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Health-related quality of life and productivity/activity impairment in patients with transfusion-dependent B-thalassaemia in the UK NHS: Data from a UK Multicentre observational study
Objectives
Patients with the most severe form of β-thalassaemia require chronic red blood cell (RBC) transfusions to sustain life (transfusion-dependent β-thalassaemia [TDT]). Treatment with chronic RBC transfusions requires iron chelation therapy and monitoring to prevent complications. Our study aimed to describe the real-world management and patient-reported outcomes (PROs) of patients with TDT in the United Kingdom.
Methods
An observational study was conducted in 9 centres, comprising a retrospective chart-review and cross-sectional PRO questionnaires (completed at enrolment). Eligible patients had a documented diagnosis of TDT (≥8 transfusions during first year of chronic transfusion therapy or ≥100 mL/kg/year of packed RBCs) ≥2 years pre-data collection. Herein we report results from patients’ EuroQOL 5 dimension-3 level (EQ-5D-3L), child-friendly EQ-5D (EQ-5D-Y), Work Productivity and Activity Impairment (WPAI), and TranQoL questionnaires.
Results
Of 165 study patients, 120 completed ≥1 PRO(s) (median age 28.5 [range=8.4–67.4] years; 48% female). Mean EQ-5D-3L utility score (ages ≥16) was 0.69 (standard deviation [SD]=0.33; n=94); 57% (n=56/98) reported ‘moderate’/‘extreme’ pain, 52% (n=49/95) reported at least ‘some problems’ performing usual activities, and 42% (n=41/97) reported ‘some problems’ with mobility. Twenty patients completed the EQ-5D-Y (8–15 years), 50% reported ‘some’/’a lot’ of pain. Median WPAI domain scores were: absenteeism (3%, interquartile range [IQR]=0–17%, n=44), presenteeism (30%, IQR=10–60%, n=49), total work productivity loss (38%, IQR=23–60%, n=44) and activity impairment (50%, IQR=20–80%, n=88). Mean TranQoL total scores were 58.6 (SD=18.4, patients aged >18, n=94), and 74.8 (SD=15.0, patients aged 7–18 years, n=27); the most affected domain was ‘school and career health’ (mean 51.3 [SD=30.0], n=92).
Conclusions
Our results indicate that TDT has a substantial impact on patients’ health-related quality of life (affecting several domains including pain, usual activities, and mobility) and work productivity, with productivity/activity impairment appearing comparable to or higher than reported in other chronic conditions (e.g. COPD, rheumatoid arthritis; Miller, 2016).
Authors
K Ryan, S Pancham, P Telfer, F Shah, J Kell, S Pollard, R Wynn, M Velangi, E Chalmers, J Hickey, C Paramore, M Jobanputra
Journal
Value in Health
Therapeutic Area
Hematology
Center of Excellence
Real-world Evidence & Data Analytics
Year
2019
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