Developing IBD Outcome Effect Size Thresholds to Inform Research, Guidelines, and Clinical Decisions
Bibliographic record
Abstract
BACKGROUND: When designing clinical trials, interpreting trial outcomes for guideline development or sharing decisions with patients in clinical practice, the clinical outcomes used and the implicit choices on what constitutes a clinically significant finding can vary greatly. This can lead to diversity or even inequity in care offered to patients with inflammatory bowel disease (IBD). The GRADE approach to guideline development has proposed a process to address this prospectively to solve these issues, but this has never been used in IBD. We aimed to develop the first international consensus set of outcome thresholds to establish their use in Crohn's disease and ulcerative colitis. METHODS: A Delphi methodology was used to develop a consensus. An online survey was conducted by inviting stakeholders from the British Society of Gastroenterology through a 2-phase process. Participants were asked to select important clinically relevant outcomes and were asked about what magnitude of the effect that they consider large, moderate, small, or trivial for each clinical trial outcome in line with the GRADE guidance. The results were fed back to all participants to ensure consensus agreement. Then, further surveys were sent to Europe and North America to ensure validity and international triangulation of the dataset. Data are presented as mean ± SD. RESULTS: A total of 131 clinical stakeholders participated, including clinicians, IBD nurses, and a small number of patients with IBD. Clinical remission and serious adverse events were considered the most critical outcomes for Crohn's disease, while clinical remission and endoscopic remission were considered the most critical outcomes for ulcerative colitis. The consensus results for thresholds of small, moderate, and large outcome effect sizes were agreed on as follows: clinical remission, 11 ± 6%, 20 ± 8%, and 31 ± 13%; endoscopic remission, 9 ± 5%, 17 ± 9%, and 28 ± 14%; and serious adverse events 6 ± 6%, 11 ± 9%, and 17 ± 12%, respectively. No significant differences were observed for responses for each condition. CONCLUSIONS: This is the first study to develop a consensus on magnitude thresholds for outcomes in IBD. These thresholds have been used in the development of the 2024 British Society of Gastroenterology guidelines for the management of IBD but can and should also be used by study designers and, most importantly, by clinicians when discussing evidence with patients as part of shared decision making. Future work to validate these findings globally and with other groups, including patients, 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 imitationNot 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.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.428 | 0.655 |
| Meta-epidemiology (narrow) | 0.002 | 0.002 |
| Meta-epidemiology (broad) | 0.003 | 0.006 |
| Bibliometrics | 0.018 | 0.008 |
| Science and technology studies | 0.002 | 0.005 |
| Scholarly communication | 0.010 | 0.013 |
| Open science | 0.005 | 0.011 |
| Research integrity | 0.004 | 0.007 |
| Insufficient payload (model declined to judge) | 0.005 | 0.001 |
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.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
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".