THE BIOCHEMICAL AND NUTRITIONAL EFFECTS OF ODEVIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS AND ALAGILLE SYNDROME.

Karthikeyan, P.; Rajwal, S.; Tremlett, K.; North-Lewis, P.

THE BIOCHEMICAL AND NUTRITIONAL EFFECTS OF ODEVIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS AND ALAGILLE SYNDROME.

All Authors

Karthikeyan, P.

Rajwal, S.

Tremlett, K.

North-Lewis, P.

LTHT Author

Karthikeyan, Palaniswamy
Rajwal, Sanjay
Tremlett, Kirsten
North-Lewis, Penny

LTHT Department

Leeds Children's Hospital
Children's Liver Unit
Children's Services
Paediatric Dietetics
Medicines Management & Pharmacy Services
Clinical Pharmacy

Non Medic

Dietitian
Paediatric Liver Pharmacist

Publication Date

2025

Item Type

Conference Abstract

Abstract

Progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS) are inherited disorders leading to cholestasis and subsequent hepatocellular damage potentially requiring a liver transplant.1 Odevixibat, an oral inhibitor of the ileal bile acid transporter, is being studied for its effectiveness in treating PFIC and ALGS.1 (1This citation refers to the reference listed below and is associated with the work of Deeks (2021) on Odevixibat.) This retrospective case series evaluates patients with PFIC and ALGS treated with odevixibat at a tertiary centre between May 2022- October 2024. Electronic case notes were reviewed at 0,6,12-month time points for nutritional and biochemical parameters (mid-upper arm circumference (MUAC), weight, height, alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), and bilirubin levels). Six patients were on odevixibat. Four patients had PFIC (2 BSEP mutations, 1 MDR3 deficiency, and 1 FIC1 deficiency) and two patients had ALGS. In the PFIC group (all female), the mean age was 4.25 (1- 8 years). Median duration of treatment with odevixibat, was 11.75 months (2-29). The notes were assessed at 0 months for all four patients and 6 months for three patients (1 patient had a liver transplant before 6 months). Two patients underwent liver transplant both in the PFIC group (1 BSEP, 1 MDR3 deficiency) at 2.5 months and 6 months respectively of treatment. In ALGS group (all male) mean age was 2.5 (2-3 years). Median duration of treatment was 14.5 (14-15) months. In the PFIC group, three patients required NG/PEG supplementation. Two patients received a medium-chain triglyceride (MCT) diet, receiving Liquigen and or Heparon Junior. They maintained same height and weight centiles at 6 months, with one requiring nasogastric NG/PEG feeding. One patient reached the 12-month assessment and was on NG/PEG feeding for 4 months and the MCT diet. She demonstrated an improvement in height and weight centiles, rising from <0.4 to the 0.4th percentile, with stable ALT, GGT, and albumin concentrations. Two patients underwent liver transplant, pre liver transplant 1 patient showed an improving height and weight centile whilst the other remained the same. In the ALGS cohort, both patients showed improved nutritional status at 6 and 12 months. Both received Liquigen via NG/PEG. Height centiles remained the same however weight centiles improved. ALT levels slowly increased over time, while GGT levels rose initially then improved by the 12- month mark. Bilirubin levels improved and albumin levels improved by 12 months. In summary, this single centre experience of the use of odevixibat, demonstrated that the ALGS group showed improved biochemical parameters. In the PFIC group, this was not seen as two patients required liver transplantation. In both groups, odevixibat has demonstrated to be a useful adjunct to improve nutritional status.