Health-related quality of life (HRQOL) is a patient-reported outcome that assesses the impact of a disease or illness on different domains of a patient's life. Different general and disease-specific measures can be used to evaluate HRQOL. This article aimed to summarize the evidence for HRQOL among patients with transfusion-dependent (TDT) and non-transfusion-dependent thalassemia (NTDT). We included HRQOL data related to standard therapy with blood transfusions, iron chelation, and/or luspatercept in TDT and NTDT, as well as curative therapies for TDT, including hematopoietic stem cell transplant (HSCT) and gene therapy. Patients with thalassemia had worse HRQOL scores compared to the general population, and chronic pain was seen to increase in frequency and severity over time with age. NTDT patients reported worse physical health and functioning, mental health, general health, and vitality than TDT patients. However, TDT patients reported worse pain, change in health, and social support than NTDT. Most therapies improved overall HRQOL among thalassemia patients. Deferasirox, an oral iron chelator, was associated with more HRQOL benefits compared to deferoxamine, an intravenous iron chelator. Luspatercept showed clinically meaningful improvement in physical functioning among TDT and NTDT. Furthermore, HSCT and gene therapy were associated with better physical, emotional, and mental domains scores.

中文翻译：

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地中海贫血标准疗法和治疗疗法的健康相关生活质量：叙述性文献综述

健康相关生活质量 (HRQOL) 是患者报告的结果，用于评估某种疾病或疾病对患者生活不同领域的影响。不同的一般和特定疾病的测量可用于评估 HRQOL。本文旨在总结输血依赖性（TDT）和非输血依赖性地中海贫血（NTDT）患者的 HRQOL 证据。我们纳入了与 TDT 和 NTDT 中输血、铁螯合和/或 luspatercept 标准治疗相关的 HRQOL 数据，以及 TDT 的治疗方法，包括造血干细胞移植 (HSCT) 和基因治疗。与一般人群相比，地中海贫血患者的 HRQOL 评分更差，而且慢性疼痛的频率和严重程度随着年龄的增长而增加。 NTDT 患者的身体健康和功能、心理健康、总体健康和活力比 TDT 患者更差。然而，与 NTDT 相比，TDT 患者的疼痛、健康状况变化和社会支持更严重。大多数疗法改善了地中海贫血患者的整体 HRQOL。与静脉注射铁螯合剂去铁胺相比，口服铁螯合剂地拉罗司与更多的 HRQOL 益处相关。 Luspatercept 在 TDT 和 NTDT 中显示出具有临床意义的身体功能改善。此外，造血干细胞移植和基因治疗与更好的身体、情感和精神领域得分相关。