Aydinok Yesim, Purushotham Sneha, Yucel Aylin, Glassberg Mrudula, Deshpande Sohan, Potrata Barbara, Trapali Myrto, Shah Farrukh
Department of Pediatric Hematology, Faculty of Medicine, Ege University Hospital, Kazımdirik, Ankara Asfaltı, 35100 Bornova, ĺzmir, Turkey.
Evidera, London, UK.
Ther Adv Hematol. 2024 Sep 18;15:20406207241270872. doi: 10.1177/20406207241270872. eCollection 2024.
β-Thalassemia is an inherited blood disorder requiring lifetime management of anemia and its complications.
This study aimed to determine the indirect costs and humanistic burden of β-thalassemia.
A systematic literature review was conducted.
Searches were conducted in Embase, MEDLINE, MEDLINE In-Process, and EconLit (November 1, 2010, to November 25, 2020). Studies reporting indirect costs and health-related quality of life (HRQoL) for patients with β-thalassemia were eligible.
Seventy-five publications were included. Mean annual days lost due to transfusion-related absenteeism ranged from 15.6 to 35 days. Patients spent a mean of 592 min (standard deviation (SD): 349) daily on disease management on transfusion days and 91 min (SD: 221) daily on non-transfusion days. Patients with non-transfusion-dependent β-thalassemia (NTDT) showed worse HRQoL versus those with transfusion-dependent β-thalassemia (TDT) on the 36-item Short Form Health Survey (75.8 vs 66.5; = 0.021). Caregivers of patients with TDT had more severe stress compared with patients (20.17 vs 18.95; = 0.006), as measured by the standardized Cohen Perceived Stress Questionnaire.
TDT is associated with substantial indirect costs and caregiver burden, and NTDT is associated with worse HRQoL. There is an unmet need for novel treatments in both TDT and NTDT that minimize patient and caregiver burden.
β地中海贫血是一种遗传性血液疾病，需要对贫血及其并发症进行终身管理。
本研究旨在确定β地中海贫血的间接成本和人文负担。
进行了一项系统的文献综述。
在Embase、MEDLINE、MEDLINE在研数据库和EconLit（2010年11月1日至2020年11月25日）中进行检索。报告β地中海贫血患者间接成本和健康相关生活质量（HRQoL）的研究符合要求。
纳入了75篇出版物。因输血相关缺勤导致的平均每年损失天数为15.6至35天。患者在输血日每天平均花费592分钟（标准差（SD）：349）进行疾病管理，在非输血日每天平均花费91分钟（SD：221）。在36项简短健康调查问卷中，非输血依赖型β地中海贫血（NTDT）患者的HRQoL比输血依赖型β地中海贫血（TDT）患者更差（75.8对66.5；P = 0.021）。根据标准化的科恩感知压力问卷测量，TDT患者的照顾者比患者有更严重的压力（20.17对18.95；P = 0.006）。
TDT与大量间接成本和照顾者负担相关，NTDT与较差的HRQoL相关。对于TDT和NTDT，都迫切需要新的治疗方法，以尽量减少患者和照顾者的负担。
