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

[31] CORRELATIONS BETWEEN PATIENT- AND CAREGIVER-REPORTED PRURITUS FROM PEDFIC 1: EVALUATION OF PRUCISION DATA IN PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
Clemson, C.
Ni, Q.
Horn, P.
VALUE IN HEALTH, 2022, 25 (07) : S322 - S322
[32] Odevixibat therapy improves clinically meaningful end points in children with progressive familial intrahepatic cholestasis: Data from the PEDFIC 1 and PEDFIC 2 trials
Thompson, Richard
D'Antiga, Lorenzo
Gonzales, Emmanuel
Karpen, Saul
Kjems, Lise
Loomes, Kathleen
Mack, Cara
Ni, Quanhong
Verkade, Henkjan
Horn, Patrick
JOURNAL OF HEPATOLOGY, 2021, 75 : S688 - S688
[33] 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
Loomes, Kathleen M.
Verkade, Henkjan J.
Thompson, Richard J.
Kamath, Binita
Hardikar, Winita
Lacaille, Florence
Mozer-Glassberg, Yael
Shteyer, Eyal
Calvo, Pier Luigi
Dalgic, Buket
Grammatikopoulos, Tassos
Rajwal, Sanjay
Vittorio, Jennifer M.
Soufi, Nisreen
McKiernan, Patrick James
Tessier, Mary Elizabeth
Yu, Qifeng
Kjems, Lise
Horn, Patrick
HEPATOLOGY, 2021, 74 : 1159A - 1160A
[34] Optimal liver transplant procedure in progressive familial intrahepatic cholestasis type 1 treated with biliary diversion or intestinal transplantation: Lessons learned from three cases treated with different approaches
Uchida, Hajime
Sakamoto, Seisuke
Komine, Ryuji
Kodama, Tasuku
Nakao, Toshimasa
Yanagi, Yusuke
Shimizu, Seiichi
Abbas, Syed Hasnain
Fukuda, Akinari
Kasahara, Mureo
PEDIATRIC TRANSPLANTATION, 2023, 27 (06)
[35] 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
McKiernan, Patrick James
Sturm, Ekkehard
Kamath, Binita
Thompson, Richard J.
Gonzales, Emmanuel M.
Lachaux, Alain
Baumann, Ulrich
Shteyer, Eyal
Czubkowski, Piotr
Artan, Reha
Dalgic, Buket
Ozen, Hasan
Gupte, Girish
Grammatikopoulos, Tassos
Karpen, Saul J.
Ni, Quanhong
Kjems, Lise
Horn, Patrick
HEPATOLOGY, 2021, 74 : 1164A - 1164A
[36] 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
D'Antiga, Lorenzo
Gonzales, Emmanuel M.
Sokal, Etienne
Hardikar, Winita
Verkade, Henkjan J.
Mattsson, Jan P.
Ni, Quanhong
Stein, Philip
HEPATOLOGY, 2022, 76 : S1547 - S1548
[37] 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
Verkade, Henkjan J.
Kuipers, Folkert
Yu, Qifeng
Valcheva, Velichka
Horn, Patrick
Kjems, Lise
HEPATOLOGY, 2021, 74 : 1171A - 1172A
[38] 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
Thompson, Richard J.
Calvo, Pier Luigi
Hardikar, Winita
Horn, Patrick
Lainka, Elke
Mack, Cara L.
Ni, Quanhong
Kjems, Lise
GASTROENTEROLOGY, 2022, 162 (07) : S1297 - S1297
[39] 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
Thompson, Richard
Calvo, Pier Luigi
Hardikar, Winita
Horn, Patrick
Lainka, Elke
Mack, Cara
Ni, Quanhong
Kjems, Lise
JOURNAL OF HEPATOLOGY, 2021, 75 : S683 - S684
[40] ANALYSIS OF LONG-TERM TREATMENT EFFECTS OF ODEVIXIBAT ON CLINICAL OUTCOMES IN CHILDREN WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS IN ODEVIXIBAT CLINICAL STUDIES VS EXTERNAL CONTROLS FROM THE NAPPED DATABASE
Hansen, Bettina
Valcheva, Velichka
Yu, Qifeng
van Wessel, Daan B. E.
Thompson, Richard J.
Gonzales, Emmanuel
Jankowska, Irena
Sokal, Etienne
Grammatikopoulos, Tassos
Kadaristiana, Agustina
Jacquemin, Emmanuel
Spraul, Anne
Lipinski, Patryk
Czubkowski, Piotr
Rock, Nathalie
Shagrani, Mohammad
Kumar, Kishwer
Alkuraya, Fowzan
Sateesh, Meddirevula
Broering, Dieter
Nicastro, Emanuele
Kelly, Deirdre A.
Nebbia, Gabriella
Arnell, Henrik
Fischler, Bjorn
Serranti, Daniele
Arikan, Cigdem
Polat, Esra
Debray, Dominique
Lacaille, Florence
Goncalves, Cristina
Hierro, Loreto
Bartolo, Gema Munoz
Mozer-Glassberg, Yael
Azaz, Amer
Brecelj, Jernej
Dezsofi, Antal
Calvo, Pier Luigi
Grabhorn, Enke
Sturm, Ekkehard
van der Woerd, Wendy L.
Kamath, Binita M.
Wang, Jian-She
Li, Liting
Durmaz, Ozlem
Onal, Zerrin
Felzen, Antonia
Nomden, Mark
Sonajalg, Jaak
Stein, Philip
GUT, 2023, 72 : A13 - A13

← 1 2 3 4 5 →