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

[41] 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
Thompson, Richard
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 Clemens
Nicastro, Emanuele
Kelly, Deirdre
Nebbia, Gabriella
Arnell, Henrik
Fischler, Bjorn
Serranti, Daniele
Arikan, Cigdem
Polat, Esra
Debray, Dominique
Lacaille, Florence
Goncalves, Cristina
Llanillo, Loreto Hierro
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
JOURNAL OF HEPATOLOGY, 2023, 78 : S969 - S970
[42] 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, Bjoern
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 (SUPPL_3) : A13 - A13
[43] 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
Verkade, Henkjan J.
Thompson, Richard J.
Sturm, Ekkehard
Baumann, Ulrich
Gonzales, Emmanuel M.
D'Antiga, Lorenzo
Valcheva, Velichka
Ni, Quanhong
Clemson, Christine
HEPATOLOGY, 2022, 76 : S1548 - S1549

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