PB1980 IMPROVING HEALTHCARE PROVIDERS’ KNOWLEDGE REGARDING THE PRINCIPLES OF GENE THERAPY FOR HEMOPHILIA THROUGH ONLINE EDUCATION
Bibliographic record
Abstract
Background: Gene therapy offers the hope of a functional cure for hemophilia. To prepare healthcare providers (HCPs) for this potential paradigm shift in care, National Hemophilia Foundation (NHF), European Haemophilia Consortium (EHC), the World Federation of Hemophilia (WFH), and Medscape Education collaborated to develop a multi‐modal, online, continuing medical education (CME) curriculum. Aims: The current study assessed the ability of online CME to improve HCPs knowledge regarding the scientific principles underlying gene therapy overall and its study in hemophilia. Methods: A 15‐minute, CME‐certified video commentary with synchronized slides was developed and launched online on 8/3/2018. Educational effectiveness was assessed with a repeated‐pairs pre‐/post‐assessment study design, in which each individual served as his/her own control. Responses to 3 multiple‐choice, knowledge questions and 1 self‐efficacy confidence question were analyzed. A chi‐squared test assessed changes pre‐ to post‐assessment. P values <0.05 are statistically significant. Effect sizes were evaluated using Cramer's V (<0.05 modest; 0.06‐0.15 noticeable effect; 0.16‐0.26 considerable effect; >0.26 extensive effect). Results: To date, 1634 HCPs (958 physicians) have participated in this activity. This analysis presents data from the subset of hematologists/oncologists (n = 77; hem/oncs) and pediatric oncologists (n = 69) who answered all pre‐/post‐assessment questions during the initial analysis period of 8/3/18‐9/5/18; data collection is ongoing. Significant improvements were observed overall (hem/oncs: P = .0007; V = .156) and in the following areas, ‐ Correct identification of transient transaminitis as the most common adverse event observed in gene therapy trials: hem/oncs: 45% vs 73% (62% relative increase), P = 0.0006, V = .277; and pediatric hematologists: 26% vs 43% (% relative increase), P = .032, V = .181 ‐ Recognition of the relatively low homology between the adeno‐associated virus (AAV) vector used in gene therapy trials for hemophilia A (AAV5) and other primate AAVs: hem/oncs: 36% vs 55% (53% relative increase), P = 0.023, V = .182 ‐ Pediatric hematologists exhibited a small increase in knowledge, but this change was not statistically significant: 33% vs 41% (24% relative increase), P = NS ‐ Both of hem/oncs (39%) and pediatric hematologists (32%) indicated increased confidence in their understanding of the science underlying gene therapy in hemophilia Lastly, the findings uncovered educational needs, such as the understanding of the FIX construct currently under study in hemophilia B trials. Summary/Conclusion: Participation in this online activity significantly improved knowledge among hem/oncs and pediatric hematologists with regard to the adverse events, viral vectors, and gene constructs in current clinical trials for gene therapy in hemophilia.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
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 teacher head, 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".