Developing IBD Outcome Effect Size Thresholds to Inform Research, Guidelines, and Clinical Decisions
Notice bibliographique
Résumé
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.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,428 | 0,655 |
| Méta-épidémiologie (sens strict) | 0,002 | 0,002 |
| Méta-épidémiologie (sens large) | 0,003 | 0,006 |
| Bibliométrie | 0,018 | 0,008 |
| Études des sciences et des technologies | 0,002 | 0,005 |
| Communication savante | 0,010 | 0,013 |
| Science ouverte | 0,005 | 0,011 |
| Intégrité de la recherche | 0,004 | 0,007 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,005 | 0,001 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».