High Health Care Burden of Transfusion-Dependent β-Thalassemia

Results revealed that patients with transfusion-dependent β-thalassemia experienced 14.8 hospitalizations annually.

Image Credit: sudok1 - stock.adobe.com

Transfusion-dependent β-thalassemia (TDT) is associated with significant health care demands and clinical challenges, according to a recent study in France.¹ Analyzing data from the French National Health Data System from January 2012 to March 2019, researchers aimed to quantify the clinical burden, treatment needs, and health care utilization of patients with TDT, a rare genetic blood disorder characterized by the body's inability to produce sufficient hemoglobin.

In the retrospective, real-world claims database analysis, researchers identified 331 eligible patients with TDT through rigorous inclusion criteria, requiring evidence of 8 or more red blood cell (RBC) transfusion episodes annually over at least 2 consecutive years. Patients with coexisting conditions such as sickle cell disease or α-thalassemia were excluded to maintain a homogenous sample of TDT-specific cases. The study cohort had a mean age of 26.1 years and was almost equally split between male (50.5%) and female patients.

Complications and Accompanying Conditions

Patients with TDT commonly experienced various health complications, according to the findings. Endocrine disorders were the most frequent, affecting 26% of patients, followed by hepatobiliary (22.7%) and cardiopulmonary complications (18.7%). Notably, 15 patients (4.5%) died during the study period, translating to a mortality rate of 1.16 deaths per 100 person-years. The mean age at death was 52.5 years, highlighting the impact of TDT on life expectancy.

Assessing the Occurrence of Blood Transfusion

Managing TDT requires extensive medical intervention, particularly frequent blood transfusions, which often lead to iron overload and a variety of symptoms that affect multiple systems of the body, subsequently warranting iron chelation therapy.² On average, patients received 13.5 transfusions and 11.2 iron chelation treatments per year.¹ The researchers noted the need for iron chelation therapy was nearly universal, with 97.3% of patients receiving it as part of their treatment regimen.

Utilization of Health Care Resources

The results revealed that patients with TDT utilized health care resources at a high rate. On average, patients experienced 14.8 hospitalizations annually, with the majority being day cases lasting under 24 hours. Additionally, patients averaged 3.3 emergency department visits, 16.9 outpatient prescriptions, and 6.3 outpatient visits per year. Health care needs were more pronounced in adult patients, who required more inpatient hospitalizations (15.5 vs 13.7) and outpatient services (7.1 vs 4.9) compared with younger individuals.

The demand for inpatient care was largely influenced by the frequency of RBC transfusions; The study showed that patients who needed more than 16 transfusions per year required an average of 21.3 hospitalizations annually, whereas patients who needed fewer than 8 transfusions per year necessitated 6.5 hospitalizations. These results indicate the extensive health care burden placed on the system to manage TDT effectively.

"This study supports the progressive nature of TDT, as higher proportions of older patients (aged ≥ 18 years) experienced clinical complications compared with younger patients (aged < 18 years)," the study stated. "Our masking of prevalence data for clinical complications in younger patients may have limited our ability to compare these data between younger and older patients. However, we hypothesize that a larger sample size would yield a similar finding that TDT-related clinical complications increase with age, as other published studies on patients with TDT and associated pathophysiology have reported."

References

1. Baldwin J, Udeze C, Li N, et al. Clinical burden and healthcare resource utilization associated with managing transfusion-dependent β-thalassemia in France. Current Medical Research and Opinion, 40(8), 1289–1295. https://doi.org/10.1080/03007995.2024.2368197

2. Beta Thalassemia. National Organizations for Rare Disorders. Updated May 23, 2023. Accessed October 30, 2024. https://rarediseases.org/rare-diseases/thalassemia-major/