Christine Clemson, Velichka Valcheva, Ekkehard Sturm et al. Native Liver Survival in Odevixibat sBA Responders: Data From the PEDFIC Studies in Patients With Progressive Familial Intrahepatic Cholestasis. Abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting. November 4–8, 2022; Washington, DC, USA.
Lorenzo D’Antiga, Hasan Özen, Elke Lainka et al. Hepatic Impairment Classifications at Baseline in Responders to Odevixibat Therapy in Children With Progressive Familial Intrahepatic Cholestasis. Abstract accepted at the 50th Annual Meeting of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN). October 13–15, 2022; Orlando, FL, USA.
Chad Gwaltney, Stephanie Bean, Meredith Venerus et al. Development of the Patient- and Observer-reported PRUCISION Instruments to Assess Pruritus and Sleep Disturbance in Pediatric Patients With Cholestatic Liver Diseases. Adv Ther. 2022:39;5126-43
Henkjan J. Verkade, Folkert Kuipers, Quanhong Ni, Velichka Valcheva . Total, Primary, and Secondary Serum Bile Acid Concentrations in Patients With Progressive Familial Intrahepatic Cholestasis With Serum Bile Acid Response or Not With Odevixibat Treatment: Assessing the Contribution of Ursodeoxycholic Acid Concentration. Gut 2022;71(suppl 3):A76.
Chad Gwaltney, Cristina Ivanescu, Lisa Karlsson et al. Validation of the PRUCISION Instruments in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis. Adv Ther. 2022:39;5105-25.
Henkjan J. Verkade, Folkert Kuipers, Quanhong Ni, Velichka Valcheva . Total, Primary, and Secondary Serum Bile Acid Concentrations in Patients With Progressive Familial Intrahepatic Cholestasis With Serum Bile Acid Response or Not With Odevixibat Treatment: Assessing the Contribution of Ursodeoxycholic Acid Concentration. Poster presented at the Annual Meeting of the British Association for the Study of the Liver (BASL). September 20–23, 2022; Leeds, UK.
Lorenzo D'Antiga, Emmanuel Gonzalès, Etienne Sokal et al. Serum Bile Acid Levels, Pruritus Scores, and Growth Over Time in Odevixibat Responders: Pooled Data From the PEDFIC Studies in Patients With Progressive Familial Intrahepatic Cholestasis. Abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting . November 4–8, 2022; Washington, DC, USA.
Prince Korah, Leslie Askonas, Philip Stein. Stability of Odevixibat Oral Capsule Contents in Liquids. Abstract accepted at the 50th Annual Meeting of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN). October 13–15, 2022; Orlando, FL, USA.
Angelo Di Giorgio, Hans Blokzijl, Emmanuel Gonzalès et al. Odevixibat Treatment in Patients With Recurrent Episodic Cholestasis and Biallelic Mutations in ATP8B1: A Retrospective Case Series. Abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting . November 4–8, 2022; Washington, DC, USA.
Kathleen M. Loomes, Emmanuel Gonzalès, Tassos Grammatikopoulos et al. Outcomes in Patients With Progressive Familial Intrahepatic Cholestasis Treated With Odevixibat Who Had Medium or Lower Pruritus Severity at Baseline: Pooled Analysis From the PEDFIC 1 and PEDFIC 2 Studies.
Henkjan J. Verkade, Folkert Kuipers, Quanhong Ni, Velichka Valcheva. Total, Primary, and Secondary Serum Bile Acid Changes and Pruritus Improvement During Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. Gut 2022;71(suppl 3):A76–A77.
Mark Nomden, Henkjan J. Verkade, Folkert Kuipers et al. Odevixibat Treatment in Responsive Patients With Bile Salt Export Pump Deficiency Restores Biliary Bile Acid Secretion, as Indicated by Serum Bile Acid Composition. Latebreaker abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting . November 4–8, 2022; Washington, DC, USA.
Ekkehard Sturm, Buket Dalgic, Florence Lacaille, et al. Outcomes With Odevixibat in Patients With Progressive Familial Intrahepatic Cholestasis by Level of Pruritus Reduction: Pooled Results From the PEDFIC Trials. Abstract accepted at the 50th Annual Meeting of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN). October 13–15, 2022; Orlando, FL, USA.
Henkjan J. Verkade, Folkert Kuipers, Quanhong Ni, Velichka Valcheva . Total, Primary, and Secondary Serum Bile Acid Changes and Pruritus Improvement During Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. Poster presented at the Annual Meeting of the British Association for the Study of the Liver (BASL). September 20–23, 2022; Leeds, UK.
Nadia Ovchinsky, Madeleine Aumar, Alastair Baker et al. Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome: Top-line Results From ASSERT, a Phase 3, Double-Blind, Randomized, Placebo-Controlled Study. Latebreaker abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting . November 4–8, 2022; Washington, DC, USA.
Richard J. Thompson, Ekkehard Sturm, Tassos Grammatikopoulos et al. Long-term Efficacy and Safety of Odevixibat in Patients With Progressive Familial Intrahepatic Cholestasis: Results With 96 Weeks or More of Treatment. Abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting . November 4–8, 2022; Washington, DC, USA.
Henkjan J. Verkade, Richard J. Thompson, Ekkehard Sturm et al. Effect of Odevixibat in Patients With Progressive Familial Intrahepatic Cholestasis Type 2 With at Least 1 Severe Mutation (BSEP3 Compound Heterozygotes): Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies. Abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting . November 4–8, 2022; Washington, DC, USA.
Georg-Friedrich Vogel, Elke Lainka, Simone Kathemann et al. Odevixibat Therapy in Patients With FIC1-Deficient Progressive Familial Intrahepatic Cholestasis and Diarrhea Following Liver Transplantation That Impacted Daily Activities: A Retrospective Case Series. Abstract accepted at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD): The Liver Meeting . November 4–8, 2022; Washington, DC, USA.
Saul J. Karpen, Greg Wright, Phillip Stein, Patrick Horn. The BOLD Study: A RCT to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BA). Abstract accepted at the 2nd International and Interdisciplinary Congress on Biliary Atresia and Related Diseases (BARD). June 17–18, 2022; Bruges, Belgium.
Richard J. Thompson, Lorenzo D’Antiga, Emmanuel Gonzalès, et al. Odevixibat Therapy Improves Clinically Meaningful Endpoints in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. Spanish-translated abstract accepted at the 28th Meeting of the Sociedad Española de Gastroenterología, Hepatología y Nutrición Pediátrica (SEGHNP). June 16–18, 2022: Palma de Mallorca, Spain.
Emmanuel Gonzalès, Patrick McKiernan, Buket Dalgic et al. Safety and Efficacy of Treatment With Odevixibat in Children With PFIC: Report of the PEDFIC 1 and PEDFIC 2 Studies. French-translated abstract accepted at the 43rd Meeting of the Groupe Francophone d'Hépatologie-Gastroentérologie et Nutrition Pédiatriques (GFHGNP). March 31–April 2, 2022; Bordeaux, France. (Adapted from accepted abstracts from AASLD 2021 by Gonzalès et al. and by McKiernan et al. and from NASPGHAN 2021 by Dalgic et al.)
Tassos Grammatikopoulos, Elke Lainka, Reha Artan et al. Efficacy and Safety of Odevixibat Therapy With Concomitant UDCA or Rifampicin in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. Abstract accepted at die Jahrestagung der Gesellschaft für Pädiatrische Gastroenterologie und Ernährung (GPGE). May 11–14, 2022; Rostock, Germany.
Christine Clemson, Quanhong Ni, Patrick Horn. Correlations Between Patient- and Caregiver-reported Pruritus From PEDFIC 1: Evaluation of PRUCISION Data in Patients With Progressive Familial Intrahepatic Cholestasis. Value Health. 2022;25(7 suppl 1):S322.
Buket Dalgic, Henkjan J. Verkade, Alain Lachaux et al. Relationships Between Decreases in Serum Bile Acids, Pruritus, and Sleep Disturbance Scores With Up to 72 Weeks of Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. Gastroenterology. 2022;162(7 suppl):S1295–S1296.
Chad Gwaltney, Stephanie Bean, Meredith Venerus et al. Development of the Patient- and Observer-reported PRUCISION Instruments to Assess Pruritus and Sleep Disturbance in Pediatric Patients With Cholestatic Liver Diseases. Adv Ther. 2022, in press.
Tassos Grammatikopoulos, Buket Dalgic, Girish Gupte et al. Efficacy and Safety Outcomes in Patients With Progressive Familial Intrahepatic Cholestasis Who Had an Odevixibat Dose Escalation: Pooled Results From the PEDFIC 1 and PEDFIC 2 Studies. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):645–6.
Lorenzo D’Antiga, Girish Gupte, Richard J. Thompson et al. Changes in Hepatic Parameters, Growth, Sleep, and Biochemical Markers With Odevixibat Treatment Across Patients With Various Types of Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2022;77(suppl 1):S518–9.
Henkjan J. Verkade, on behalf of study co-authors. Effects on Serum Bile Acids, Pruritus, and Safety With up to 72 Weeks of Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Oral presentation at the PFIC Network Scientific Conference. April 29–30, 2022; Pittsburgh, PA, USA. (Adapted from original presentations at AASLD 2021 by Loomes et al. and by McKiernan et al. and at NASPGHAN 2021 by Kamath et al.)
Henkjan J. Verkade, Folkert Kuipers, Quanhong N et al. Total, Primary, and Secondary Serum Bile Acid Concentrations in Patients With Progressive Familial Intrahepatic Cholestasis With Serum Bile Acid Response or Not With Odevixibat Treatment: Assessing the Contribution of Ursodeoxycholic Acid Concentration. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):847–8.
Girish Gupte, Piotr Czubkowski, Florence Lacaille et al. Disease Burden and Natural History of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical Characteristics Among Odevixibat-Treated Patients in the Phase 3 PEDFIC Studies. Gastroenterology. 2022;162(7 suppl):S1295.
Girish Gupte, Richard J. Thompson, Lorenzo D’Antiga et al. Analysis of Quality of Life, Hepatic Biochemical Markers, and Sleep in Patients With Progressive Familial Intrahepatic Cholestasis Who Had a Pruritus Response With Odevixibat Treatment. J Hepatol. 2022;77(suppl 1):S519.
Patrick McKiernan, Ekkehard Sturm, Binita M. Kamath et al. Hepatic Parameters, Growth, and Sleep With Responders and Nonresponders to Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Abstract accepted at the Annual Meeting of the British Society of Pediatric Gastroenterology, Hepatology, and Nutrition (BSPGHAN). April 25–27, 2022; Birmingham, UK.
Binita M. Kamath, Richard J. Thompson, Ulrich Baumann et al. Efficacy And Safety Of Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion. Abstract accepted at die Jahrestagung der Gesellschaft für Pädiatrische Gastroenterologie und Ernährung (GPGE). May 11–14, 2022; Rostock, Germany.
Chad Gwaltney, Cristina Ivanescu, Lisa Karlsson et al. Validation of the PRUCISION Instruments in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis. Adv Ther. 2022, in press.
Richard J. Thompson, Henrik Arnell, Reha Artan, et al. Odevixibat Treatment in Progressive Familial Intrahepatic Cholestasis: A Randomised, Placebo-controlled, Phase 3 Trial. Lancet Gastroenterol Hepatol. 2022;S2468-1253(22)00093-0 (online ahead of print).
Lorenzo D’Antiga, Girish Gupte, Richard J. Thompson et al. Changes in Hepatic Parameters, Growth, Sleep, and Biochemical Markers With Odevixibat Treatment Across Patients With Various Types of Progressive Familial Intrahepatic Cholestasis. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):664–5.
Binita M. Kamath, Richard J. Thompson, Quanhong Ni et al. Efficacy and Safety of Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion. Gastroenterology. 2022;162(7 suppl):S1296.
Cara L. Mack, Chad Gwaltney, Quanhong Ni et al. Improvements in Quality of Life in Odevixibat Responders and Nonresponders: An Analysis of Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies. J Hepatol. 2022;77(suppl 1):S517.
Hasan Özen, Etienne Sokal, Florence Lacaille et al. Efficacy and Safety Outcomes With Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis Due to Deficiencies in Multidrug Resistance Protein 3 (PFIC Type 3) or Myosin 5B (PFIC Type 6). Abstract accepted at the Annual Meeting of the British Society of Pediatric Gastroenterology, Hepatology, and Nutrition (BSPGHAN). April 25–27, 2022; Birmingham, UK.
Richard J. Thompson, Reha Artan, Ulrich Baumann, et al. Odevixibat Effects on Cholestasis-Related Parameters: Analysis of Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Gastroenterology. 2022;162(7 suppl):S1296–S1297.
Richard J. Thompson, Emmanuel Gonzalès, Reha Artan et al. Efficacy and Safety of Odevixibat Over 72 Weeks of Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2022;77(suppl 1):S521.
Richard J. Thompson, Reha Artan, Lorenzo D’Antiga et al. Long-term Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor in Children With Progressive Familial Intrahepatic Cholestasis: Interim Results From PEDFIC 2, an Open-label Phase 3 Trial. Abstract accepted at the Annual Meeting of the British Society of Pediatric Gastroenterology, Hepatology, and Nutrition (BSPGHAN). April 27–29, 2021; Virtual.
Johanna Ohlendorf, Imeke Goldschmidt, Norman Junge et al. Ileal Bile Acid Transporter Inhibition Reduces Post-transplant Diarrhea and Growth Failure in FIC1 Disease–A Case Report. Children (Basel). 2022;9(5):669. (Externally written)
Girish Gupte, Richard J. Thompson, Lorenzo D’Antiga et al. Analysis of Quality of Life, Hepatic Biochemical Markers, and Sleep in Patients With Progressive Familial Intrahepatic Cholestasis Who Had a Pruritus Response With Odevixibat Treatment. J Pediatr Gastroenterol Nutr. 2022;74(S2 suppl 2):647–9.
Henkjan J. Verkade, Folkert Kuipers, Quanhong Ni et al. Total, Primary, and Secondary Serum Bile Acid Changes and Pruritus Improvement During Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2022;77(suppl 1):S517–8.
Richard J. Thompson, Pier Luigi Calvo, Winita Hardikar, et al. Long-term Treatment With Odevixibat Improves Multiple Sleep Parameters in Patients With Progressive Familial Intrahepatic Cholestasis: A Pooled Responder Analysis From the Phase 3 PEDFIC Studies. Gastroenterology. 2022;162(7 suppl):S1297.
Odevixibat (Bylvay) for Progressive Familial Intrahepatic Cholestasis-Associated Pruritus. Med Lett Drugs Ther. 2022;64(1644):28–29.
Heo, Young-A. Odevixibat in progressive familial intrahepatic cholestasis: a profile of its use. Adis Drug Q&A – Drugs & Therapy Perspectives. 2022.
Richard J. Thompson, Lorenzo D’Antiga, Emmanuel Gonzalès, et al. Odevixibat Therapy Improves Clinically Meaningful Endpoints in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. Gastroenterology. 2022;162(7 suppl):S1296.
Richard J. Thompson, Lise Kjems, Winita Hardikar et al. Improved Quality of Life in Children With Progressive Familial Intrahepatic Cholestasis Following 24 Weeks of Treatment With Odevixibat, an Ileal Bile Acid Transporter Inhibitor: Results From the Phase 3 PEDFIC 1 Study. Value Health. 2021;24(5 suppl 1):S155.
Richard Thompson, Reha Artan, Ulrich Baumann et al. Odevixibat Effects on Cholestasis-related Parameters: Analysis of Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2021;75(suppl 2):S687.
Emmanuel M. Gonzalès, Richard J. Thompson, Binita Kamath et al. Relationships Between Changes in Autotaxin, Pruritus, and Serum Bile Acids After Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis: Data From a Pooled Analysis. Hepatology. 2021;74(suppl 1):30A–1A.
Emma D. Deeks. Odevixibat: First Approval. Drugs. 2021;81(15):1781–6.
Buket Dalgic, Henkjan Verkade, Alain Lachaux et al. Relationships Between Decreases in Serum Bile Acids, Pruritus, and Sleep Disturbance Scores With Up to 72 Weeks of Odevixibat Treatment in Patients With Progressive Familial Intrahepatic Cholestasis. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S49–50.
Tassos Grammatikopoulos, Carrie Thompson, Nisreen Soufi et al. Long-term Safety and Tolerability of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis: Results From the Phase 3 PEDFIC 1 and PEDFIC 2 Studies. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):781–2.
Tassos Grammatikopoulos, Elke Lainka, Reha Artan et al. Efficacy and Safety of Odevixibat Therapy With Concomitant UDCA or Rifampicin in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S53–4.
Chad Gwaltney, Cristina Ivanescu, Lisa Karlsson et al. Validation of the PRUCISION Caregiver-reported Pruritus Measure Using Data From the Phase 3, Randomised PEDFIC 1 Trial in Paediatric Patients With Progressive Familial Intrahepatic Cholestasis. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):814–15.
Richard Thompson, Pier Luigi Calvo, Winita Hardikar et al. Long-term Treatment With Odevixibat Improves Multiple Sleep Parameters in Patients With Progressive Familial Intrahepatic Cholestasis: A Pooled Responder Analysis From the Phase 3 PEDFIC Studies. J Hepatol. 2021;75(suppl 2):S683–4.
Kathleen M. Loomes, Henkjan J. Verkade, Richard J. Thompson et al. Effects on Serum Bile Acids, Pruritus, and Safety With Up to 72 Weeks of Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Hepatology. 2021;74(suppl 1):1159A–60A.
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille, et al. Effects of Odevixibat on Pruritus and Bile Acids in Children With Cholestatic Liver Disease: Phase 2 Study. Clin Res Hepatol Gastroenterol. 2021;45(5):101751.
Richard Thompson, Lorenzo D’Antiga, Emmanuel Gonzalès et al. Odevixibat Therapy Improves Clinically Meaningful Endpoints in Children With Progressive Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and PEDFIC 2 Trials. J Hepatol. 2021;75(suppl 2):S688.
Patrick James McKiernan, Ekkehard Sturm, Binita Kamath et al. Hepatic Parameters, Growth, and Sleep With Responders and Nonresponders to Odevixibat Treatment: Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With Progressive Familial Intrahepatic Cholestasis. Hepatology. 2021;74(suppl 1):1164A.
Leonardo Ruiz-Casas, Sonia O’Hara, Claudia Mighiu, et al. Burden of Illness of Progressive Familial Intrahepatic Cholestasis in the US, UK, France, and Germany: Study Rationale and Protocol of the PICTURE Study. Expert Rev Pharmacoecon Outcomes Res. 2021;21(2):247–53.
Girish Gupte, Piotr Czubkowski, Florence Lacaille et al. Disease Burden and Natural History of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical Characteristics Among Odevixibat-Treated Patients in the Phase 3 PEDFIC Studies. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S197–8.
Richard J. Thompson, Henkjan J. Verkade, Emmanuel Gonzalès et al. Long-term Treatment Benefits of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis: Improvements in Total Bilirubin, Growth, and Sleep as Secondary and Exploratory Outcome Measures From the Phase 3 PEDFIC 1 and PEDFIC 2 Studies. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):819–20.
Richard Thompson, Lorenzo D’Antiga, Binita M. Kamath et al. Rapid and Durable Effect of Odevixibat on Clinical and Biochemical Parameters of Cholestasis in Children With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2021;75(suppl 2):S279–80.
Hasan Özen, Etienne Sokal, Florence Lacaille, Buket Dalgic, Quanhong Ni, Lise Kjems, Patrick Horn. Efficacy and Safety Outcomes With Odevixibat in Children With Progressive Familial Intrahepatic Cholestasis Due to Deficiencies in Multidrug Resistance Protein 3 (PFIC Type 3) or Myosin 5B (PFIC Type 6). Hepatology. 2021;74(suppl 1):1160A–1A.
Binita Kamath, Richard Thompson, Quanhong Ni et al. Efficacy And Safety Of Odevixibat In Children With Progressive Familial Intrahepatic Cholestasis With Prior Partial External Biliary Diversion. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S204–6.
Philip Stein, Greg Wright, Lise Kjems et al. Drug-drug Interaction Study to Evaluate the Interaction of A4250 (Odevixibat) With Midazolam, a Sensitive CYP3A4 Substrate, and Itraconazole, a P-gp Inhibitor, in Healthy Adult Subjects. J Pediatr Gastroenterol Nutr. 2021;72(suppl 1):873–4.
Henkjan J. Verkade, Folkert Kuipers, Qifeng Yu, Velichka Valcheva, Patrick Horn, Lise Kjems. Natural Variation of Serum Bile Acid Levels and Pruritus in Children With Progressive Familial Intrahepatic Cholestasis: Pretreatment Data From Patients in the Phase 3 PEDFIC 1 Study. Hepatology. 2021;74(suppl 1):1171A–2A.
Nadia Ovchinsky, Jessica Savage, Lise Kjems, Patrick Horn. The ASSERT Study: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat In Patients With Alagille Syndrome. J Pediatr Gastroenterol Nutr. 2021;73(suppl 1):S191–2.
Richard Thompson, Patrick Horn, Roderick H.J. Houwen et al. Substantial Clinical Benefits With Odevixibat Treatment Across Progressive Familial Intrahepatic Cholestasis Genetic Deficiencies: Subgroup Analysis of Serum Bile Acids, Pruritus, and Safety Using Pooled Data From the PEDFIC 1 and 2 Studies. J Hepatol. 2021;75(suppl 2):S684–5.
Henkjan Verkade, Folkert Kuipers, Quanhong Ni et al. Pretreatment Serum Bile Acid Parameters and Predictability of Response to Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis. J Hepatol. 2021;75(suppl 2):S688–9.
Christoph Slavetinsky, Ekkehard Sturm. Odevixibat and Partial External Biliary Diversion Showed Equal Improvement of Cholestasis in a Patient with Progressive Familial Intrahepatic Cholestasis. BMJ Case Rep. 2020;13(6):e234185.
Saul Karpen, Greg Wright, Phillip Stein, Patrick Horn. The BOLD Study: A Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Infants With Biliary Atresia After Kasai Portoenterostomy. J Pediatr Gastroenterol Nutr 2020;71(suppl 1):S435–6.
Richard J. Thompson, Ulrich Baumann, Piotr Czubkowski et al. Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor, in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2: Results From PEDFIC 1, a Randomized, Double-blind, Placebo-controlled Phase 3 Trial. AASLD 2020 Late-breaking Abstracts. Abstract LO4; page 9.
Richard J. Thompson, Reha Artan, Lorenzo D’Antiga et al. Long-term Efficacy and Safety of Odevixibat, an Ileal Bile Acid Transporter Inhibitor in Children With Progressive Familial Intrahepatic Cholestasis: Interim Results From PEDFIC 2, an Open-label Phase 3 Trial. AASLD 2020 Late-breaking Abstracts. Abstract LP19; page 34–5.
Philip Stein, Greg Wright, Lise Kjems et al. Drug-drug Interaction Study to Evaluate the Interaction of A4250 (Odevixibat) With Itraconazole, a P-gp Inhibitor in Healthy Adult Subjects. J Pediatr Gastroenterol Nutr. 2020;71(suppl 1):S433–4.
Per-Göran Gillberg, Jan Mattsson, Kristina Torfgård et al. Clinical Pharmacology of Odevixibat, a Potent, Selective Ileal Bile Acid Transport Inhibitor with Minimal Systemic Exposure. J Pediatr Gastroenterol Nutr. 2019;69(suppl 2):S113.
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille et al. Effects of the Ileal Bile Acid Transport Inhibitor A4250 on Pruritus and Serum Bile Acids in Patients With Biliary Atresia: Phase 2 Study Results. J Hepatol. 2019;70(1 suppl):e411.
Christoph Slavetinsky, Ekkehard Sturm. Impact of an Ileal Bile Acid Transporter Inhibitor Versus Partial External Biliary Diversion in Progressive Familial Intrahepatic Cholestasis – A Case Providing Direct Comparison of Medical and Surgical Therapies. J Pediatr Gastroenterol Nutr. 2019;68(suppl 1):892–3.
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille et al. Effects of the Ileal Bile Acid Transport Inhibitor A4250 on Serum Bile Acids, Pruritus, and Sleep in Patients With Alagille Syndrome: Phase 2 Study Results. J Hepatol. 2019;70(1 suppl):e120. Z
Ulrich Baumann, Ekkehard Sturm, Florence Lacaille, Emmanuel Gonzalès, Henrik Arnell, Björn Fischler, Marianne Hørby Jørgensen, Richard J. Thompson, Jan P. Mattsson, Per-Göran Gillberg, Kristina Torfgård. Effects of the Ileal Bile Acid Transport Inhibitor A4250 on Pruritus, and Serum Bile Acids in Patients With Biliary Atresia: Phase 2 Study Results. J Pediatr Gastroenterol Nutr. 2019;68(suppl 1):767–8.
Ekkehard Sturm, Ulrich Baumann, Florence Lacaille, Emmanuel Gonzalès, Henrik Arnell, Björn Fischler, Marianne Hørby Jørgensen, Richard Thompson, Jan Mattsson, Mats Ekelund, Erik Lindström, Per-Göran Gillberg, Kristina Torfgård, Paresh Soni. The Ileal Bile Acid Transport Inhibitor A4250 Reduced Pruritus and Serum Bile Acid Levels in Children With Cholestatic Liver Disease and Pruritus: Final Results From a Multiple-dose, Open-label, Multinational Study. J Pediatr Gastroenterol Nutr. 2017;65(suppl 2):S168–9.
Samer Al-Dury, Annika Wahlström, Staffan Wahlin et al. Pilot Study With IBAT Inhibitor A4250 for the Treatment of Cholestatic Pruritus in Primary Biliary Cholangitis. Sci Rep. 2018;8(1):6658.
Emmanuel Gonzalès, Ekkehard Sturm, Ulrich Baumann et al. Correlation of Autotaxin Levels, Serum Bile Acids, and Pruritus in a Multiple-dose, Open label, Multinational Study of the Ileal Bile Acid Transport Inhibitor A4250. J Hepatol. 2018;68(suppl 1):S632.
Ekkehard Sturm, Ulrich Baumann, Florence Lacaille et al. The Ileal Bile Acid Transport Inhibitor A4250 Reduced Pruritus and Serum Bile Acid Levels in Children With Cholestatic Liver Disease and Pruritus: Final Results From a Multiple-dose, Open-label, Multinational Study. Hepatology. 2017;66(suppl 1):646A–7A.
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