Udeze Chuka, Ly Nelly F, Ingleby Fiona C, Fleming Sophia D, Conner Sarah C, Howard Jo, Li Nanxin, Shah Farrukh
Vertex Pharmaceuticals Incorporated, Boston, Massachusetts.
IQVIA, London, UK.
Clin Ther. 2025 Jan;47(1):37-43. doi: 10.1016/j.clinthera.2024.09.024. Epub 2024 Nov 1.
Patients with transfusion-dependent β-thalassemia (TDT) have reduced levels of β-globin, leading to ineffective erythropoiesis and iron overload. Patients with TDT depend on regular red blood cell transfusions (RBCTs) and iron chelation therapy for survival and management of disease- and treatment-related clinical complications. This study describes the clinical and economic burden in patients with TDT in England.
This longitudinal, retrospective study linked the Clinical Practice Research Datalink (CPRD) database with secondary care data from the Hospital Episode Statistics database to identify patients with a diagnosis of β-thalassemia between July 1, 2008, and June 30, 2018. Included patients had a diagnosis of β-thalassemia prior to the index date, ≥8 RBCTs per year for ≥2 consecutive years, and ≥1 year of follow-up data available from the index date. Each eligible patient was exact matched with up to 5 controls in the CPRD. Proportions of deaths and rates of mortality, acute and chronic complications, and healthcare resource utilization (HCRU) were calculated during the follow-up period.
Of 11,359 identified patients with β-thalassemia, 237 patients with TDT met the eligibility criteria and were matched with 1184 controls. The mean age at the index date was approximately 25 years in the patient and control groups. The proportion of deaths (7.17% vs 1.18%; P < 0.05) and mortality rate (1.19 deaths per 100 person-years vs 0.20 deaths per 100 person-years) were higher among patients with TDT compared to controls. Endocrine complications and bone disorders were the most prevalent complications among patients with TDT (58.23%) and included osteoporosis (29.11%), diabetes mellitus (28.27%), and hypopituitarism (28.27%). Patients with TDT had a mean of 13.62 RBCTs per patient per year (PPPY). HCRU was substantially higher among patients with TDT, wherein patients with TDT had higher rates of prescriptions recorded in primary care (24.09 vs 8.61 PPPY), outpatient visits (16.69 vs 1.31 PPPY), and inpatient hospitalizations (17.41 vs 0.24 PPPY) than controls. Inpatient hospitalizations were primarily <1 day, with 16.62 events PPPY lasting <1 day and 0.79 events PPPY lasting ≥1 day. Patients with TDT aged ≥18 years had increased rates of mortality, clinical complications, and HCRU than those aged <18 years.
Patients with TDT in England have higher mortality than matched controls, substantial disease-related clinical complications, and substantial HCRU. High mortality and clinical complications highlight the need for additional innovative therapies for TDT.
依赖输血的β地中海贫血（TDT）患者的β珠蛋白水平降低，导致红细胞生成无效和铁过载。TDT患者依靠定期红细胞输血（RBCT）和铁螯合疗法来维持生存并管理与疾病及治疗相关的临床并发症。本研究描述了英国TDT患者的临床和经济负担。
这项纵向回顾性研究将临床实践研究数据链（CPRD）数据库与医院事件统计数据库中的二级护理数据相链接，以识别2008年7月1日至2018年6月30日期间诊断为β地中海贫血的患者。纳入患者在索引日期之前已诊断为β地中海贫血，连续≥2年每年接受≥8次RBCT，且从索引日期起有≥1年的随访数据。每个符合条件的患者在CPRD中与多达5名对照进行精确匹配。在随访期间计算死亡率、急性和慢性并发症发生率以及医疗资源利用（HCRU）情况。
在11359名已识别的β地中海贫血患者中，237名TDT患者符合纳入标准，并与1184名对照进行匹配。患者组和对照组在索引日期的平均年龄约为25岁。与对照组相比，TDT患者的死亡率（7.17%对1.18%；P<0.05）和死亡率（每100人年1.19例死亡对每100人年0.20例死亡）更高。内分泌并发症和骨骼疾病是TDT患者中最常见的并发症（58.23%），包括骨质疏松症（29.11%）、糖尿病（28.27%）和垂体功能减退（28.27%）。TDT患者每人每年平均接受13.62次RBCT。TDT患者的HCRU显著更高，其中TDT患者在初级保健中的处方记录率（每人每年24.09次对8.61次）、门诊就诊率（每人每年16.69次对1.31次）和住院率（每人每年17.41次对0.24次）均高于对照组。住院主要为<1天，每人每年有16.62次住院持续<1天，0.79次住院持续≥1天。年龄≥18岁的TDT患者比<18岁的患者死亡率、临床并发症和HCRU更高。
英国的TDT患者比匹配的对照组死亡率更高，有大量与疾病相关的临床并发症和大量的HCRU。高死亡率和临床并发症凸显了对TDT额外创新疗法的需求。
