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

[1] Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: data from MARCH-PFIC
D'Antiga, Lorenzo
Miethke, Alexander
Mogul, Douglas
Nunes, Tiago
Garner, Will
Vig, Pamela
Thompson, Richard
JOURNAL OF HEPATOLOGY, 2023, 78 : S61 - S61
[2] Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with progressive familial intrahepatic cholestasis: data from MARCH-PFIC
Thompson, Richard
Mogul, Douglas
Nunes, Tiago
Garner, Will
Vig, Pamela
Miethke, Alexander
JOURNAL OF HEPATOLOGY, 2023, 78 : S370 - S370
[3] IMPROVEMENTS IN PRURITUS WITH MARALIXIBAT ARE ASSOCIATED WITH IMPROVED QUALITY OF LIFE FOR PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS: DATA FROM THE MARCH-PFIC TRIAL
Aqul, Amal A.
Miethke, Alexander G.
Lin, Chuan-Hao
Porta, Gilda
Sokal, Etienne M.
Mogul, Douglas B.
Nunes, Tiago
Baek, Marshall
Vig, Pamela
Baumann, Ulrich
Thompson, Richard J.
HEPATOLOGY, 2023, 78 : S2092 - S2093
[4] EFFICACY AND SAFETY OF MARALIXIBAT IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (MARCH-PFIC): A RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY
Miethke, Alexander
Moukarzel, Adib
Porta, Gilda
Esquer, Joshue Covarrubias
Czubkowski, Piotr
Ordonez, Felipe
Candusso, Manila
Aqul, Amal A.
Squires, Robert H.
Sokal, Etienne
D'Agostino, Daniel
Baumann, Ulrich
D'Antiga, Lorenzo
Kasi, Nagraj
Laborde, Nolwenn
Arikan, Cigdem
Lin, Chuan-Hao
Gilmour, Susan
Mittal, Naveen
Chiou, Fang Kuan
Horslen, Simon P.
Huber, Wolf-Dietrich
Van Leerberghe, Arthur
Ronn, Susanne Weber
Nunes, Tiago
Lascau, Anamaria
Longpre, Lara
Garner, Will
Vig, Pamela
Hupertz, Vera F.
Gonzalez-Peralta, Regino P.
Ekong, Udeme
Hartley, Jane
Laverdure, Noemie
Ovchinsky, Nadia
Thompson, Richard J.
HEPATOLOGY, 2023, 77 (05) : E117 - E118
[5] MARALIXIBAT LEADS TO SIGNIFICANT IMPROVEMENTS IN CHOLESTATIC PRURITUS FOR CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS WITHOUT A GENETIC DIAGNOSIS: DATA FROM THE MARCH-PFIC TRIAL
Horslen, Simon P.
Moukarzel, Adib
Miethke, Alexander G.
Mittal, Naveen
Ekong, Udeme
Kasi, Nagraj
Mogul, Douglas B.
Nunes, Tiago
Aguilar, Raul
Vig, Pamela
Gilmour, Susan M.
Thompson, Richard J.
HEPATOLOGY, 2023, 78 : S2096 - S2097
[6] Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Miethke, Alexander G.
Moukarzel, Adib
Porta, Gilda
Esquer, Joshue Covarrubias
Czubkowski, Piotr
Ordonez, Felipe
Mosca, Antonella
Aqul, Amal A.
Squires, Robert H.
Sokal, Etienne
D'Agostino, Daniel
Baumann, Ulrich
D'Antiga, Lorenzo
Kasi, Nagraj
Laborde, Nolwenn
Arikan, Cigdem
Lin, Chuan-Hao
Gilmour, Susan
Mittal, Naveen
Chiou, Fang Kuan
Horslen, Simon P.
Huber, Wolf -Dietrich
Jaecklin, Thomas
Nunes, Tiago
Lascau, Anamaria
Longpre, Lara
Mogul, Douglas B.
Garner, Will
Vig, Pamela
Hupertz, Vera F.
Gonzalez-Peralta, Regino P.
Ekong, Udeme
Hartley, Jane
Laverdure, Noemie
Ovchinsky, Nadia
Thompson, Richardj
LANCET GASTROENTEROLOGY & HEPATOLOGY, 2024, 9 (07): : 620 - 631
[7] Maralixibat improves growth in patients with progressive familial intrahepatic cholestasis: data from the MARCH/MARCH-ON trials
Gonzalez-Peralta, Regino P.
Miethke, Alexander
Aqul, Amal A.
Mogul, Douglas B.
Nunes, Tiago
Garner, Will
Vig, Pamela
Thompson, Richard J.
JOURNAL OF HEPATOLOGY, 2024, 80 : S323 - S324
[8] MARALIXIBAT LEADS TO SIGNIFICANT IMPROVEMENT IN CHOLESTATIC PRURITUS FOR CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS DUE TO TJP2 OR MYO5B DEFICIENCY: DATA FROM THE MARCH-PFIC TRIAL
Miethke, Alexander G.
Porta, Gilda
Moukarzel, Adib
Lin, Chuan-Hao
D'Agostino, Daniel
Mogul, Douglas B.
Nunes, Tiago
Aguilar, Raul
Vig, Pamela
Chiou, Fang Kuan
Thompson, Richard J.
HEPATOLOGY, 2023, 78 : S2095 - S2096
[9] Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): data from the MARCH-ON study
Miethke, Alexander
Moukarzel, Adib
Porta, Gilda
Esquer, Joshue Covarrubias
Czubkowski, Piotr
Ordonez, Felipe
Candusso, Manila
Aqul, Amal A.
Squires, Robert H.
Sokal, Etienne
D'Agostino, Daniel
Baumann, Ulrich
D'Antiga, Lorenzo
Kasi, Nagraj
Laborde, Nolwenn
Arikan, Cigdem
Lin, Chuan-Hao
Gilmour, Susan
Mittal, Naveen
Chiou, Fang Kuan
Horslen, Simon P.
Huber, Wolf-Dietrich
Nunes, Tiago
Lascau, Anamaria
Longpre, Lara
Mogul, Douglas
Aguilar, Raul
Vig, Pamela
Hupertz, Vera
Gonzalez-Peralta, Regino
Ekong, Udeme
Hartley, Jane
Laverdure, Noemie
Ovchinsky, Nadia
Thompson, Richard
JOURNAL OF HEPATOLOGY, 2023, 78 : S123 - S123
[10] Topics - Opinion on the Benefit Assessment of Maralixibat (New Indication: Progressive familial intrahepatic Cholestasis (PFIC), ≥ 3 Months)
Jansen, Petra Lynen
ZEITSCHRIFT FUR GASTROENTEROLOGIE, 2025, 63 (01): : 80 - 81

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