OBJECTIVES: Patients with transfusion-dependent β-thalassemia (TDT) have reduced or absent β-globin production and require regular red blood cell transfusions (RBCTs) for survival. This study aimed to describe the healthcare resource utilization (HCRU) associated with TDT in the Netherlands.

METHODS: This longitudinal, retrospective cohort study utilized the PHARMO Data Network in the Netherlands to identify patients with an ambulatory visit or hospital admission for β-thalassemia between 01/01/2014 and 12/31/2020 (study selection period). Eligible patients with TDT were required to have ≥8 RBCTs in any consecutive 12-month period. The index date was the date of the 8th transfusion in a 12-month period during the study selection timeframe. Patients were required to have data for 12 consecutive months before and after their index date. Patients with hereditary persistence of fetal hemoglobin, sickle cell disease, α-thalassemia, or hematopoietic stem cell transplant were excluded. Patients were followed from the index date until the earliest occurrence of death, loss to follow-up, or the end of the study period (12/31/2021). Demographics were assessed at the index date, and RBCTs and HCRU were summarized during the follow-up period.

RESULTS: A total of 54 patients met the inclusion criteria for TDT. The mean age at index was 17.7 years and 42.6% were female. The mean duration of follow-up was 3.3 years. Patients with TDT received 13.0 RBCTs per-patient-per-year (PPPY) on average in the follow-up period. Patients had a mean of 11.3 inpatient hospitalizations and 8.4 outpatient specialist visits (both PPPY). Most patients had an RBCT-related inpatient hospitalization with duration of <1 day (47/54 [87.0%], 7.4 hospitalizations PPPY). Patients also experienced inpatient hospitalizations with duration of ≥1 day (34/54 [63.0%], 0.5 hospitalizations PPPY).

CONCLUSIONS: There is substantial HCRU associated with the care of patients with TDT in the Netherlands, driven by frequent RBCTs and hospitalizations.