Education about Transplant-Based Therapies in Sickle Cell Disease: Removing Barriers with Newer Technologies

Background: Sickle cell disease (SCD) affects about 100,000 people in the United States. Poor socioeconomic status, low literacy rates and lack of resources for SCD education have been previously reported (Thomas et al. ASH 2019). Yet publicly available videos explaining potentially curative therapies, such as hematopoietic stem cell transplant (HSCT) and HSC-based genomic therapies developed in the past decade garner fewer than 1000 views a month. These therapies are advancing at a rapid pace and optimizing patient outcomes. The low uptake of current video-based education about these complex therapies indicates that current educational model may not serve the sickle cell community (SCC) of patients, their families and caregivers. The accessibility gap in video education is concerning, given the barriers associated with text-based education, largely available in English and also frequently containing medical jargon. Further, these videos lack adaptivity and personalization to the diversity in starting points, questions, and concerns of those in the SCC, who could benefit from HSCT and HSC-based genomic therapies, especially children. A challenge of accessible, interactive education opportunities is the feasibility of medical professionals learning the specific starting points, questions, and concerns across a diverse community and feeding this information back into educational materials.

Methods: With the search for publicly available and accessible educational information, we identified the current knowledge gap regarding the role of stem cells and productions of defective red cells in SCD and understanding of HSC-based therapies for SCC. We explored the potential of different modalities preferred across varied age groups with diverse cultural and language backgrounds to meet patients' need to understand therapies and potential complications. These modalities included animation videos with characters appealing to children using two- and three-dimensional platforms and age-appropriate language. Learning modules will include a pre-quiz to facilitate assessment of prior knowledge, along with interactive quizzes and checkpoints to gauge understanding throughout the module. The scores from the pre- and post-quizzes will be used to evaluate the module's effectiveness in educating users about SCD and the role of transplant-based therapies. The goal is to create an interactive learning module to help identify knowledge gaps among patients, families and caregivers and develop an ongoing process to address these gaps.

Discussion: Education about HSC-based therapies across the SCC is critical in establishing a well-informed consent and empower families with better understanding of these therapies and their potential complications. Using a 3D model software, animations can be created either in a cartoon style targeted towards children or in a realistic rendering style suitable for adolescents and young adults. The focus of these animations is developing understanding of SCD with respect to HSC differentiation in the bone marrow, visualization of SCD pathophysiology and then address the underlying process with HSC-based therapies. We are further exploring augmented and virtual reality (AR/VR) tools across different platforms which can be integrated into interactive learning modules for patients and families to complete before their consultations, on their own time and in a comfortable setting. AI can be used to enhance accessibility by incorporating subtitles and different language narrations. Moreover, AR/VR can also accommodate different starting points, for example, where patients can choose the sequence and density of information (e.g., I need more information, I need to back up and get a definition) at given moments. This can further increase the accessibility of educational material for diverse population groups, effectively overcoming language and formal education barriers that often exist in healthcare environments. Additionally, AR/VR tools can provide the flexibility of initiating the education at different starting points.

Conclusion: Development of educational content for transplant-based therapies for SCD can overcome knowledge gaps and accessibility challenges by incorporating newer tools such as AI, AR/VR to expand the accessibility and improve user experience by adjusting the density and sequence of content as well as its delivery method.

Anandaraj:Medtronics: Current Employment. Blaylark:Bluebird Bio: Membership on an entity's Board of Directors or advisory committees; Agios: Membership on an entity's Board of Directors or advisory committees. Wagner:Vertex Pharmaceuticals: Consultancy; Garuda Therapeutics: Membership on an entity's Board of Directors or advisory committees; Rocket Pharma: Consultancy, Current equity holder in private company, Membership on an entity's Board of Directors or advisory committees; ASC Therapeutics: Membership on an entity's Board of Directors or advisory committees; Magenta Therapeutics: Current equity holder in private company. Gupta:Vertex Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees; Beam Therapeutics: Research Funding; Jazz Pharmaceuticals: Research Funding; Emerging Therapies Solutions: Speakers Bureau; bluebird bio, Inc.: Research Funding; Orchard Therapeutics: Research Funding.