P498 Ustekinumab Real World Evidence Study (UndieS): study design with information about positioning and the choice of ustekinumab
Notice bibliographique
Résumé
Abstract Background Strict eligibility criteria of phase 3 trials limit the external validity of their results. Well-designed real-world studies are intended to complement these by describing the effectiveness and safety of these medicines in routine clinical practice. As therapeutic options for the treatment of Crohn’s disease (CD) expands, the ideal sequence of medications, and the influences on this, needs defining. Methods UndieS is a phase IV, multi-centre, prospective, observational study of patients with CD initiated on ustekinumab as part of their routine care. 600 participants will be recruited from 22 centres across the UK with a follow-up period of 2 years. Assessments at baseline and weeks 8, 16/20, 56, 80 and 104 include modified Harvey-Bradshaw Index (mHBI), IBD-Control, Treatment Satisfaction Questionnaire for Medication and PRO-2. The primary outcome is steroid-free remission (mHBI ≤ 4) at one year. Secondary outcome measures include describing changes in disease activity, PROs and tolerability. Results Recruitment started in March 2020 with 437 patients recruited so far. The summary baseline characteristics are 50% male, median (range) age at consent 41 (18-91) years (14% > 65 years), median mHBI 5 (0-39). The median duration of Crohn’s disease before starting ustekinumab was 8 (0-60) years. Disease location (as per Montreal classification) is: L1: 35%, L2: 20%, L3: 44% and isolated L4: 1%. 24% of participants reported perianal disease. The proportion of participants using concomitant immunomodulators or oral corticosteroids on starting ustekinumab (15% and 11% respectively) is lower than in the UNITI clinical trial program. 59% of participants had a disease activity score suggesting that they would not meet the threshold required for entry into UNITI and suggested by NICE for moderately-to-severely active CD, although 81% of these had evidence of inflammation. Inadequate response to or adverse effects from a previous treatment was a common indication (52% of participants). 32% of the participants are bio-naïve; 64% and 10% have previous exposure to anti-TNF and vedolizumab respectively and 6% have been exposed to both. Details of prior biologic exposure is shown in figure 1. The most common reasons for the specific choice of ustekinumab were intolerance to immunomodulators and concern about infection risk. Bio-naïve patients were older (median 45 vs. 39 years; p=0.02) and had a shorter disease duration (7 vs. 9 years; p=0.04) than those with prior biologic exposure. Conclusion Our cohort is representative of those patients with CD being treated with ustekinumab in a real-world setting. Some important differences between these patients and those recruited to phase 3 clinical trials are identified.
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 | 0,032 | 0,027 |
| Méta-épidémiologie (sens strict) | 0,002 | 0,001 |
| Méta-épidémiologie (sens large) | 0,003 | 0,002 |
| Bibliométrie | 0,001 | 0,002 |
| Études des sciences et des technologies | 0,001 | 0,002 |
| Communication savante | 0,002 | 0,002 |
| Science ouverte | 0,001 | 0,002 |
| Intégrité de la recherche | 0,002 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,026 | 0,004 |
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 ».