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

作者：Jennifer Drahos, Adriana Boateng-Kuffour, Melanie Calvert, Laurice Levine, Neelam Dongha, Nanxin Li, Zahra Pakbaz, Farrukh Shah, Nick Ainsworth, Antony P. Martin · 发表于：Quality of Life Research · 年份：2025 · DOI：10.1007/s11136-025-03961-8 · 被引用次数：10 · 研究领域：Hemoglobinopathies and Related Disorders、Blood groups and transfusion、Erythropoietin and Anemia Treatment

To characterize the health-related quality of life (HRQoL) and economic impacts of transfusion-dependent β-thalassemia (TDT). 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. 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. Participants reported substantial humanistic and economic burdens associated w...