Exploring Patient, Caregiver Perceptions of Thalassemia Care

Several surveys focusing on specific health aspects of thalassemia patients have been published in medical literature.1-3 Yet, there is limited data on the quality and quantity of healthcare services for such patients.4

To better understand thalassemia treatment and care, Eleftheria C. Economidou, MD, of the department of pediatrics at Larnaca General Hospital in Larnaca, Cyprus, and colleagues conducted a large international survey capturing patient and caregiver perspectives from 48 different countries.4 “To our knowledge, this is one of the first international surveys of thalassaemia patients and their caregivers, conducted in 2023, aiming to capture the extent as well as the quality and quantity of healthcare services provided to this specific patient population in various countries around the world,” the study authors wrote in their report.

Results from the study were published in Medicina. The authors of the publication represent the Thalassemia International Federation (TIF), an international nonprofit, nongovernmental organization comprised of more than 200 national patient support organizations, headquartered in Cyprus.

Key Findings From the Survey

According to the researchers, most thalassemia patients worldwide are receiving adequate treatment and care, including regular blood transfusions, chelation therapy, adequate monitoring, and appropriate imaging.4 Of the 2082 surveyed, 1647 (79.5%) began transfusion therapy between the ages of 1 and 4 years old and only 100 (4.8%) began therapy later than 10 years. A total of 49 (2.4%) were not transfusion-dependent. 

For those receiving iron chelation therapy, 885 (42.7%) started therapy between 1 and 4 years old and 359 (17.3%) began therapy later than 10 years. The majority of respondents (1470; 71.0%) receive iron chelation therapy regularly as prescribed and that they receive it at the right dose with continuous availability (1243; 60.0%).

In terms of delays for blood supplies at the centers where they were receiving treatment, 877 (42.3%) respondents stated they experienced no delays and 973 (47.0%) reported occasional delays.

Overall, approximately half of the patients reported being very satisfied (11%) or satisfied (38%) with the quality of healthcare services, while about a third of patients reported being unsatisfied or very unsatisfied with the quality of healthcare services.

Regarding access to treatment, one-third of patients reported that their access to treatment was difficult or very difficult on account of the high cost of treatment and the expenses associated with traveling to receive treatment. Similarly, more than half of the patients reported that they were missing 10 days or more per year from school or work to receive treatment (11 to 15 days, 18.7%; 16 or more days, 37.6%).

Furthermore, the researchers found that many patients still lack comprehensive services, face challenges in accessing guideline-specific recommended care, are not receiving timely care due to financial issues, and are not satisfied with the level of healthcare services they receive. 

Despite demonstrating improvements in many areas of social parameters and the medical management of thalassaemia patients compared to previous reports, the findings of the current survey document significantly fewer benefits, primarily in the area of prevention.

Addressing Unmet Needs

Over the past few decades, significant improvements in the care of thalassemia patients have occurred, but there are still unmet needs that need to be addressed. For continued progress, the authors are calling for greater attention from both the clinical and public health communities, as well as from policymakers worldwide.4

Additionally, they recommend the development and implementation of national prevention and control programs that promote, amongst other things, the development of reference centers that facilitate collaboration between healthcare professionals. 

“Considering that many thalassaemia patients around the world are managed in peripheral/rural centres with limited resources, the need for expert networking and tele-consultations has become imperative,” the authors noted in their study.

Importantly, the investigators acknowledged that these findings are an early assessment of global patient and caregiver perspectives, emphasizing the need for caution when interpreting the results. 

When assessing the data based on WHO regions, there were stark differences between the different WHO regions in all parameters examined. This included mean age distribution, marital and employment status, educational attainment, and access to health services and treatment. “Regions with better healthcare services, such as Europe, show a higher mean age of patients; higher educational, marital, and employment status; and better access to treatment that is provided by state healthcare services,” the study authors stated in their report.

Future Directions

Looking to the future, the authors, acting on behalf of the TIF, intend to sustain collaborations with bodies in the European Union and World Health Organization to advance public health and disease-specific policies for rare and hereditary blood disorders.4 “Despite demonstrating improvements in many areas of social parameters and the medical management of thalassaemia patients compared to previous reports, the findings of the current survey document significantly fewer benefits, primarily in the area of prevention,” they concluded in their report.

According to Dr Economidou and colleagues, TIF partners with clinical specialists in various medical specialties to enhance the quality of healthcare services for thalassemia patients globally, in addition to collaborating with universities and academic institutions to conduct scientific research, conferences, and publications in the field of thalassemia prevention, control, and management.


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