Health-related quality of life and economic impacts in adults with transfusion-dependent β-Thalassemia: findings from a prospective longitudinal real-world study

Purpose
To characterize the health-related quality of life (HRQoL) and economic impacts of transfusion-dependent β-thalassemia (TDT).

Methods
A prospective longitudinal real-world study was conducted in adults with TDT in the US and Europe (France, Germany, Italy, UK) over 6 months. Eligibility criteria included receiving ≥ 8 blood transfusions/year in each of the 2 years before enrollment. Study outcomes assessed at months 0, 3, and 6, included demographics, clinical characteristics, patient-reported outcome measures (EQ-VAS, FACT-G, FACIT-F, 11-point Numerical Rating Scale (NRS) of pain, TranQoL, and WPAI), and responses to bespoke questions on financial burden/distress.

Results
Overall, 155 adults with TDT participated (mean age [standard deviation (SD)]: 38.5 [10.8] years; 65.8% female). Most participants resided in the US (53.5%) or UK (22.6%). EQ-VAS, FACT-G, and FACIT-F mean (SD) scores at month 0 were lower among participants than the US general population (67.1 [20.4] versus 80.4 [15.6], 70.1 [19.0] versus 77.0 [16.1], and 27.9 [13.7] versus 43.6 [9.4], respectively; all p < 0.001). Mean (SD) TranQoL total score was 53.9 (18.5) and overall level of pain was “moderate” (NRS mean [SD]: 3.5 [2.8]). Part- or full-time employed participants missed a mean of 5.9 h of work in the preceding 7 days with out-of-pocket expenses being a moderate-to-major burden for 55% of participants. The findings were consistent over time.

Conclusions
Participants reported substantial humanistic and economic burdens associated with living with TDT. Our findings highlight the need for alternative treatment options that contribute to improvements in HRQoL and reduce economic burden in this patient population.