The Canadian Registry for Amyloidosis Research: A national multi-disciplinary registry for real-world evidence (P12-8.014)
Notice bibliographique
Résumé
Objective: The objective of the Canadian Registry for Amyloidosis Research (CRAR) is to improve the understanding of the Canadian amyloidosis landscape. CRAR aims to fill knowledge gaps to improve patient care and monitoring across Canada. Background: CRAR is a nationwide disease registry of transthyretin (ATTR) and light-chain (AL) amyloidosis. Recent advances in disease-modifying medical therapy have improved prognosis for both subtypes, however there is a critical need for real-world evidence to address knowledge gaps, particularly longer-term therapeutic outcomes and surveillance strategies. Design/Methods: A multi-stakeholder process was undertaken to develop a consensus dataset for ATTR- and AL-amyloidosis. This process included a review of existing registries, surveys to rank the importance of data items, and an in-person meeting of the CRAR steering committee, comprised of clinical experts in neurology, cardiology, and hematology, and patient organization representatives. Patients and patient organizations supported the development and implementation of a patient-reported dataset. Results: Consensus registry objectives include understanding real-world care of amyloidosis patients in Canada; defining the impact of treatments on patient outcomes; and empowering the Canadian community while providing a platform for collaborative research. Consensus data items include clinic-reported data on disease onset, progression, severity, treatments, and outcomes, as well as patient-reported outcomes. Both prospective and retrospective (including deceased) patient cohorts are included. Further baseline data will be presented on an initial cohort of patients. Conclusions: CRAR has been established to collect a prospective and retrospective, longitudinal, multidisciplinary dataset that will help evaluate ATTR and AL amyloidosis care and outcomes. CRAR has successfully launched at multiple sites and is expanding to additional specialty amyloidosis centres across Canada. The growth of this program will promote opportunities to perform multi-institutional observational studies that provide real-world efficacy and safety data and inform cost-effectiveness of therapies, while supporting patient recruitment for clinical trials and engaging patients in the collection of outcome data. Disclosure: Dr. Massie has received personal compensation in the range of $500-$4,999 for serving as a Expert consultant with INESS (Quebec Public Health agency). Dr. Massie has received personal compensation in the range of $500-$4,999 for serving as a Lecturer with Amylyx Pharmaceuticals. Diego Delgado has nothing to disclose. Dr. Venner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octapharma. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Powell Mansfield. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta (now J&J). Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NovoNordisk. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Bril has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Bril has received research support from UCB. The institution of Dr. Bril has received research support from Argenx. The institution of Dr. Bril has received research support from Momenta (now J&J). The institution of Dr. Bril has received research support from Immunovant. The institution of Dr. Bril has received research support from Alexion. The institution of Dr. Bril has received research support from Takeda. The institution of Dr. Bril has received research support from Grifols. The institution of Dr. Bril has received research support from CSL. The institution of Dr. Bril has received research support from Octapharma. The institution of Dr. Bril has received research support from Akcea. The institution of Dr. Bril has received research support from Ionis. The institution of Dr. Bril has received research support from Viela Bio (now Horizon). Dr. Bril has received intellectual property interests from a discovery or technology relating to health care. Donna Reece has nothing to disclose. Dr. Baker has nothing to disclose. Victoria Hodgkinson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe Pharma. Victoria Hodgkinson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dynacure. Victoria Hodgkinson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Victoria Hodgkinson has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Ms. Boyartchuk has nothing to disclose. Dr. Davis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astrazeneca/Ionis. Dr. Davis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Pfizer. Dr. Davis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Janssen. Dr. Davis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bayer. Nowell Fine has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Journal of the American College of Cardiology: Case Reports. The institution of Nowell Fine has received research support from Pfizer, Akcea.
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 | 0,087 | 0,264 |
| Méta-épidémiologie (sens strict) | 0,001 | 0,002 |
| Méta-épidémiologie (sens large) | 0,004 | 0,002 |
| Bibliométrie | 0,016 | 0,024 |
| Études des sciences et des technologies | 0,007 | 0,003 |
| Communication savante | 0,013 | 0,005 |
| Science ouverte | 0,008 | 0,009 |
| Intégrité de la recherche | 0,005 | 0,006 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,062 | 0,022 |
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 ».