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

• Published in: EJHaem Vol. 2; no. 4; pp. 738 - 749
• Main 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
• Format: Journal Article
• Language: English
• Published: United States John Wiley & Sons, Inc 01.11.2021; John Wiley and Sons Inc; Wiley
• Subjects: Blood diseases; blood transfusion; Blood transfusions; Caregivers; Data collection; DNA nucleotidylexotransferase; Ferritin; Health care; Health services; healthcare resource use; Iron; iron chelation therapy; Mixed methods research; Mortality; Observational studies; Patients; Pediatrics; Productivity; Quality of life; Questionnaires; Sickle Cell, Thrombosis, and Benign Haematology; Stem cell transplantation; Thalassemia; transfusion‐dependent β‐thalassaemia; United Kingdom > UK; England; healthcare resource use; blood transfusion; iron chelation therapy; quality of life; transfusion‐dependent β‐thalassaemia
• ISSN: 2688-6146; 2688-6146
• DOI: 10.1002/jha2.282

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.