Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: data from MARCH-PFIC

被引：0

作者：

Miethke, Alexander ^{[1
]}

Moukarzel, Adib ^{[2
]}

Porta, Gilda ^{[3
]}

Esquer, Joshue Covarrubias ^{[4
]}

Czubkowski, Piotr ^{[5
]}

Ordonez, Felipe ^{[6
]}

Candusso, Manila ^{[7
]}

Aqul, Amal A. ^{[8
]}

Squires, Robert H. ^{[9
]}

Sokal, Etienne ^{[10
]}

D'Agostino, Daniel ^{[11
]}

Baumann, Ulrich ^{[12
]}

D'Antiga, Lorenzo ^{[13
]}

Kasi, Nagraj ^{[14
]}

Laborde, Nolwenn ^{[15
]}

Arikan, Cigdem ^{[16
]}

Lin, Chuan-Hao ^{[17
]}

Gilmour, Susan ^{[18
]}

Mittal, Naveen ^{[19
]}

Chiou, Fang Kuan ^{[20
]}

Horslen, Simon P.

Huber, Wolf-Dietrich ^{[21
]}

David-Feliciano, Susan ^{[22
]}

Chien, Elaine ^{[22
]}

Mogul, Douglas ^{[22
]}

Garner, Will ^{[22
]}

Nunes, Tiago ^{[22
]}

Lascau, Anamaria ^{[22
]}

Vig, Pamela ^{[22
]}

Hupertz, Vera ^{[23
]}

Gonzalez-Peralta, Regino ^{[24
]}

Ekong, Udeme ^{[25
]}

Hartley, Jane ^{[26
]}

Laverdure, Noemie

Ovchinsky, Nadia ^{[27
]}

Thompson, Richard

机构：

[1] Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

[2] Hotel Dieu De France St Joseph Univ Hosp, Beirut, Lebanon

[3] Hosp Sirio Libanes, Sao Paulo, Brazil

[4] Nois Mexico SA CV, Guadalajara, Jalisco, Mexico

[5] Childrens Mem Hlth Inst, Gastroenterol Hepatol, Nutrit Disorders & Pediat, Warsaw, Poland

[6] Cardioinfantil Fdn Lacardio, Bogota, Colombia

[7] Osped Pediatrico Bambino Gesu Irccs, Lazio, Italy

[8] Univ Texas Southwestern Med Ctr Dallas, Dallas, TX USA

[9] UPMC Childrens Hosp Pittsburgh, Pittsburgh, PA USA

[10] Uclouvian, Clin Univ St Luc, Pediat Hepatol, Brussels, Belgium

[11] Hosp Italiano Buenos Aires, Buenos Aires, Argentina

[12] Hannover Med Sch, Pediat Gastroenterol & Hepatol, Hannover, Germany

[13] Hosp Papa Giovanni XXIII, Paediat Hepatol Gastroenterol & Transplantat, Bergamo, Italy

[14] Univ South Carolina, Charleston, SC USA

[15] Hop Enfants CHU Toulouse, Toulouse, France

[16] Koc Univ, Sch Med, Istanbul, Turkiye

[17] Childrens Hosp Los Angeles, Los Angeles, CA USA

[18] Univ Alberta, Pediat, Edmonton, AB, Canada

[19] Univ Texas Hlth Sci Ctr San Antonio, San Antonio, TX USA

[20] KK Womens & Childrens Hosp, Singapore, Singapore

[21] Med Univ Vienna, Vienna, Austria

[22] Mirun Pharmaceut Inc, Foster City, CA USA

[23] Cleveland Clin Childrens, Cleveland, OH USA

[24] AdventHlth Children & AdventHlth Transplant Inst, Pediat Gastroenterol Hepatol & Liver Transplant, Orlando, FL USA

[25] Georgetown Univ Hosp, Medstar Georgetown Transplant Inst, Washington, DC USA

[26] Birmingham Women & Childrens Hosp, Birmingham, England

[27] Childrens Hosp Montefiore, Bronx, NY USA

来源：

JOURNAL OF HEPATOLOGY | 2023年 / 78卷

关键词：

D O I：

暂无

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

WED-282

引用

页码：S387 / S388

页数：2

共 43 条

[21] 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)
Ozen, Hasan
Sokal, Etienne
Lacaille, Florence
Dalgic, Buket
Ni, Quanhong
Kjems, Lise
Horn, Patrick
HEPATOLOGY, 2021, 74 : 1160A - 1161A
[22] 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
Thompson, Richard J.
Artan, Reha
Baumann, Ulrich
Czubkowski, Piotr
Dalgic, Buket
Durmaz, Ozlem
Gonzales, Emmanuel
Grammatikopoulos, Tassos
Gupte, Girish
Horn, Patrick
Lachaux, Alain
Mckiernan, Patrick
Ozen, Hasan
Rajwal, Sanjay R.
Roquelaure, Bertrand
Sturm, Ekkehard
Verkade, Henkjan J.
Yu, Qifeng
Kjems, Lise
GASTROENTEROLOGY, 2022, 162 (07) : S1296 - S1297
[23] 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
Thompson, Richard
Artan, Reha
Baumann, Ulrich
Czubkowski, Piotr
Dalgic, Buket
Durmaz, Ozlem
Gonzales, Emmanuel
Grammatikopoulos, Tassos
Gupte, Girish
Horn, Patrick
Lachaux, Alain
McKiernan, Patrick
Ozen, Hasan
Rajwal, Sanjay
Roquelaure, Bertrand
Sturm, Ekkehard
Verkade, Henkjan
Yu, Qifeng
Kjems, Lise
JOURNAL OF HEPATOLOGY, 2021, 75 : S687 - S687
[24] NATIVE LIVER SURVIVAL IN ODEVIXIBAT SBA RESPONDERS: DATA FROM THE PEDFIC STUDIES IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
Clemson, Christine
Valcheva, Velichka
Sturm, Ekkehard
Verkade, Henkjan J.
Thompson, Richard J.
Ni, Quanhong
Yu, Qifeng
Mattsson, Jan P.
HEPATOLOGY, 2022, 76 : S206 - S206
[25] ATP8B1 and ABCB11 Analysis in 62 Children with Normal Gamma-Glutamyl Transferase Progressive Familial Intrahepatic Cholestasis (PFIC): Phenotypic Differences Between PFIC1 and PFIC2 and Natural History
Davit-Spraul, Anne
Fabre, Monique
Branchereau, Sophie
Baussan, Christiane
Gonzales, Emmanuel
Stieger, Bruno
Bernard, Olivier
Jacquemin, Emmanuel
HEPATOLOGY, 2010, 51 (05) : 1645 - 1655
[26] Phase 2 open-label efficacy and safety study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with progressive familial intrahepatic cholestasis: 48-week interim efficacy analysis
Thompson, Richard J.
Kelly, Deirdre A.
McClean, Patricia
Miethke, Alexander G.
Soufi, Nisreen
Rivet, Christine
Jankowska, Irena
Mack, Cara L.
Karthikeyan, Palaniswamy
Gu, Joan
Jaecklin, Thomas
Squires, Robert H.
Loomes, Kathleen M.
HEPATOLOGY, 2017, 66 : 57A - 57A
[27] 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
Gonzales, Emmanuel M.
Thompson, Richard J.
Kamath, Binita
Lacaille, Florence
Lainka, Elke
Shteyer, Eyal
D'Antiga, Lorenzo
Houwen, Roderick
Verkade, Henkjan J.
Durmaz, Ozlem
Grammatikopoulos, Tassos
Loomes, Kathleen M.
Karpen, Saul J.
Mack, Cara Lynn
Ni, Quanhong
Kjems, Lise
Horn, Patrick
HEPATOLOGY, 2021, 74 : 30A - 31A
[28] NATIVE LIVER SURVIVAL IN ODEVIXIBAT SERUM BILE ACID RESPONDERS: DATA FROM PEDFIC STUDIES IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
Jeanes, Yvonne
Seamark, Leah
Radcliffe, Olivia
Gotto, James
Watson, Bridie
Trudgeon, Jenna
Whiteman, Nicola
Stockton, Elizabeth
Apekey, Tanefa
Colgan, Aisling
Digby, Katie
Hickson, Mary
Williams, Marianne
GUT, 2023, 72 (SUPPL_2) : A49 - A50
[29] ODEVIXIBAT THERAPY IMPROVES CLINICALLY MEANINGFUL ENDPOINTS IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: DATA FROM THE PEDFIC 1 AND PEDFIC 2 TRIALS
Thompson, Richard J.
D'Antiga, Lorenzo
Gonzales, Emmanuel
Karpen, Saul J.
Kjems, Lise
Loomes, Kathleen
Mack, Cara L.
Ni, Quanhong
Verkade, Henkjan J.
Horn, Patrick
GASTROENTEROLOGY, 2022, 162 (07) : S1296 - S1296
[30] 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
Thompson, Richard
Horn, Patrick
Houwen, Roderick H. J.
Lacaille, Florence
Ni, Quanhong
Stein, Philip
Tessier, Mary Elizabeth
Thompson, Carrie
Vittorio, Jennifer
Kjems, Lise
JOURNAL OF HEPATOLOGY, 2021, 75 : S684 - S685

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