Addressing Thalassaemia Management from Patients' Perspectives: An International Collaborative Assessment

Area of Science:

• Public Health
• Hematology
• Healthcare Management

Background:

• Effective chronic disease management, including hereditary and rare diseases like thalassaemia, is crucial for healthcare system quality.
• Assessing healthcare services for thalassaemia patients globally is essential for identifying areas of improvement.

Purpose of the Study:

• To evaluate healthcare services for thalassaemia patients across different countries.
• To analyze patient and caregiver experiences alongside public health indicators.

Main Methods:

• Review of global databases (WHO, World Bank) and scientific literature.
• A structured, patient-tailored questionnaire survey of 2082 thalassaemia patients and caregivers in 2023.

Main Results:

• Most patients initiated transfusion therapy early (1-4 years), but 50% experienced delays.
• Patient satisfaction with services was mixed, with significant dissatisfaction regarding access due to travel and treatment costs.
• Educational attainment varied, with 25% not completing high school and 24% holding bachelor's degrees.

Conclusions:

• While thalassaemia care has improved over decades, significant unmet needs persist.
• National healthcare systems must address access barriers, including financial and logistical challenges, to improve patient outcomes.
• Further focus is needed to achieve satisfactory patient outcomes and enhance the quality of care for thalassaemia patients.