Open AccessRoutine 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
Author(s) -
Shah Farrukh,
Telfer Paul,
Velangi Mark,
Pancham Shivan,
Wynn Robert,
Pollard Sally,
Chalmers Elizabeth,
Kell Jonathan,
Carter Angela M.,
Hickey Joe,
Paramore Clark,
Jobanputra Minesh,
Ryan Kate
Publication year - 2021
Publication title -
ejhaem
Language(s) - English
Resource type - Journals
ISSN - 2688-6146
DOI - 10.1002/jha2.282
Subject(s) - medicine , interquartile range , observational study , blood transfusion , pediatrics , retrospective cohort study , emergency medicine
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.