Author(s)
Ryan K1, Pancham S2, Telfer P3, Shah F4, Kell J5, Pollard S6, Wynn R7, Velangi M8, Chalmers E9, Hickey J10, Paramore C11, Jobanputra M12
1Manchester Royal Infirmary, Manchester, UK, 2Birmingham City Hospital, Birmingham, UK, 3The Royal London Hospital, London, UK, 4Whittington Hospital, London, UK, 5University Hospital of Wales, Cardiff, UK, 6Bradford Royal Infirmary, Bradford, UK, 7Royal Manchester Children's Hospital, Manchester, UK, 8Birmingham Children's Hospital, Birmingham, UK, 9Glasgow Children's Hospital, Glasgow, UK, 10OPEN VIE, Marlow, UK, 11bluebird bio Inc, Cambridge, MA, USA, 12bluebird bio UK, Basingstoke, MA, UK
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).
Conference/Value in Health Info
2019-11, ISPOR Europe 2019, Copenhagen, Denmark
Code
PRO146
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare and Orphan Diseases
