Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium

被引：25

作者：

Sola, Elsa ^{[1
,2
,3
]}

Pose, Elisa ^{[1
,2
,3
]}

Campion, Daniela ^{[4
]}

Piano, Salvatore ^{[5
]}

Roux, Olivier ^{[6
]}

Simon-Talero, Macarena ^{[7
]}

Uschner, Frank ^{[8
]}

de Wit, Koos ^{[9
]}

Zaccherini, Giacomo ^{[10
]}

Alessandria, Carlo ^{[4
]}

Beuers, Ulrich ^{[9
]}

Caraceni, Paolo ^{[10
]}

Francoz, Claire ^{[6
]}

Mookerjee, Rajeshwar P. ^{[11
]}

Trebicka, Jonel ^{[8
]}

Vargas, Victor ^{[7
]}

Serra, Miquel ^{[12
]}

Torres, Ferran ^{[13
]}

Montagnese, Sara ^{[14
]}

Krag, Aleksander ^{[15
,16
,17
]}

Hernaez, Ruben ^{[18
,19
,20
]}

Korenjak, Marko ^{[21
]}

Watson, Hugh ^{[22
,23
]}

Abraldes, Juan G. ^{[24
]}

Kamath, Patrick S. ^{[25
]}

Gines, Pere ^{[1
,2
,3
]}

机构：

[1] Hosp Clin Barcelona, Liver Unit, Villarroel 170, Barcelona 08036, Spain

[2] Univ Barcelona, Sch Med & Hlth Sci, Barcelona, Spain

[3] Ctr Invest Biomed Red Enfermedades Hepat & Digest, Inst Invest Biomed August Pi & Sunyer IDIBAPS, Barcelona, Spain

[4] Univ Turin, Citta Salute & Sci Hosp, Div Gastroenterol & Hepatol, Turin, Italy

[5] Univ Padua, Dept Med DIMED, Unit Internal Med & Hepatol UIMH, Padua, Italy

[6] Hosp Beaujon, AP HP, Hepatol & Liver Intens Care Unit, Clichy, France

[7] Univ Autonoma Barcelona, Hosp Vall dHebron & Vall dHebron Res Unit VHIR, Ctr Invest Biomed Red Enfermedades Hepat & Digest, Liver Unit, Barcelona, Spain

[8] Goethe Univ Frankfurt, Dept Internal Med 1, Frankfurt, Germany

[9] Univ Amsterdam, Acad Med Ctr, Dept Gastroenterol & Hepatol, Amsterdam, Netherlands

[10] Alma Mater Studiorum Univ Bologna, Dept Med & Surg Sci, Bologna, Italy

[11] UCL, Inst Liver & Digest Hlth, London, England

[12] Univ Pompeu Fabra, Ctr Res Hlth & Econ, Barcelona, Catalonia, Spain

[13] Hosp Clin Barcelona, Inst Invest Biomed August Pi & Sunyer, Biostat & Data Management Core Facil, Barcelona, Spain

[14] Univ Padua, Dept Med, Padua, Italy

[15] Odense Univ Hosp, Dept Gastroenterol & Hepatol, Odense, Denmark

[16] Odense Univ Hosp, Odense Patient Data Exploratory Network OPEN, Odense, Denmark

[17] Univ Southern Denmark, Inst Clin Res, Odense, Denmark

[18] Houston TX Ctr, Michael E DeBakey Vet Affairs Med Ctr, Sect Gastroenterol, Houston, TX USA

[19] Michael E DeBakey VA Med Ctr, Ctr Innovat Qual Effectiveness & Safety IQuESt, Houston, TX USA

[20] Baylor Coll Med, Dept Med, Sect Gastroenterol & Hepatol, Houston, TX 77030 USA

[21] European Liver Patients Assoc ELPA, Brussels, Belgium

[22] Evotec ID, Lyon, France

[23] Aarhus Univ, Dept Hepatol & Gastroenterol, Aarhus, Denmark

[24] Univ Alberta, Div Gastroenterol, CEGIIR, Cirrhosis Care Clin,Liver Unit, Edmonton, AB, Canada

[25] Mayo Clin, Div Gastroenterol & Hepatol, Rochester, MN USA

来源：

JOURNAL OF HEPATOLOGY | 2021年 / 74卷 / 01期

关键词：

Cirrhosis; Liver transplant; Clinical trials; Endpoints; Ascites; AKI; Hepatic encephalopathy; Hyponatremia; Infections; ACLF; Quality of life; QUALITY-OF-LIFE; SPONTANEOUS BACTERIAL PERITONITIS; V-2 RECEPTOR ANTAGONIST; LONG-TERM PROGNOSIS; INTRAHEPATIC PORTOSYSTEMIC SHUNTS; GELATINASE-ASSOCIATED LIPOCALIN; SERUM SODIUM CONCENTRATION; PARACENTESIS PLUS ALBUMIN; URINARY ETHYL GLUCURONIDE; ACUTE KIDNEY INJURY;

D O I：

10.1016/j.jhep.2020.08.009

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

Management of decompensated cirrhosis is currently geared towards the treatment of complications once they occur. To date there is no established disease-modifying therapy aimed at halting progression of the disease and preventing the development of complications in patients with decompensated cirrhosis. The design of clinical trials to investigate new therapies for patients with decompensated cirrhosis is complex. The population of patients with decompensated cirrhosis is heterogeneous (i.e., different etiologies, comorbidities and disease severity), leading to the inclusion of diverse populations in clinical trials. In addition, primary endpoints selected for trials that include patients with decompensated cirrhosis are not homogeneous and at times may not be appropriate. This leads to difficulties in comparing results obtained from different trials. Against this background, the LiverHope Consortium organized a meeting of experts, the goal of which was to develop recommendations for the design of clinical trials and to define appropriate endpoints, both for trials aimed at modifying the natural history and preventing progression of decompensated cirrhosis, as well as for trials aimed at managing the individual complications of cirrhosis. (C) 2020 European Association for the Study of the Liver. Published by Elsevier B.V.

引用

页码：200 / 219

页数：20

共 50 条

[1] American Association for the Study of Liver Diseases Endpoints Conference: Design and Endpoints for Clinical Trials in Primary Biliary Cirrhosis
Silveira, Marina G.
Brunt, Elizabeth M.
Heathcote, Jenny
Gores, Gregory J.
Lindor, Keith D.
Mayo, Marlyn J.
HEPATOLOGY, 2010, 52 (01) : 349 - 359
[2] Clinical trials design: protocols and endpoints
MG Wyllie
International Journal of Impotence Research, 2000, 12 : S53 - S58
[3] Clinical trials design: protocols and endpoints
Wyllie, MG
INTERNATIONAL JOURNAL OF IMPOTENCE RESEARCH, 2000, 12 (Suppl 4) : S53 - S58
[4] Role of Fecal Microbiota Transplantation in Decompensated Cirrhosis: An Initial Experience of Clinical Endpoints
Roy, Akash
Dhiman, Radha K.
Premkumar, Madhumita
Duseja, Ajay
Taneja, Sunil
Verma, Nipun
JOURNAL OF GASTROENTEROLOGY AND HEPATOLOGY, 2019, 34 : 537 - 537
[5] Design and Endpoints of Clinical Trials, Current and Future
Sherman, Morris
DIGESTIVE DISEASES AND SCIENCES, 2019, 64 (04) : 1050 - 1057
[6] Clinical trials: design, endpoints and interpretation of outcomes
Megan Othus
Mei-Jie Zhang
Robert Peter Gale
Bone Marrow Transplantation, 2022, 57 : 338 - 342
[7] Clinical trials: design, endpoints and interpretation of outcomes
Othus, Megan
Zhang, Mei-Jie
Gale, Robert Peter
BONE MARROW TRANSPLANTATION, 2022, 57 (03) : 338 - 342
[8] Design and endpoints of clinical trials in hepatocellular carcinoma
Llovet, Josep M.
Di Bisceglie, Adrian M.
Bruix, Jordi
Kramer, Barnett S.
Lencioni, Riccardo
Zhu, Andrew X.
Sherman, Morris
Schwartz, Myron
Lotze, Michael
Talwalkar, Jayant
Gores, Gregory J.
JNCI-JOURNAL OF THE NATIONAL CANCER INSTITUTE, 2008, 100 (10): : 698 - 711
[9] Novel endpoints and design of early clinical trials
Parulekar, WR
Eisenhauer, EA
ANNALS OF ONCOLOGY, 2002, 13 : 139 - 143
[10] Design and Endpoints of Clinical Trials, Current and Future
Morris Sherman
Digestive Diseases and Sciences, 2019, 64 : 1050 - 1057

← 1 2 3 4 5 →