Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA

• Main Authors: Hansen, Bettina E., Vandriel, Shannon M., Vig, Pamela, Garner, Will, Mogul, Douglas B., Loomes, Kathleen M., Piccoli, David A., Rand, Elizabeth B., Jankowska, Irena, Czubkowski, Piotr, Gliwicz-Miedzinska, Dorota, Gonzales, Emmanuel M., Jacquemin, Emmanuel, Bouligand, Jerome, D'Antiga, Lorenzo, Nicastro, Emanuele, Arnell, Henrik, Fischler, Bjorn, Sokal, Etienne, Demaret, Tanguy, Siew, Susan, Stormon, Michael, Karpen, Saul J., Romero, Rene, Ebel, Noelle H., Feinstein, Jeffrey A., Roberts, Amin J., Evans, Helen M., Sundaram, Shikha S., Chaidez, Alexander, Hardikar, Winita, Shankar, Sahana, Fischer, Ryan T., Lacaille, Florence, Debray, Dominique, Lin, Henry C., Jensen, M. Kyle, Jaramillo, Catalina, Karthikeyan, Palaniswamy, Indolfi, Giuseppe, Verkade, Henkjan J., Larson-Nath, Catherine, Quiros-Tejeira, Ruben E., Valentino, Pamela L., Rogalidou, Maria, Dezsofi, Antal, Squires, James E., Schwarz, Kathleen, Calvo, Pier Luigi, Bernabeu, Jesus Quintero, Zizzo, Andreanne N., Nebbia, Gabriella, Bulut, Pinar, Santos-Silva, Ermelinda, Fawaz, Rima, Nastasio, Silvia, Karnsakul, Wikrom, Tamara, Maria Legarda, Busoms, Cristina Molera, Kelly, Deirdre A., Sandahl, Thomas Damgaard, Jimenez-Rivera, Carolina, Banales, Jesus M., Mujawar, Quais, Li, Li-Ting, She, Huiyu, Wang, Jian-She, Kim, Kyung Mo, Oh, Seak Hee, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Lee, Way Seah, Hajinicolaou, Christina, Lertudomphonwanit, Chatmanee, Waisbourd-Zinman, Orith, Arikan, Cigdem, Alam, Seema, Carvalho, Elisa, Melere, Melina, Eshun, John, Onal, Zerrin, Desai, Dev M., Wiecek, Sabina, Pinto, Raquel Borges, Wolters, Victorien M., Garcia, Jennifer, Beretta, Marisa, Kerkar, Nanda, Brecelj, Jernej, Rock, Nathalie, Lurz, Eberhard, Blondet, Niviann, Shah, Uzma, Thompson, Richard J., Kamath, Binita M., GALA, Global Alagille Alliance
• Format: Article
• Published: Lippincott, Williams & Wilkins 2024
• Subjects: R Medicine (General)
• Online Access: http://eprints.um.edu.my/47136/; https://doi.org/10.1097/HEP.0000000000000727
• Institution: Universiti Malaya

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...