A new study has found that patients aged 16 years or older with Alagille syndrome (ALGS) who initiate treatment with maralixibat and who survive to adulthood with their native liver have generally good outcomes.
The study results were presented at the European Association for the Study of the Liver (EASL) Congress this June in Vienna, Austria.
“Previous data in ALGS has been largely focused on pediatric populations; however, 24-40.3% of patients with ALGS reach 18 years of age with their native liver and may require treatment for cholestasis and pruritus,” the authors wrote. “This analysis reported, for the first time, the efficacy and safety of maralixibat in adult patients with ALGS; ≥16 years transitioning to adult care and participants aged >16 years who initiated treatment in the ALGS clinical program.”
ALGS is a rare genetic disorder affecting multiple organ systems and is characterized by the accumulation of bile in the liver and hepatic damage due to a lack of intrahepatic bile ducts. Symptoms include cholestasis, pruritus, xanthomas, and progressive liver disease.
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The research team assessed the efficacy and safety of maralixibat in a cohort of 14 patients with ALGS, 11 of whom initiated maralixibat treatment before 16 years of age and the remainder after 16 years of age.
The results revealed that patients treated with maralixibat as children had significant improvements in pruritus and serum bile acid levels that were maintained into adulthood, and that the treatment was generally well tolerated. Treatment-related adverse events included diarrhea and abdominal pain, but there were no severe adverse events and no patient discontinued the treatment due to adverse events.
The authors concluded that maralixibat has the potential to positively impact the treatment for adults with ALGS who survive to adulthood with their native liver.
Hirschfield G, Mogul D, Baek M, et al. Impact of maralixibat on cholestatic pruritus in adults aged 16 years and older with Alagille syndrome. Presented at the: European Association for the Study of the Liver (EASL) Congress, Vienna, Austria; June 21-24, 2023.