330. TREATMENT RESPONSE CRITERIA FOR ANCA-ASSOCIATED VASCULITIS: RESULTS OF A SCOPING REVIEW
Notice bibliographique
Résumé
Background: A review of outcome measures to assess response to treatment in ANCA-associated vasculitis (AAV) will help advance methodology for clinical trials in this disease. Methods: As part of an ongoing international project to develop response criteria, we performed a scoping review to assess the tools used as outcome measures in randomized clinical trials (RCTs) of AAV. Medline, Embase, Cochrane Central, and ClinicalTrials.gov were searched from inception until November 2018 to identify RCTs enrolling patients with granulomatosis with polyangiitis and/or microscopic polyangiitis. Results: Among the 24 RCTs included in the review (Figure 1), various versions of the Birmingham Vasculitis Activity Score (BVAS) were the most widely used instruments for disease assessment. BVAS was almost always reduced to a dichotomous variable (0 or > 0) providing distinction between remission and active disease. Reduction in BVAS and/or achievement of BVAS=0 represented a main study outcome (primary or secondary endpoint) in 20/24 (83%) RCTs; 6 of these trials used the BVAS/WG. Damage, mainly assessed by the Vasculitis Damage Index (VDI), was an outcome for 14/24 (58%) RCTs. Physician global assessment and patient-reported outcomes (PROs) [measures of health-related quality of life (HRQoL) and/or patient global assessment] were assessed in 7 (29%) and 14 (58%) RCTs, respectively. Assessment of renal function or activity was a major outcome or specifically included in definitions of remission/relapse in 23/24 (96%) RCTs. Timing for outcome measure assessment differed substantially, with baseline, 6 months (15/20 RCTs), and 12 months (14/20) after enrollment being the most common time points for reporting BVAS and VDI. Assessment of disease state occurred as early as 1-4 weeks after enrollment, with 6/24 (25%) RCTs assessing disease activity at 6 weeks and 13/24 (54%) at 3 months. Conclusion: Outcome measures used in RCTs of AAV include the repeated use of vasculitis specific tools to assess disease state, but with heterogeneity in the definitions for remission/relapse and timing of assessment. Intermediate states of disease activity are currently poorly defined or evaluated. Furthermore, other important outcomes in AAV, including PROs, damage measures, and global assessments are often not included as primary or confirmatory secondary outcomes in RCTs in AAV. This review highlights the need for more homogeneous outcome assessment in RCTs for AAV and the current lack of a composite measure that integrates various endpoints relevant to physicians and patients. Overview of outcomes assessed in randomized controlled trials of ANCA-associated vasculitis Disclosures: Dr. Robin Christensen reports to be, as an employee at the Parker Institute, Bispebjerg and Frederiksberg Hospital (RC), supported by a core grant from the Oak Foundation (OCAY-13-309)Dr. Alfred Mahr received consultant fees and speaker honoraria from Celgene and Roche Chugai.Dr. Pagnoux reports receiving funds for the following activities: Consulting: ChemoCentryx, Genentech/Roche, Genzyme/Sanofi.Dr. Merkel reports receiving funds for the following activities: Consulting: AbbVie, Biogen, AstraZeneca, Boeringher-Ingelheim, Bristol-Myers Squibb, Celgene, ChemoCentryx, Genentech/Roche, Genzyme/Sanofi, GlaxoSmithKline, InflaRx, Insmed, Jannsen, Kiniksa; Research Support: AstraZeneca, Boeringher-Ingelheim, Bristol-Myers Squibb, Celgene, ChemoCentryx, Genentech/Roche, GlaxoSmithKline, Kypha, TerumoBCT; Royalties: UpToDateDr. David Jayne has received research grants from Chemocentryx, GSK, Roche/Genentech and Sanofi-Genzyme. He has received consultancy fees from Astra-Zeneca, Boehringer-Ingelheim, Celgene, ChemoCentryx, Chugai, GSK, Infla-RX, Insmed and Takeda.
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 | 0,005 | 0,013 |
| Méta-épidémiologie (sens strict) | 0,001 | 0,001 |
| Méta-épidémiologie (sens large) | 0,005 | 0,004 |
| Bibliométrie | 0,009 | 0,007 |
| Études des sciences et des technologies | 0,001 | 0,001 |
| Communication savante | 0,002 | 0,002 |
| Science ouverte | 0,001 | 0,001 |
| Intégrité de la recherche | 0,002 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,009 | 0,002 |
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 ».