英国输血依赖型β地中海贫血患者的常规管理、医疗资源使用以及患者和护理人员报告的结果：一项混合方法观察性研究。

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.

作者信息

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

机构信息

Whittington Hospital London UK.

Centre for Genomics and Child Health Blizard Institute Queen Mary University of London London UK.

出版信息

EJHaem. 2021 Sep 8;2(4):738-749. doi: 10.1002/jha2.282. eCollection 2021 Nov.

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 ( = 165) 1961.0 (1090.0-3003.0) μg/L (38% > 2500 μg/L); R2 liver iron ( = 119) 5.4 (2.9-11.6) mg/g (16% ≥15 mg/g); T2* cardiac iron ( = 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; = 94 [≥16 years]) and mean Transfusion-dependent quality of life score was 58.6 (±18.4; = 94 [≥18 years]). For Work Productivity and Activity impairment, mean activity impairment for patients ≥18 years ( = 88) was 48% (±32%) and for carers ( = 29) was 28% (±23%).

CONCLUSIONS

TDT presents significant burden on patients, carers and healthcare resources.

目的

我们评估了英国输血依赖型β地中海贫血（TDT）儿科和成年患者的常规医疗管理、临床状况以及患者和护理人员报告的结局。

方法

一项多中心、观察性混合方法研究评估了来自英国9个中心的165例患者（50%为男性；中位年龄24.1岁[四分位间距（IQR）11.8 - 37.2岁]）。

结果

患者每年平均有13.7次（标准差[SD]±3.2）输血事件（平均回顾观察期4.7年[±0.7年]）。观察期内最后一次评估时铁过载参数的中位数（IQR）为：血清铁蛋白（n = 165）1961.0（1090.0 - 3003.0）μg/L（38%＞2500μg/L）；肝脏R2铁（n = 119）5.4（2.9 - 11.6）mg/g（16%≥15mg/g）；T2*心脏铁（n = 132）30.3（22.0 - 37.1）ms（10%＜10ms）。观察期内所有患者均接受了≥1种铁螯合剂治疗；21%接受了联合治疗。患者每年平均有7.8次（±8.1）与输血无关的住院就诊或入院。成年患者的平均EQ - 5D效用评分是0.69（±0.33；n = 94[≥16岁]），平均输血依赖生活质量评分是58.6（±18.4；n = 94[≥18岁]）。对于工作生产力和活动受损情况，≥18岁患者（n = 88）的平均活动受损率为48%（±32%），护理人员（n = 29）的平均活动受损率为28%（±23%）。