Moderator of 1 Session
Presenter of 7 Presentations
Roundtable expert discussion (ID 2051)
Implementing Changes for Alagille Syndrome in Practice (ID 2003)
Across the spectrum of disease: Clinical experience from European centres of expertise (ID 2049)
Q&A and Close (ID 2052)
Welcome and introduction (ID 2000)
Panel discussion and Q&A (ID 2004)
H-O007 - EFFECTS OF MARALIXIBAT IN CHILDREN WITH ALAGILLE SYNDROME: A FRENCH REAL-LIFE DATA ANALYSIS. (ID 1923)
Abstract
Objectives and Study
Alagille Syndrome (ALGS) is a rare genetic liver disease often responsible for severe cholestasis, intractable pruritus, and elevated serum bile acid concentration (sBA). Maralixibat is an ileal bile acid transporter (IBAT) inhibitor, available in France in the setting of an Expanded Access Program (EAP) for treatment of pruritus in ALGS patients. We report on clinical and biological data of the French cohort of ALGS patients treated with maralixibat during this EAP.
Methods
Patients were treated with maralixibat at the starting daily dose of 400 µg/kg.d, while maintaining their other antipruritic medications at constant dosage. Clinical data and biological data including sBA were prospectively collected at baseline (BL) and during the follow-up (M3, M6 then every 6 months). Pruritus was assessed by the child and or his parents using a Visual Analog Itching Scale (VAIS) graded from 0 to 10 and by the physician using a Clinical Skin Scratch Scale (CSS) graded from 0 to 4 . A clinically meaningful decrease of pruritus was defined as a ≥1 point decrease in CSS and a ≥ 3 points decrease in VAIS.
Results
15 patients with a median age of 3.6 years (range: 2.8-6.10) were included from January 2021. With treatment, mean VAIS and CSS scores decreased significantly at all time points. A non-significant decrease of means BA was observed. Mean total serum bilirubin levels remained stable throughout the study (Figure). A clinically meaningful decrease of pruritus was observed in 10/15 patients. The median duration of exposure to treatment was 12 months (range: 3-24 months). Maralixibat was definitively stopped in three patients (inefficacy, diarrhea, and non drug-related death), and transiently interrupted in one (diarrhea).
Conclusions
This real-life study confirms the efficacy of maralixibat to decrease pruritus in ALGS patients with acceptable tolerability.