Maralixibat improves xanthomas and hypercholesterolemia in children with Alagille syndrome, according to a poster presentation at the European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN 2023) annual meeting.
Xanthomas represent a unique problem among patients with Alagille syndrome, affecting around one-quarter of the patient population. However, xanthomas associated with Alagille syndrome are poorly characterized and there remains no approved therapeutics to treat this condition.
Maralixibat is an existing drug often used to treat cholestatic pruritus in this patient population. The researchers sought to evaluate the merits of maralixibat in alleviating symptoms of xanthomas in patients with Alagille syndrome. To do so, they evaluated the baseline characteristics of xanthomas in patients with Alagille syndrome and tracked whether maralixibat use resulted in their reduction.
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The authors obtained data from the Maralixibat Alagille Syndrome program. They assessed xanthomas using the Clinician Xanthomas Scale (CXS); a response was defined as a 1 point reduction or greater in the CXS. They also tracked cases in which the CXS indicated unchanged/worsening xanthomas in patients with Alagille syndrome.
Read more about Alagille syndrome etiology
Among the 63 patients with Alagille syndrome recruited, 27 had xanthomas at baseline. A higher CXS was associated with poorer liver enzyme parameters: higher total/direct bilirubin, higher total cholesterol and low-density lipoprotein, higher serum bile acids, and lower high-density lipoprotein. A high CXS at baseline was also associated with poorer physical functioning.
Using maralixibat treatment, the proportion of patients with no xanthomas, meaning a CXS of 0, increased from 60% to 86% over a period of 96 weeks. Within the same period, the proportion of patients with moderate and severe xanthomas decreased from 33% to 9% and 9% to 6%, respectively. There was also a significant decrease in total cholesterol and an increase in high-density lipoprotein. Participants who were assessed for quality of life reported an increase in this domain after using maralixibat.
“Xanthoma reduction was associated with improved quality of life and total cholesterol,” the authors concluded.
Reference
Hoskins B, Mogul D, Aguilar R, Karnsakul W. Maralixibat improves xanthomas and hypercholesterolemia in children with Alagille syndrome: an integrated analysis from two clinical trials. Presented at: European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN 2023) annual meeting, Vienna, Austria; May 17-20, 2023.
