Investigating Health Literacy in Thalassemia: Founding a Patient-Led Research Approach
Authors: Sheth Sujit , Colasanti Ralph , Cannon Lily , Deitrick Jill , John Holly , Gilroy Keely S , Lombard Louise , Michalaki Eleni , Larkin Tobias , Levine Laurice M , Steagall Dina , Cappellini Maria Domenica 
Published: December 2023

Background:
Thalassemia is a hereditary hemolytic anemia (HHA) with a variable phenotype and clinical severity, and constantly changing demographic profile. Despite advances in management, there are many unmet needs in this diverse population, with needs varying among different groups.
The Thalassemia Advocacy Advisory Council (Thal AAC) was founded to define and better understand the unmet needs of the thalassemia patient community. This ten-person group consists of patients, caregivers, advocates, and healthcare professionals (HCPs) from six countries (USA N=5, Cyprus N=1, Italy N=1, Greece N=1, Taiwan N=1, Brazil N=1). We describe how the Thal AAC sought to better define the global community's unmet needs following a multi-step process of collecting, reviewing, and prioritizing the unmet needs experienced by patients with thalassemia (Figure). The process was coordinated, moderated, and supported by Agios Pharmaceuticals.
Method:
Thal AAC members shared their personal perspective on community unmet needs through a qualitative survey, which included open-ended questions aiming to collect each member's perspective on the top three unmet needs for patients and HCPs in their region and globally. Results were consolidated and reviewed by the AAC in a series of workshops. Two key unmet needs were highlighted: Among patients, there is a lack of understanding of the disease and both long- and short-term complications, which can lead to poor adherence to standard of careAmongst HCPs, improved education and advocacy are needed to support patient access to care, including innovative therapy options and research
Based on these findings, the group aligned on the need for better education and understanding of the disease from a patient and HCP perspective, with health literacy (HL) being the central theme that tied these points together.
With the goal of broadening understanding of patient HL, the Thal AAC defined four key sub-topics to investigate further: Patients' knowledge of the diseaseChallenges faced by patients in improving their HLPossible motivators for improving HLChannels and formats through which patients seek disease information.
A non-exhaustive evidence audit of English-language literature published in the last 10 years on HL in thalassemia was conducted within PubMed and Cochrane databases using key search terms targeting thalassemia, similar hereditary hemolytic diseases, and HL. The published literature, was supplemented with patient advocacy group knowledge and data from other community-based research.
Results:
The audit showed that there is limited published literature or evidence for HL in the context of thalassemia and related hemolytic anemias (N=58). The format and delivery of information emerged as an important research topic in the context of rare diseases generally, but not thalassemia specifically. Although barriers and challenges to improving HL were studied in the literature (N=9), thalassemia-related items make up only ~10% (N=1), and there is relatively no research into the motivators for improving HL related to disease and outcomes. Thus, patients and their HCPs share unmet informational and educational needs, particularly regarding the long-term implications of inadequate treatment and care.
Conclusion:
The findings of the audit indicate a clear gap in thalassemia HL research. This gap may create scenarios in which not every patient with thalassemia accesses optimal care, which may have consequences for their long-term health. This provided the impetus for the AAC to launch a community-based, multi-national patient advocacy research initiative, comprising of a global survey and follow-up qualitative interviews, to better understand the community's perspective on HL in thalassemia. Insights from this survey will be used to share learnings and calls to action with community stakeholders, with an aim of contributing to the improvement of thalassemia patient outcomes and care. Thus, the AAC's findings have led to new insights which, in turn, have formed the foundation of a community-based research project.