Routine management, healthcare resource use and patient and carer-reported outcomes of patients with transfusion-dependent β-thalassaemia in the United Kingdom: A mixed methods observational study.
Shah, Farrukh Telfer, Paul Velangi, Mark Pancham, Shivan Wynn, Robert Pollard, Sally Chalmers, Elizabeth Kell, Jonathan Carter, Angela M Hickey, Joe
Shah, Farrukh
Telfer, Paul
Velangi, Mark
Pancham, Shivan
Wynn, Robert
Pollard, Sally
Chalmers, Elizabeth
Kell, Jonathan
Carter, Angela M
Hickey, Joe
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Affiliation
Sandwell and West Birmingham NHS Trust; Centre for Genomics and Child Health Blizard Institute Queen Mary University of London; Royal Manchester Children's Hospital
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Publication date
2021-09-08
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Abstract
Objectives: We evaluated routine healthcare management, clinical status and patient- and carer-reported outcomes in UK paediatric and adult patients with transfusion-dependent β-thalassaemia (TDT).
Methods: A multi-centre, observational mixed-methodology study evaluated 165 patients (50% male; median age 24.1 [interquartile range (IQR)] 11.8-37.2] years) from nine UK centres.
Results: Patients had a mean of 13.7 (standard deviation [SD] ±3.2) transfusion episodes/year (mean retrospective observation period 4.7 [±0.7] years). The median (IQR) for iron overload parameters at the last assessment during the observation period were: serum ferritin (n = 165) 1961.0 (1090.0-3003.0) μg/L (38% > 2500 μg/L); R2 liver iron (n = 119) 5.4 (2.9-11.6) mg/g (16% ≥15 mg/g); T2* cardiac iron (n = 132) 30.3 (22.0-37.1) ms (10% < 10 ms). All patients received ≥1 iron chelator during the observation period; 21% received combination therapy. Patients had a mean of 7.8 (±8.1) non-transfusion-related hospital attendances or admissions/year. Adult patients' mean EQ-5D utility score was 0.69 (±0.33; n = 94 [≥16 years]) and mean Transfusion-dependent quality of life score was 58.6 (±18.4; n = 94 [≥18 years]). For Work Productivity and Activity impairment, mean activity impairment for patients ≥18 years (n = 88) was 48% (±32%) and for carers (n = 29) was 28% (±23%).
Conclusions: TDT presents significant burden on patients, carers and healthcare resources.
Citation
Shah F, Telfer P, Velangi M, Pancham S, Wynn R, Pollard S, Chalmers E, Kell J, Carter AM, Hickey J, Paramore C, Jobanputra M, Ryan K. Routine management, healthcare resource use and patient and carer-reported outcomes of patients with transfusion-dependent β-thalassaemia in the United Kingdom: A mixed methods observational study. EJHaem. 2021 Sep 8;2(4):738-749. doi: 10.1002/jha2.282.
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Article