American Association for the Study of Liver Diseases endpoints conference: design and endpoints for clinical trials in primary biliary cirrhosis
Open accessAbstract
A group of multidisciplinary experts on primary biliary cirrhosis (PBC) and its complications convened on May 31, 2009, under the aegis of the American Association for the Study of Liver Diseases (AASLD) in order to identify the most appropriate design and endpoints for clinical trials of PBC based on current evidence and expert experience.The natural history of PBC was reviewed as well as current therapies.The current approaches to evaluating therapies for disease progression and symptoms as priorities were discussed.Appropriate aspects of trial design including entry criteria, study duration, and appropriate handling of issues such as stratification of subjects and use of ursodeoxycholic acid (UDCA), were identified and discussed.After a full day of presentations, in a consensus manner, appropriate endpoints for clinical efficacy trials regarding PBC and its complications were agreed upon and are reported in this summary.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Treatment failure should be defined using validated surrogate endpoints with reproducible prognostic performance across cohorts.
“Treatment failure must be defined on validated surrogate endpoints to account for the slow progression of disease, i.e. endpoints that have significant reproducible prognostic performance in different cohorts of patients [100].”
From EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. (2017) · cites this paper as [100]
The Endpoints of Clinical Trials in PBC section says slow progression makes death or transplantation impractical trial endpoints, and the Biochemical Markers section recommends biochemical markers as primary endpoints. But the paper does not say treatment failure must be defined by validated surrogates with reproducible prognostic performance across different cohorts; it allows several ways to assess response. Page numbers were not available in our copy. (LLM, conf=0.85)
Experimental — accuracy not yet measured
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
The citation lists haven’t been fetched yet
References 87 · Cited by 87
Research works printed but not linked: 4
- [9]9. Kim WR, Lindor KD, Locke GR 3rd, Therneau TM, Homburger HA, Batts KP, et al. Epidemiology and natural history of primary biliary cir- rhosis in a US community. Gastroenterology 2000;119:1631-1636.
- [26]26. HEPATOLOGY, Vol. 52, No. 1, 2010 SILVEIRA ET AL. 357
- [74]74. Myers R, Shaheen A, Swain M, Lee S, Cole S, Coffey-Williamson S, et al. Rituximab for primary biliary cirrhosis refractory to ursodeoxy- cholic acid [Abstract]. HEPATOLOGY 2007;46:550A.
- [82]82. HEPATOLOGY, Vol. 52, No. 1, 2010 SILVEIRA ET AL. 359
- Authors
- Silveira, M. G., Brunt, E. M., Heathcote, J., Gores, G. J., Lindor, K. D., & Mayo, M. J.
- Affiliations
- Mayo Clinic (US)
- Washington University in St. Louis (US)
- University of Toronto (CA)
- The University of Texas Southwestern Medical Center (US)
- Corresponding author
- Mayo, Marlyn J.
- Venue
- Hepatology
- Published
- 2010-07
- Volume
- 52
- Issue
- 1
- Pages
- 349-359
- ISSN
- 0270-9139 (ISSN-L)
- OpenAlex
- W2057537884
- Semantic Scholar
- 4ffa8aab5a8136a1eb819bad9059f63d21c0b9e0
- Cited by
- 87
- References
- 87
- Citations by year
- 2025: 4 · 2024: 4 · 2023: 3 · 2022: 7 · 2021: 2 · 2020: 4 · 2019: 5 · 2018: 6 · 2017: 5 · 2016: 6 · 2015: 6 · 2014: 11 · 2013: 7 · 2012: 6
- FWCI
- 4.78
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Hepatology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Hepatology › Liver Diseases and Immunity
- Health Sciences › Medicine › Surgery › Pediatric Hepatobiliary Diseases and Treatments
- Health Sciences › Medicine › Epidemiology › Liver Disease Diagnosis and Treatment
- Keywords
- primary biliary cirrhosis, clinical trial endpoints, clinical trial design, natural history of disease, ursodeoxycholic acid, disease progression, symptom assessment, patient stratification
- MeSH terms
- Clinical Trials as Topic, Humans, Liver Cirrhosis, Biliary, Research Design, Societies, Medical, United States, Patient Selection
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
