OBJECTIVES: Transfusion-dependent β-thalassemia (TDT) is a severe form of β-thalassemia requiring frequent, lifelong red blood cell transfusions and iron chelation therapy. This longitudinal study examined the impact of TDT on participants’ health-related quality of life and work productivity.

METHODS: A longitudinal survey was administered at three timepoints (month 0 [M0], month 3 [M3], month 6 [M6]) in the US, the UK, France, Germany, and Italy and included multiple patient-reported outcome (PRO) measures: FACT-G, EQ-5D-5L, FACIT-Fatigue (only M1), and WPAI. Key eligibility criteria included receiving ≥8 blood transfusions in each of the last 2 years. Descriptive analyses were conducted at M3. These data were compared with M0 data (previously reported), where available.

RESULTS: The survey was completed by 121 adult participants with TDT living in the US (n=60) and the UK, Germany, Italy, and France (n=61) at M3. Overall, 70.0% were female, their mean age was 38.5 years (standard deviation [SD]=10.5), and 33.9% had full-time employment (≥32 hours/week). Participants received a mean of 18.2 (SD=7.9) transfusions annually, equivalent to a mean of 1 transfusion every 2.9 weeks. Participants spent a median of 19.0 hours (interquartile range=13.5–26.0) managing their condition in the past month. At M3, the mean FACT-G score was 69.3 (SD=19.8). The mean EQ-5D VAS score was 65.4 (SD=19.2). The mean FACIT-Fatigue score was 27.9 (SD=13.7), which is lower than that of the US general population (mean=43.6 [SD=12.6]) and comparable to that of patients with cancer and anemia (23.9 [SD=12.6]). According to the WPAI, employed participants had an average of 17.8% (SD=28.3%) absenteeism, 34.2% (SD=28.7%) presenteeism, and 43.5% (SD=32.4%) reduction in work productivity in the past week. PRO findings were comparable between M0 and M3.

CONCLUSIONS: Despite receiving currently available treatments, adults with TDT continue to experience substantial and sustained humanistic burden and indirect impacts.