Publications by authors named "Maria Lorena Cavalieri"
Background And Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.
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- Alagille syndrome (ALGS) is a complex disorder primarily affecting the liver, and this study investigated the long-term liver health of affected children through a large, international sample.
- The study included 1,433 children diagnosed with ALGS across 67 medical centers in 29 countries, revealing that only about 40% reached adulthood with their native liver intact.
- Elevated total bilirubin levels in infants are linked to significantly increased risks of developing severe liver-related problems, which can help doctors in making treatment decisions and evaluating therapies for ALGS patients.
In January 2015, a 20-month-old child and her family took part in recreational activities at Carrasco and Malvín beaches (Montevideo, Uruguay). An intense harmful algae bloom (HAB) was developing along the coast at that time. A few hours after the last recreational exposure episode, the family suffered gastrointestinal symptoms which were self-limited except in the child's case, who was admitted to hospital in Uruguay with diarrhea, vomiting, fatigue, and jaundice.
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