Disease Burden in Transfusion-Dependent β-Thalassemia (TDT) Patients and Caregivers in Italy: Results of a Cross-Sectional Descriptive Online Survey

Background: Patients with transfusion-dependent thalassemia (TDT) require lifelong blood transfusions; data on the psychosocial and economic impact on patients and their caregivers remain limited. The study aims to address this gap by describing the burden of TDT on pediatric, young, and adult patients, as well as their caregivers, within real-world settings in Italy. Methodology: This is a cross-sectional, descriptive study conducted between May and December 2021. TDT patients and their caregivers from Italian sites were invited to complete an online survey with the Ratip and Modell questionnaire (1996) and another bespoke questionnaire. All subjects signed an online informed consent before responding to the questionnaires. Results: Seventy-nine subjects completed the survey: 59 patients (43 adults and 16 children) and 20 caregivers (the majority of adult patients were aged 41-50 years). The age at diagnosis differed between adults and pediatric patients, being earlier in the latter (0-2 months in 43.8%). Nearly half of patients received more than one transfusion/month. More than half of the patients experienced blood shortages, particularly during the summer. School absenteeism was reported as a major issue. Conclusion: TDT continues to impose a significant burden, negatively impacting daily life, work, education, and social interactions. The availability of blood resources remains a critical issue, particularly in high-prevalence regions. The study highlights the need for ongoing research, innovative approaches, and enhanced support to ensure improved well-being for TDT patients and their caregivers.