholestasis can often lead to severe pruritus, one of the main reasons patients seek medical attention. 

There are a number of other medications used to treat severe pruritus, including ursodeoxycholic acid, cholestyramine, naltrexone, and rifampicin. Nevertheless, studies suggest they have no major impact on severe pruritus. 

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The research team thus decided to look into the use of ileal bile acid transporter blockers for cholestatic liver disease. They conducted a literature review assessing adult patients with ALGS who were using the therapy for the purpose mentioned. They collected data on serum bile acids, alanine transaminase (ALT), and total bilirubin. In addition, they also collected data for a number of questionnaires, including the Itch Scale score, Multidimensional Fatigue Scale score, and Pediatric Quality of Life score.

Read more about Alagille syndrome etiology 

Four studies were included in their final analysis. The researchers discovered that liver function tests did not show any improvements. Nonetheless, itch severity as determined by the Itch Scale score was significantly reduced, by 1.8 points, and there was a significant reduction of -75.8 μmol/L in serum bile acids. In addition, the Multidimensional Fatigue Scale showed an improvement of 11.4 points, while the Pediatric Quality of Life score improved by 8.3 points.

The most common adverse events reported in this study were gastrointestinal, especially abdominal pain and diarrhea. 

“In this systematic review and meta-analysis, 94 patients with Alagille syndrome from across four trials were included,” the authors of the study wrote. “Further trials may consider spacing the dosing frequency to reduce the gastrointestinal side effects while still retaining the positive effects on pruritus and [serum bile acids].” 


Muntaha HST, Munir M, Sajid SH, et al. Ileal bile acid transporter blockers for cholestatic liver disease in pediatric patients with Alagille syndrome: a systematic review and meta-analysisJ Clin Med. 2022;11(24):7526. doi:10.3390/jcm11247526