Addressing Thalassaemia Management from a Patients’ Perspective: an International Collaborative Assessment

Abstract

Background and objectives: Effective management for chronic diseases, hereditary and rare diseases and Thalassaemia in particular constitutes an important indicator for the quality of healthcare systems. We aimed at assessing healthcare services in different countries by using publicly available health indicators and by surveying Thalassaemia patients and their caregivers. Materials and Methods: We reviewed official worldwide databases from the WHO, World Bank, and scientific resources and also used a structured patient tailored self-completed questionnaire to survey Thalassaemia patients and their caregivers in 2023. Results: A total of 2,082 participants were surveyed (mean age 27 years, males 42%). About 1 in 4 respondents had less than high school education, while 24% had a bachelor degree. About a third of respondents were married and had either full- or part-time employment. The vast majority (~80%) had initiated transfusion therapy between 1 – 4 years of age. Only 42% reported no delays in receiving blood transfusion, while 47% reported occasional delays and 8% serious delays. About half of patients reported being very satisfied (11%) or satisfied (38%) with the quality of provided services, while 1 in 3 patients reported being unsatisfied or very unsatisfied, and their access to treatment was difficult or very difficult due to traveling expenses and high cost of treatment. Conclusion: Important improvements in the care of Thalassaemia patients have been documented during the past decades. Nevertheless, our recent survey reveals many unmet needs where additional focus is required through national healthcare systems in order to address them effectively and achieve satisfactory patient outcomes.