Addressing the Health Care Challenges of Transfusion-Dependent β-Thalassemia

Understanding the Burden of Transfusion-Dependent β-Thalassemia

Transfusion-dependent β-thalassemia (TDT) is a rare genetic blood disorder that significantly elevates health care demands. Healthcare providers in France examined data from the French National Health Data System between 2012 and 2019. The objective was to grasp the clinical burden, treatment necessities, and health care usage in patients diagnosed with TDT.

Clinical Insights from the Study

In this retrospective analysis, 331 patients meeting strict eligibility criteria were included, each requiring eight or more red blood cell (RBC) transfusions annually over two consecutive years. Patients with additional conditions such as sickle cell disease were excluded, ensuring a focused study group specific to TDT. The cohort’s average age was 26.1 years, with a near-equal distribution of male and female patients.

Health Complications Frequently Encountered

• Endocrine disorders: 26% of patients experienced these.
• Hepatobiliary complications: 22.7% were affected.
• Cardiopulmonary issues: 18.7% of patients faced related health problems.

During the study, there were 15 patient deaths, indicating a mortality rate of 1.16 deaths per 100 person-years. The average age at death was 52.5 years, underlining the severe effects of TDT on life expectancy.

Blood Transfusion Management

Management of TDT necessitates frequent RBC transfusions, which can lead to iron overload problems. On average, patients received 13.5 transfusions and 11.2 iron chelation treatments yearly. Notably, 97.3% of patients required iron chelation as an integral part of their treatment.

Resource Utilization Among TDT Patients

Patients presented a substantial utilization of health care resources. Annually, they averaged 14.8 hospitalizations, primarily for day cases lasting under 24 hours. Furthermore, they experienced 3.3 emergency department visits, received 16.9 outpatient prescriptions, and attended 6.3 outpatient visits per year. Adult patients exhibited a greater need for both inpatient and outpatient services compared to younger cohorts, indicating a growing demand for health care.

The intensity of inpatient care directly correlated with transfusion frequency. Patients needing more than 16 transfusions annually required about 21.3 hospitalizations, while those needing fewer than 8 transfusions averaged only 6.5 hospitalizations.

Overall, this analysis demonstrates the remaining health care burdens and highlights a clear need for enhanced management solutions for TDT patients.