Understanding health literacy among patients with thalassemia: Results from a global patient survey by the Thalassemia Advocacy Advisory Council

Abstract Introduction: Despite advances in the management of thalassemia, a rare and under-recognized hereditary anemia, unmet needs remain in this diverse patient population. The Thalassemia Advocacy Advisory Council (AAC), an international group of patients, caregivers, advocacy organizations, and healthcare professionals (HCPs), supported by Agios Pharmaceuticals, was formed as a novel approach to better understand these needs and support initiatives to potentially improve outcomes and care. An assessment and subsequent evidence audit of published literature and advocacy group/community-based research identified health literacy (i.e., understanding of thalassemia, its complications, and treatment approaches) as a critical gap for patients. Based on the findings, the Thalassemia AAC developed a patient survey to gain insights into the global community's perspectives and to identify strategies to potentially address health literacy needs and support informed patient advocacy. Here, we report the results from the Thalassemia AAC global patient survey. Methods: A bespoke, 12–15-minute survey was self-administered to adults (≥18 years) with a self-reported physician diagnosis of alpha (α)- or beta (β)-thalassemia, excluding those diagnosed with α- or β-thalassemia trait or those currently enrolled in mitapivat clinical trials (e.g., ENERGIZE [NCT04770753] or ENERGIZE-T [NCT04770779]). Participants from the United States, Brazil, Italy, Greece, the United Arab Emirates, Saudi Arabia, and Kuwait were recruited via a specialist survey recruitment agency or a patient advocacy organization network. All participants provided informed consent. The primary objective was to describe health literacy in patients with thalassemia; secondary objectives included studying barriers and motivational aspects that affect disease understanding and self-management. The survey comprised six sections and was completed online. Survey responses were summarized as number and percentage for categorical variables, and as net percentages for continuous variables. Results: In total, 122 patients with thalassemia from the United States (n=25), Brazil (n=25), Italy (n=25), Greece (n=15), the United Arab Emirates (n=15), Saudi Arabia (n=10), and Kuwait (n=7) participated in the survey. Participants reported a high level of confidence in their knowledge of thalassemia, with the majority stating that they were confident regarding their understanding of the symptoms of thalassemia (85%, n=104) and living with the disease (89%, n=109). Additionally, almost half of participants (43%, n=53) stated that their knowledge of thalassemia had improved a lot over the previous 5 years. Almost all participants (98%, n=119) recognized that if not treated properly, thalassemia can lead to serious complications that may require future treatment. However, only a small minority of participants (14%, n=17) were aware that patients with non-transfusion-dependent thalassemia require monitoring for iron overload. Even fewer participants (6%, n=7) correctly recognized that a hemoglobin level of ≤10 g/dL is associated with an increased complication risk; more than half (53%, n=65) incorrectly selected a threshold of ≤7 g/dL. Among a variety of sources, including internet search engines, social media, and peers, HCPs were the most frequently reported source of thalassemia management information, with two-thirds (67%, n=82) identifying them as their most trusted source. Conclusions: Despite participants reporting high confidence in disease understanding, several key knowledge gaps were identified. Of particular importance, most patients were unaware of the hemoglobin level associated with increased rates of complications, and the need for regular monitoring of patients with non-transfusion-dependent thalassemia. The responses regarding hemoglobin may reflect the lower treatment targets still used by many providers, and therefore a lack of awareness among HCPs. Overall, these findings suggest a potential overestimation of disease understanding among respondents. Participants' use of HCPs as a trusted source of thalassemia information emphasizes that efforts to support clinicians with patient education may provide an opportunity to help address these knowledge gaps.