Department of Pediatric Hematology, Faculty of Medicine, Cairo University, Cairo, Egypt.
Thalassemia Center, Latifa Hospital, Dubai, United Arab Emirates.
Blood Rev. 2024 Jan;63:101138. doi: 10.1016/j.blre.2023.101138. Epub 2023 Oct 13.
β-Thalassemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management and devising solutions to address challenges attributed to resource limitations. The region has also participated in the majority of clinical trials and development programs of iron chelators and more novel ineffective erythropoiesis-targeted therapy. In this review, we provide a practical overview of management for patients with transfusion-dependent β-thalassemia, primarily driven by such experiences, with the aim of transferring knowledge to colleagues in other regions facing similar challenges.
β-地中海贫血是全球最常见的单基因疾病之一，在中东地区尤其高发。因此，我们在疾病管理方面拥有长期经验，并针对资源有限带来的挑战制定解决方案。该地区还参与了大多数铁螯合剂和新型无效红细胞生成靶向治疗的临床试验和开发项目。在这篇综述中，我们主要基于这些经验，为依赖输血的β-地中海贫血患者的管理提供实用概述，旨在将知识传授给面临类似挑战的其他地区的同事。
