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. Issue 4 (8th September 2021)

• Record Type: Journal Article
• Authors: 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
• Is Part Of: EJHaem. Volume 2:Issue 4(2021)
• Page Start: 738
• Page End: 749
• Publication Date: 2021-09-08
• Subjects: blood transfusion -- healthcare resource use -- iron chelation therapy -- quality of life -- transfusion‐dependent β‐thalassaemia; Hematology -- Periodicals; 616.15
• DOI: 10.1002/jha2.282
• Languages: English
• ISSNs: 2688-6146

Abstract

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.