Objective: In β-thalassemia, a genetic blood disorder, patients can develop chronic anemia and become dependent on red blood cell transfusions (RBCT). We examined healthcare resource utilization (HCRU) and costs observed in treating transfusion-dependent β-thalassemia (TDβT) in the USA.

Methods: Cooley’s Anemia Foundation identified and recruited adult patients (aged ≥18 years), diagnosed with β-thalassemia, who had ≥1 RBCT in the previous 6 months, to participate in this study. Following informed consent in May 2021, patients completed standardized self-reported outcome instruments (including the Functional Assessment of Cancer Therapy–Anemia [FACT–An], Patient Health Questionnaire [PHQ-9], and Work Productivity and Activity Impairment [WPAI]) and questions about their demographics, treatment, inpatient and outpatient utilization, and other costs.

Results: A total of 100 patients participated. Mean age was 36.0 years (standard deviation [SD]=10.4) and 35% were male. Patients had been diagnosed with TDβT for an average of 34.4 years (SD=10.3). Among them, 98% had health insurance; 57% had commercial coverage, 24% Medicaid, and 24% Medicare. In the 6 months prior to assessment, patients experienced a mean of 9.6 (SD=4.3) transfusion events; >1 per month (66%), once monthly (31%), and every other month (3%). Reported TDβT-related HCRU included outpatient visits (92% of patients), emergency department visits (7%), inpatient stays (3%), and lab/blood test visits (83%). Other associated costs included those for prescription medication and transportation; transportation being the largest.

Conclusion: The survey results demonstrated that patients diagnosed with TDβT and treated with RBCT incurred considerable treatment-related HCRU, and non-healthcare-related costs such as transportation, in the 6-month period prior to assessment. Further investigation is needed to determine the actual cost of this utilization to the patients’ insurer and payer. Medical treatments that would decrease the dependency on RBCT in TDβT might decrease this HCRU and cost burden for both the patient and payer.