Marrow-ly we learn along: A needs assessment of the educational needs of pediatric and young adult patients with bone marrow failure

Abstract Diagnoses of bone marrow failure (BMF), whether acquired or inherited, are rare. The incidence of idiopathic aplastic anemia, the most common acquired cause of BMF, is 2-3 cases per million people (Shimano, K. et al. 2021) while the incidence of Fanconi anemia, the most common inherited cause of BMF is 1-5 per million people (Moore, CA et al. 2023). With such a rare and potentially life-threatening disease, ensuring access to accurate and age-appropriate educational resources for patients and their families is critical to their understanding of this diagnosis and complying with medical treatments. Our primary objective was to determine the gaps in currently available educational materials for pediatric and young adult patients diagnosed with bone marrow failure based on patient, caregiver, and provider feedback. Our second objective was to query what specific qualities patients, families, and providers are seeking in their educational materials and resources. Our team, composed of experts in bone marrow failure from across the United States, crafted a needs assessment survey for eliciting the opinions of pediatric and young adult patients diagnosed with bone marrow failure, their caregivers, and providers related to educational resources and materials they received or sought out from the time of diagnosis. Patients from birth to 25 years diagnosed with a form of bone marrow failure (inherited or acquired) were eligible. Patients 12 years or older or caregivers of patients of any age who met inclusion criteria were able to participate in the survey as a primary respondent. Providers of these patients were eligible to take a provider-directed version of the survey. Descriptive statistics were utilized for analysis. Twenty-nine providers of varying academic levels across the country responded to our survey. Overall, most providers were aware of patients and their families gaining information from three sources, an internet search (93%, 27/29), non-profit organization (62%, 18/29), or social media (72%, 21/29). Regarding format of learning material, providers believed that patients and their caregivers preferred to utilize pamphlets (62%, 18/29), websites of written materials (86%, 25/29), and social media (69%, 20/29). Most providers agreed pamphlets (83%, 24/29) and websites of written materials (72%, 21/29) were the most conducive to learning with social media was (34%, 10/29) the least. An individual analysis of these resources also found that providers' experiences aligned with these perceptions; qualitative data also collected in the survey particularly noted the utility of pamphlets and websites of written materials and the difficulties and challenges of using social media, videos, and books. Based on an interim analysis, caregiver and patient data collected thus far similarly reflect the findings of our provider survey. Overall, our interim analysis demonstrates notable trends in what providers of pediatric patients with BMF and their families, observe to be the most optimal types of resources for providing a grounded and thorough education on diagnosis and management of these complex conditions. As medical providers, we must ensure all patients and their families who receive a diagnosis of BMF promptly receive appropriate medical information regarding diagnosis and management. Efforts should be made to ensure that educational materials and resources are available in their preferred language and in the most accessible learning format. While society is becoming increasingly reliant on technology, it is notable that, based on provider opinion, most groups prefer to read words about their diagnosis, rather than watch a video or listen to content about it. Once all patient and caregiver survey data have been collected and analyzed, the next steps of our study will be to craft resources that seek to meet the needs identified through our mixed-methods project.