Understanding Health Literacy Among Patients with Thalassemia: A Global Patient Survey by the Thalassemia Advocacy Advisory Council

Background: Thalassemia is a rare, under-recognized hereditary hemolytic anemia with a variable clinical presentation, resulting in a variety of symptoms and complications that can substantially impact patient health-related quality of life. Despite advances in the management of thalassemia, unmet needs remain in this diverse patient population. The Thalassemia Advocacy Advisory Council (AAC), an international group comprising patients, caregivers, advocacy organizations, and healthcare professionals (supported by Agios Pharmaceuticals) was launched to define and better understand the unmet needs of the thalassemia community, as well as to support improvement in patient outcomes and care. Following an initial assessment to understand priority areas, health literacy (ie, understanding of thalassemia, its complications, and treatment approaches) was identified as a key unmet need for patients with thalassemia. Subsequently, the results of an evidence audit of published literature and advocacy group/community-based research on health literacy in thalassemia further demonstrated unmet informational and educational needs, which informed key areas for exploration. Based on these findings, the Thalassemia AAC developed a patient survey, with the aim of better understanding the global community's perspectives and identifying strategies to potentially address patients' health literacy needs. Here, we describe the methodology of this global patient survey. Methods: This initiative utilizes a bespoke, self-administered, approximately 12-15-minute survey developed by the Thalassemia AAC. Adults (aged ≥18 years) with a diagnosis of alpha- or beta-thalassemia are eligible to participate in this study, excluding those diagnosed with alpha- or beta-thalassemia trait or those who are currently enrolled in a clinical trial involving mitapivat (for example, ENERGIZE [NCT04770753] or ENERGIZE-T [NCT04770779]). All participants must provide informed consent before taking part in the survey. Approximately 105 participants from the Middle East (United Arab Emirates, Saudi Arabia, Qatar, Bahrain, Kuwait, Oman), Italy, USA, Greece, and Brazil are planned to be recruited via two recruitment streams: 1) a specialist survey recruitment agency, or 2) a patient advocacy organization network. The primary objective is to describe health literacy in patients with thalassemia; secondary objectives include studying barriers and motivational aspects that affect disease understanding and self-management, and how these survey findings compare with previously conducted research. The survey comprises six sections: (i) Screening (including patient age and clinical characteristics); (ii) Demographics (including optional questions), to better understand the patient profile; (iii) Channels and formats, to understand where respondents search for information and their preferred content format; (iv) Disease knowledge, to collect information on disease understanding; (v) Challenges and barriers, to understand what drives the patient to seek a greater understanding and self-management of their disease; and (vi) Motivators, to uncover any positive external factors that influence the patient to seek an increased understanding and self-management of their disease. Participants complete the survey online and data are captured electronically. Survey responses will be summarized descriptively as number and percentage for categorical variables, and mean with standard deviation or median with interquartile range for continuous variables. Qualitative, free text responses will be reviewed and categorized. A limitation of this survey is that its questions are bespoke and have not been validated. Results: The survey is in progress and available results are currently being analyzed. Conclusions: The Thalassemia AAC identified health literacy as a key unmet need for patients with thalassemia. This global patient survey will provide insight into the current state of health literacy (ie, knowledge of thalassemia, its complications, and treatment approaches) in the patient community, to support future improvement of thalassemia patient outcomes and care.