P44: Knowledge and Perception of Gene Therapy among Spanish Speaking Patients and Caregivers in the Bleeding Disorders Community
Poster Presenter
Mabel Crescioni
Director, Public Health & Outcomes Research
Hemophilia Federation of America United States
Objectives
The objective of this study is to understand Spanish speaking bleeding disorder patients and/or their caregivers’ knowledge of gene therapy and their preference of how, and from whom, they would like to acquire this knowledge.
Method
For this study, an online instrument was developed in Spanish using Likert-type scales, multiple choice, and open-ended questions. Surveys (n=64) were collected from August 2020-September 2020. Descriptive and qualitative analysis were conducted.
Results
Overall, a little over half (56%) of the respondents indicated only knowing a little bit about or not being able to explain about gene therapy; about 20% could clearly explain or had extensive knowledge; and 22% never heard the term. The majority of respondents (59%) were excited about the potential for gene therapy to improve life for people with bleeding disorders. When caregivers were asked if they would consider using an approved gene therapy to treat their or their child’s hemophilia, 36% responded “yes” and 12% of caregivers replied that they would leave it up to their child. The top three qualitative responses of what effect gene therapy would have for people with hemophilia included being unsure of the effect; gene therapy having a positive effect; and hopes for a cure or that the body would be able to produce enough factor. Cost, insurance coverage, and the potential risks of gene therapy were all top concerns. The preferred language to learn about gene therapy was Spanish (69%). When asked to select the preferred methods for learning about gene therapy from a discrete list of options, the type five responses were: online education (n=33), educational videos (n=31), speaking to knowledgeable community members (n=31), attending in-person meetings/sessions (n=30), and webinars (n=30). Participants were also asked to select their most trusted sources for gene therapy education from a discrete list. The top five responses were Hemophilia Federation of America (n=41), National Hemophilia Foundation (n=29), local member organizations (n=24), healthcare providers (n=19), and lastly, Centers for Disease Control and Prevention and the Food and Drug Administration (both at n=16). Even though healthcare providers were one of the top responses when asked about trustworthy sources for hemophilia and gene therapy education, only 28% of respondents thought that their hematologist knew enough about gene therapy to answer their questions and 58% were unsure.
Conclusion
Potential gene therapy topics that can be addressed in the future include gene therapy basics; approved therapies for bleeding disorders; benefits/risks of potential gene therapies; and health insurance cost and coverage. Preferred methods of learning could include a hybrid of virtual sessions, in-person meetings, and self-paced learning/modules. This approach was different from results from a previous English survey where respondents preferred in-person learning. The preference to virtual learning could be attributed to the survey being conducted during the COVID-19 pandemic and the related importance of practicing social distancing and stay-at-home orders. As trusted sources, it is important that healthcare providers, national patient advocacy organizations, and government organizations have the education and resources needed to engage patients and caregivers on gene therapy education in Spanish. Lastly, only 28% of respondents thought that their provider was knowledgeable enough to discuss gene therapy, presenting the opportunity to not only understand if providers are knowledgeable about gene therapy, but also, how, or if, they educate their Spanish speaking patients on the subject.
