Lifespan treatment in transfusion-dependent β-thalassemia (TDT) is expected to impact quality of life. This study aimed at evaluating health-related quality of life (HRQoL), well-being, and the burden of TDT on Italian patients. Patients (≥14 years) were invited to complete a cross-sectional, online volunteer survey. HRQoL was measured by the 36-item short-form health survey (SF-36) and wellbeing was measured by the Italian version of the Psychological General-Well-Being-Index (PGWBI). A total of 105/167 completed questionnaires were analyzed (46% males; median age 44, (IQR = 11)). Patients reported lower HRQoL compared with the general population in all SF-36 domains (except for emotional well-being (p = 0.7024) and role limitations due to emotional problems (p = 0.1389)). PGWBI domains general health and vitality and the total PGWBI score were all significantly lower (p = 0.0001) compared with the general population. On average, patients spent 16.62 h/month engaged in care activities that were additional to the time required for completing transfusions. Of the 16.62 h/month, 11.7 h/month were required for therapy management and 4.92 h/month for family management. This study found lower HRQoL and well-being in physical and psychological domains compared with the general population. Further, patients were found to have a high perceived burden of disease.
Keywords: PGWBI; SF-36; burden of disease; health-related quality of life; iron chelation therapy; physical health; psychological health; psychological well-being; β-thalassemia transfusion-dependent.