OBJECTIVES: Patients with transfusion-dependent β-thalassemia (TDT) require regular red blood cell transfusions (RBCTs) for survival. This study sought to understand the healthcare resource utilization (HCRU) among patients with TDT in Italy.

METHODS: This retrospective cohort study utilized an administrative database of Italian regions and local health units to identify patients with ≥8 RBCTs over a 12-month period and ≥1 iron chelation therapy (ICT) prescription between January 1, 2011, and February 1, 2019. The index date was the first transfusion of the ≥8 RBCTs during a 12-month period. Eligible patients were required to be continuously enrolled for 1 year before and after their index date. Each patient was matched to 5 controls without disease by age, region, gender, and index year. Patients and controls were followed from index until death, exiting the database, or end of study period (February 1, 2020), whichever occurred first. Demographics and rate of HCRU (per-patient-per-year [PPPY]) were assessed at index and during follow-up, respectively. A t-test was used for significance testing for HCRU (P<0.05).

RESULTS: A total of 214 patients met the inclusion criteria for TDT and were matched to 1,070 controls. The mean age of patients with TDT at index was 46.7 years, and 45.8% were male. Matched controls had similar demographics. Patients averaged 18.2 RBCTs PPPY. Compared to matched controls, patients had significantly higher mean utilization of prescriptions (20.30 vs 4.91; P<0.001), outpatient services (21.92 vs 1.58; P<0.001), and all-cause hospitalization (1.35 vs 0.09; P<0.001) (all PPPY). In 1 year, patients averaged 16.1 days in the hospital compared to matched controls, who averaged 1.5 days.

CONCLUSIONS: This analysis shows the significant economic burden for patients with TDT in Italy, which is likely underestimated given the lack of data on emergency room admissions in the database.