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 hemophiliaLastly, 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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