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Record W3212731641 · doi:10.1093/ibd/izab259

Recommendations for Standardizing Clinical Trial Design and Endoscopic Assessment in Postoperative Crohn’s Disease

2021· article· en· W3212731641 on OpenAlexaff
Jurij Hanžel, Vipul Jairath, Peter De Cruz, Leonardo Guizzetti, Lisa M. Shackelton, Peter Bossuyt, Marjolijn Duijvestein, Parambir S. Dulai, Johannes Grossmann, Robert Hirten, Reena Khanna, Julián Panés, Laurent Peyrin‐Biroulet, Miguel Regueiro, David T. Rubin, Siddharth Singh, Ryan W. Stidham, William J. Sandborn, Brian G. Feagan, Geert D’Haens, Christopher Ma

Bibliographic record

VenueInflammatory Bowel Diseases · 2021
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicInflammatory Bowel Disease
Canadian institutionsUniversity of CalgaryWestern University
FundersUCB PharmaAllerganGenentechAkebia TherapeuticsCelltrionAbbVieNovo NordiskTeva Pharmaceutical IndustriesGilead SciencesActoGeniXSanofiCelgeneBiogenGlaxoSmithKlineNational Institute of Diabetes and Digestive and Kidney DiseasesTillotts PharmaAmgenPfizerAstraZenecaEli Lilly and CompanyBristol-Myers Squibb
KeywordsMedicineCrohn's diseaseClinical trialPlaceboDiseaseClinical endpointInternal medicineSurgeryPhysical therapyAlternative medicinePathology

Abstract

fetched live from OpenAlex

BACKGROUND: The lack of standardized methods for clinical trial design and disease activity assessment has contributed to an absence of approved medical therapies for the prevention of postoperative Crohn's disease (CD). We developed recommendations for regulatory trial design for this indication and for endoscopic assessment of postoperative CD activity. METHODS: An international panel of 19 gastroenterologists was assembled. Modified Research and Development/University of California Los Angeles methodology was used to rate the appropriateness of 196 statements using a 9-point Likert scale in 2 rounds of voting. Results were reviewed and discussed between rounds. RESULTS: Inclusion of patients with a history of completely resected ileocolonic CD in regulatory clinical trials for the prevention of postoperative recurrence was appropriate. Given the absence of approved medical therapies, a placebo-controlled design with a primary end point of endoscopic remission at 52 weeks was appropriate for drug development for this indication; however, there was uncertainty regarding the appropriateness of a coprimary end point of symptomatic and endoscopic remission and the use of currently available patient-reported outcome measures. The modified Rutgeerts Score, endoscopic assessment of the anastomosis, and a minimum of 5cm of neoterminal ileum were also appropriate; although the appropriateness of other indices including the Simple Endoscopic Score for CD for endoscopic assessment of postoperative CD activity was uncertain. CONCLUSIONS: A framework for regulatory trial design for the prevention of postoperative CD recurrence and endoscopic assessment of disease activity has been developed. Research to empirically validate end points for these trials is needed.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.748
metaresearch head score (Gemma)0.755
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch
Consensus categoriesMetaresearch
DomainCandidate signal: Methods · Consensus signal: Methods
Study designCandidate signal: Theoretical or conceptual · Consensus signal: none
GenreCandidate signal: Methods · Consensus signal: none
Teacher disagreement score0.252
Threshold uncertainty score0.311

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.7480.755
Meta-epidemiology (narrow)0.0050.005
Meta-epidemiology (broad)0.0090.017
Bibliometrics0.0140.015
Science and technology studies0.0050.014
Scholarly communication0.0160.013
Open science0.0160.008
Research integrity0.0540.043
Insufficient payload (model declined to judge)0.0050.009

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.040
GPT teacher head0.353
Teacher spread0.314 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; the direct Gemma label and the distilled Codex classifier agree on what is shown here.

Study designTheoretical or conceptual
DomainMethods
GenreMethods

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations12
Published2021
Admission routes1
Has abstractyes

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