Publications

Abstracts

1. Hansen BE, Vandriel SM, Vig P, Garner W, Li LT, She H, Wang JS, Gilbert MA, Jankowska I, Czubkowski P, Gliwicz-Miedzińska D, Gonzales EM, Jacquemin E, Bouligand J, Spinner NB, Loomes KM, Piccoli DA, D'Antiga L, Nicastro E, Sokal É, Demaret T, Ebel NH, Feinstein JA, Fawaz R, Nastasio S, Lacaille F, Debray D, Arnell H, Fischler B, Siew S, Stormon M, Karpen SJ, Romero R, Kim KM, Baek WY, Hardikar W, Shankar S, Roberts AJ, Evans HM , Jensen MK, Kavan M, Sundaram SS, Chaidez A, Karthikeyan P, Davison S, Sanchez MC, Cavalieri ML, Verkade HJ, Lee WS, Squires JE, Hajinicolaou C, Lertudomphonwanit C, Fischer RT, Larson-Nath C, Mozer-Glassberg Y, Arikan C, Lin HC, Bernabeu JQ, Alam S, Kelly D, Carvalho E, Ferreira CT, Indolfi G, Quiros-Tejeira RE, Bulut P, Calvo PL, Önal Z, Valentino PL, Desai DM, Eshun J, Rogalidou M, Dezsőfi A, Wiecek S, Nebbia G, Pinto RB, Wolters VM, Tamara ML, Zizzo AN, Garcia J, Schwarz K, Beretta M, Sandahl TD, Jimenez-Rivera C, Kerkar N, Brecelj J, Mujawar Q, Rock N, Busoms CM, Karnsakul W, Lurz E, Santos-Silva E, Blondet N, Bujanda L, Shah U, Thompson RJ, Kamath BM and The Global ALagille Alliance (GALA) Study Group. Maralixibat-treated patients with Alagille syndrome (ALGS) demonstrate improved event-free survival in a natural history comparison with patients from the GALA database: Application of real-world evidence analytics. Accepted oral presentation to European Society of Paediatric Gasteroenterology, Hepatology and Nutrition (ESPGHAN), June 2022.

 

2. Hansen BE, Vandriel SM, Vig P, Garner W, Li LT, She H, Wang JS, Gilbert MA, Jankowska I, Czubkowski P, Gliwicz-Miedzińska D, Gonzales EM, Jacquemin E, Bouligand J, Spinner NB, Loomes KM, Piccoli DA, D’Antiga L, Nicastro E, Sokal É, Demaret T, Ebel NH, Feinstein JA, Fawaz R, Nastasio S, Lacaille F, Debray D, Arnell H, Fischler B, Siew S, Stormon M, Karpen SJ, Romero R, Kim KM, Baek WY, Hardikar W, Shankar S, Roberts AJ, Evans HM, Jensen MK, Kavan M, Sundaram SS, Chaidez A, Karthikeyan P, Davison S, Sanchez MC, Cavalieri ML, Verkade HJ, Lee WS, Squires JE, Hajinicolaou C, Lertudomphonwanit C, Fischer RT, Larson-Nath C, Mozer-Glassberg Y, Arikan C, Lin HC, Bernabeu JQ, Alam S, Kelly D, Carvalho E, Ferreira CT, Indolfi G, Quiros-Tejeira RE, Bulut P, Calvo PL, Önal Z, Valentino PL, Desai DM, Eshun J, Rogalidou M, Dezsőfi A, Wiecek S, Nebbia G, Pinto RB, Wolters VM, Tamara ML, Zizzo AN, Garcia J, Schwarz K, Beretta M, Sandahl TD, Jimenez-Rivera C, Kerkar N, Brecelj J, Mujawar Q, Rock N, Molera C, Karnsakul W, Lurz E, Santos-Silva E, Blondet N, Bujanda L, Shah U, Thompson RJ, Kamath BM, and The Global ALagille Alliance (GALA) Study Group. Application of real-world evidence analytics: A 6-year event-free survival analysis in Alagille syndrome of the GALA clinical research database and maralixibat treated patients. Hepatology 2021; 74(Suppl. 6):pp.1387A-1389A. Oral presentation - late-breaking. The Liver Meeting Digital Experience (TLMdX), November 2021. Selected as 'Best of The Liver Meeting' in the Pediatric Liver Diseases category.

 

3. Vandriel SM, Li LT, She H, Wang JS, Gilbert MA, Jankowska I, Czubkowski P, Gliwicz D, Gonzale EM, Jacquemin E, Bouligand J, Spinner NB, Loomes KM, Piccoli DA, D'Antiga L, Nicastro E, Sokal E, Demaret T, Lacaille F, Debray D, Siew S, Stormon M, Fawaz R, Nastasio S, Kim KM, Baek WY, Feinstein JA, Ebel NH, Karpen SJ, Romero R, Karthikeyan P, Davison S, Arnell H, Fischler B, Squires JE, Hardikar W, Shankar S, Jensen KM, Kavan M, Larson-Nath C, Roberts AJ, Evans HM, Verkade HJ, Lertudomphonwanit C, Waisbourd-Zinman O, Sundaram SS, Chaidez A, Fischer RT, Lee WS, Lin H, Kelly D, Bulut P, Calvo PL, Bernabeu JQ, Indolfi G, Nebbia G, Valentino PL, Tamara ML, Desai DM, Zizzo AN, Quiros-Tejeira RE, Kerkar N, Schwarz K, Wolters VM, Mujawar Q, Molera C, Sandahl TD, Jimenez-Rivera C, Santos-Silva E, Blondet N, Brecelj J, Banales JM, Sanchez MC, Cavalieri ML, Wiecek S, Karnsakul W, Lurz E, Alam S, Önal Z, Rogalidou M, Arikan C, Garcia J, Thompson RJ, Hansen BE, Kamath BM, The GALA Study Group. Differential prevalence of extrahepatic clinical manifestations in patients with Jagged1 and NOTCH2-associated Alagille syndrome: Results from the International Multicenter GALA Study Group. J Pediatr Gastroenterol Nutr 2021; 72(Suppl. 1):pp.796-799. Oral presentation. 6th World Congress of Paediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN), June 2021. Nominated for The Annual Alex Mowat Prize for the best oral presentation in Hepatology. 

4. Vandriel SM, Li L, She H, Wang JS, Gilbert MA, Spinner NB, Loomes KM, Piccoli DA, D’Antiga L, Nicastro E, Calvo PL, Hardikar W, Shankar S, Fawaz RL, Nastasio S, Bulut P, Jankowska I, Czubkowski P, Gliwicz-Miedzińska D, Sokal E, Demaret T, Siew SM, Stormon M, Lacaille F, Debray D, Kim KM, Baek WY, Feinstein JA, Ebel NH, Karpen SJ, Romero R, Karthikeyan P, Davison S, Arnell H, Fischler B, Squires JE, Verkade HJ, Jensen MK, Kavan M, Roberts AJ, Evans HM, Lertudomphonwanit C, Lee WS, Sundaram SS, Chaidez A, Fischer RT, Mozer-Glassberg Y, Larson-Nath C, Lin H, Bujanda L, Kelly D, Karnsakul W, Quintero-Bernabeu J, Indolfi G, Mujawar Q, Valentino PL, Nebbia G, Quiros-Tejeira RE, Kerkar N, Schwarz K, Wolters VM, Alam S, Jimenez-Rivera C, Santos-Silva E, Brecelj J, Sanchez MC, Cavalieri ML, Desai DM, Önal Z, Legarda-Tamara M, Molera C, Arikan C, Wiecek S, Gonzales EM, Thompson RJ, Hansen BE, Kamath BM, and The GALA Study Group. Phenotypic divergence of JAGGED1 and NOTCH2-associated Alagille Syndrome: Results from the International Multicenter GALA Study Group. Hepatology 2020; 72(Suppl. 1):pp.882A-884A. Poster presentation. American Association of the Study of Liver Diseases Annual Meeting, November 2020. AASLD Presidential Poster of Distinction (top 10% among all scored poster abstracts).

5. Vandriel SM, Li L, She H, Wang JS, Czubkowski P, Jankowska I, Gliwicz D, Piccoli DA, Loomes K, Spinner N, Sokal E, Demaret T, D'Antiga L, Nicastro E, Fawaz R, Nastasio S, Siew S, Stormon M, Arnell H, Fischler B, Kim K, Baek WY, Karpen S, Romero R, Hardikar W, Shankar S, Roberts AJ, Evans H, Sanchez MC, Cavalieri ML, Karthikeyan P, Davison S, Jensen MK, Kavan M, Verkade HJ, Lee WS, Squires JE, Lertudomphonwanit C, Fischer RT, Lin H, Karnsakul W, Kelly D, Quiros-Tejeira RE, Targa C, Carvalho E, Alam S, Banales JM, Indolfi G, Larson-Nath C, Bulut P, Calvo PL, Valentino PL, Arıkan C, Schwarz K, Kanavaki I, Dezsőfi A, Borges-Pinto R, Wiecek S, Mujawar Q, Brecelj J, Kerkar N, Santos-Silva E, Jimenez-Rivera C, Waisbourd-Zinman O, Nebbia G, Gonzales E, Thompson R, Hansen B, Kamath BM, and The GALA Study Group. Clinical Features and Natural History of 1154 Alagille Syndrome Patients: Results from the International Multicenter GALA Study Group. J Hepatology 2020; 73(Suppl. 1):pp.S554-S555. Poster presentation. The Digital International Liver Congress™ 2020, August 2020. EASL Young Investigator Bursary and Poster of Distinction (top 10% among all scored poster abstracts).

6. Vandriel SM, Li L, She H, Wang JS, Piccoli DA, Loomes KM, Spinner N, D'Antiga L, Nicastro E, Jankowska I, Czubkowski P, Gliwicz D, Sokal E, Demaret T, Fawaz R, Nastasio S, Hardikar W, Shankar S, Arnell H, Fischler B, Fischer RT, Alam S, Bulut P, Kim KM, Baek WY, Calvo PL, Valentino PL, Karthikeyan P, Davison S, Siew S, Stormon M, Karpen SJ, Romero R, Roberts AJ, Evans HM, Sanchez MC, Chazarreta M, Jensen MK, Kavan M, Verkade HJ, Lee WS, Squires JE, Lertudomphonwanit C, Lin H, Karnsakul W, Kelly D, Quiros-Tejeira RE, Targa CT, Carvalho E, Banales JM, Indolfi G, Larson-Nath C, Quintero J, Arıkan C, Schwarz K, Kanavaki I, Dezsőfi A, Borges-Pinto R, Wiecek S, Mujawar Q, Brecelj J, Kerkar N, Santos-Silva E, Jimenez-Rivera C, Waisbourd-Zinman O, Nebbia G, Gonzales E, Thompson RJ, Hansen BE, Kamath BM, and The GALA Study Group. Characterization of the NOTCH2-Associated Alagille Syndrome Phenotype: Results from the International Multicenter GALA Study Group. Oral presentation accepted for World Congress of Paediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) 2020. Nominated for The Annual Alex Mowat Prize for the best oral presentation in Hepatology. (Conference cancelled due to COVID-19)

7. Vandriel SM, Li L, She H, Wang JS, Jankowska I, Czubkowski P, Gliwicz D, Piccoli DA, Loomes KM, Spinner N, Sokal E, Demaret T, D'Antiga L, Nicastro E, Fawaz R, Nastasio S, Siew S, Stormon M, Arnell H, Fischler B, Kim KM, Baek WY, Karpen SJ, Romero R, Hardikar W, Shankar S, Roberts AJ, Evans HM, Sanchez MC, Cavalieri ML, Karthikeyan P, Davison S, Jensen MK, Kavan M, Verkade HJ, Lee WS, Squires JE, Lertudomphonwanit C, Fischer RT, Lin H, Karnsakul W, Kelly D, Quiros-Tejeira RE, Ferreira CT, Carvalho E, Alam S, Banales JM, Indolfi G, Larson-Nath C, Bulut P, Calvo PL, Valentino PL, Arıkan C, Schwarz K, Kanavaki I, Dezsőfi A, Borges-Pinto R, Wiecek S, Mujawar Q, Brecelj J, Kerkar N, Santos-Silva E, Jimenez-Rivera C, Waisbourd-Zinman O, Nebbia G, Gonzales E, Thompson RJ, Hansen BE, Kamath BM, and The GALA Study Group. Clinical Features and Natural History of 1154 Alagille Syndrome Patients: Results from the International Multicenter GALA Study Group. Oral presentation accepted for World Congress of Paediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) 2020. Nominated for The Annual Alex Mowat Prize for the best oral presentation in Hepatology. (Conference cancelled due to COVID-19)

 

8. Vandriel S, Wang JS, Li L, Piccoli DA, Loomes KM, Sokal EM, Demaret T, Arnell H, Fischler B, Fawaz RL, Nastasio S, Hardikar W, Shankar S, Lee WS, Lertudomphonwanit C, Karnsakul W, Karpen SJ, Fischer RT, Quiros RE, Melere M, Bujanda L, Indolfi G, Nicastro E, Calvo PL, Valentino PL, Schwarz KB, Kanavaki I, Dezsőfi A, Mujawar Q, Pinto RB, Brecelj J, Kerkar N, Sanchez MC, Karthikeyan P, Santos-Silva E, Jimenez-Rivera C, Mozer-Glassberg Y, Alam S, Hansen BE, Kamath BM, and The GALA Study Group. Clinical features and outcomes in an international cohort of 731 Alagille syndrome patients from 19 countries. Hepatology 2019; 70(Suppl. 1):pp.55A-56A. Oral presentation. American Association of the Study of Liver Diseases Annual Meeting, Boston, USA, November 2019. Selected as 'Best of The Liver Meeting' in the Pediatric Liver Diseases category.

 

Presentations

Presenter - Shannon M. Vandriel

The Global ALagille Alliance (GALA) Study: 1600 children with ALGS and counting

Discovering New Paths on The Alagille Trail, 9th International Symposium and Scientific Meetings on Alagille Syndrome. July 17, 2021.

 

Presenter - Dr. Binita M. Kamath

Looking Beyond the Hepatic Phenotype: Lessons from the Alagille Consortium (GALA)

6th World Congress of Paediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN). June 2-5, 2021.

Presenter - Dr. Binita M. Kamath

The Global ALagille Alliance Study - Looking Beyond the Liver - Study Overview

Alagille Syndrome Alliance Virtual Roundtable Meeting. September 17-18, 2020.

Presenter - Shannon M. Vandriel

Clinical Features and Natural History of 1154 Alagille Syndrome Patients: Results from the International Multicenter GALA Study Group

Alagille Syndrome Alliance Virtual Roundtable Meeting. September 17-18, 2020.

Presenter - Dr. Binita M. Kamath

The Global ALagille Alliance Study - Looking Beyond the Liver

8th International Symposium and Scientific Meeting on Alagille Syndrome. June 20-23, 2019.