Summary

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• This summary is based on the review of one systematic review/meta-analysis. ^[1]
• Maralixibat (Livmarli) demonstrated significant effectiveness in reducing pruritus in patients with Alagille syndrome, indicated by a 1.8-point reduction in the ItchRO scores. Serum bile acid (sBAs) levels were also reduced by 75.8 µmol/L, reflecting improved bile acid regulation.
• The drug improved patient-reported outcomes, with an 11.4-point improvement on the Multidimensional Fatigue Scale and an 8.3-point increase on the Pediatric Quality of Life (QoL) Scale, indicating reduced fatigue and enhanced quality of life in pediatric patients.
• No significant differences in effectiveness were reported among different population subgroups within the study, which focused on pediatric patients with Alagille syndrome.
• Maralixibat (Livmarli) was associated with an increase in alanine aminotransferase (ALT) levels by 40 U/L, raising potential concerns regarding liver enzyme elevation in patients with Alagille syndrome.
• No other significant safety outcomes were reported in the reviewed studies.
• The study focused on pediatric patients with Alagille syndrome (ALGS), a rare inheritable disease, showing that maralixibat significantly improved pruritus, reduced serum bile acids, and enhanced quality of life, making these outcomes particularly relevant for this patient population.