Presentation (CTI)

OBJECTIVES: Transfusion-dependent β-thalassemia (TDT) is a severe inherited blood disorder requiring frequent, lifelong red blood cell transfusions and iron chelation therapy. Despite available treatments, adults with TDT experience considerable clinical and humanistic burden. This analysis characterized UK and French health utilities among adults with TDT using EQ-5D-5L data generated from two large real-world studies.
METHODS: Re-analysis of existing data from two real-world patient survey studies that included the EQ-5D-5L was conducted. Study 1 was a longitudinal survey study conducted in the United States (US), the United Kingdom (UK), France, Germany, and Italy. Study 2 was a cross-sectional survey study conducted in adults with TDT in the US and UK. In both studies, eligible participants received ≥8 red blood cell transfusions in each of the previous 2 years. Descriptive analyses were conducted to summarize EQ-5D utility values using UK and French value sets.
RESULTS: Study 1 included 155 adults with TDT (mean age: 38.5 years; 65.8% female) and Study 2 included 221 adults with TDT (mean age: 40.6 years; 59.1% female). Across both studies, transfusions were most commonly administered every 3 weeks, with participants receiving a mean of 18.7 transfusions per-patient-per-year in Study 1 and 18.1 in Study 2. The mean EQ-5D utility values based on the UK value set were 0.67 (SD: 0.28) at baseline in Study 1 and 0.64 (0.33) in Study 2. The mean utility values based on the French value set were 0.63 (0.32) and 0.60 (0.36) in Studies 1 and 2, respectively.
CONCLUSIONS: Across two large real-world studies, adults with TDT reported low EQ-5D utility values despite ongoing transfusion and supportive care. These findings demonstrate a persistent humanistic burden in TDT and provide real-world EQ-5D utility evidence to inform health economic evaluations.