OBJECTIVES: Transfusion-dependent beta-thalassemia (TDT) is a genetic blood disorder whose treatment is characterized by lifelong red blood cell transfusions (RBCTs) and iron chelation therapy (ICT). This study aims to describe the economic burden of patients with TDT in the United States (US).

METHODS: This retrospective cohort study used administrative claims from US MarketScan Commercial, Medicare, and Medicaid Multi-state databases to identify patients with >1 inpatient or >2 outpatient claims for beta-thalassemia or Hemoglobin E/beta-thalassemia between March 1, 2010 – March 1, 2019. Eligible patients with TDT were required to have ≥8 RBCTs in any 12-month period on or after the date of the earliest qualifying beta-thalassemia claim (first RBCT date = index date) and at least 12 months of post-index continuous enrollment with medical and pharmacy benefits. At least 3 days between service dates of RBCT claims were required to be considered discrete RBCTs. Patients were followed from index date to insurance disenrollment, death, or end of study period (March 1, 2020), whichever came first. Patients with hematopoietic stem cell transplant or sickle cell disease claims were excluded from this analysis. Baseline demographics, RBCT frequency, healthcare resource utilization, and costs were summarized per patient per year (PPPY).

RESULTS: A total of 4,504 patients with a diagnosis of beta-thalassemia were identified, of which 198 met criteria for TDT and other inclusion/exclusion criteria. Mean patient age at index date was 22.6 years and 56.6% were female; 81.8% of patients were in the Commercial database, 16.7% in Medicaid, and 1.5% in Medicare. Patients received a mean 12.52 RBCTs annually. Mean total annual healthcare costs were $137,987 PPPY. Mean annual costs of RBCTs were $27,887 and ICT were $70,402 PPPY.