The Canadian Registry for Amyloidosis Research: A national multi-disciplinary registry for real-world evidence (P12-8.014)
Bibliographic record
Abstract
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.
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 | 0.087 | 0.264 |
| Meta-epidemiology (narrow) | 0.001 | 0.002 |
| Meta-epidemiology (broad) | 0.004 | 0.002 |
| Bibliometrics | 0.016 | 0.024 |
| Science and technology studies | 0.007 | 0.003 |
| Scholarly communication | 0.013 | 0.005 |
| Open science | 0.008 | 0.009 |
| Research integrity | 0.005 | 0.006 |
| Insufficient payload (model declined to judge) | 0.062 | 0.022 |
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".