关键词: Hemoglobinopathy hydroxycarbamide hydroxyurea sickle cell anemia sickle cell disease

来  源:   DOI:10.1080/17512433.2024.2390915

Abstract:
UNASSIGNED: Sickle cell disease is an inherited disorder characterized by hemoglobin S polymerization leading to vaso-occlusion and hemolytic anemia. These result in a variety of pathological events, causing both acute and chronic complications. Millions around the world are affected by sickle cell disease with predominance in sub-Saharan Africa. Hydroxyurea was the first drug approved for use in sickle cell disease to reduce the occurrence of painful crises and blood transfusions in patients with frequent, moderate to severe painful crises.
UNASSIGNED: With the development of new therapeutics, the role of hydroxyurea is evolving. This narrative review aims to provide clinical data, safety information, and supplementary evidence for the role of hydroxyurea in the current era of sickle cell disease. A comprehensive literature search of databases, including PubMed and Cochrane Library, was conducted from 1963 to 2024.
UNASSIGNED: Even though new medications have been approved for sickle cell disease, hydroxyurea remains the gold standard. Hydroxyurea is not only a disease modifier but it has additional clinical benefits, it is affordable, and its longevity has prompted expanded research in areas such as underutilization and pharmacogenomics. As the treatment landscape evolves, hydroxyurea\'s long-standing record of efficacy and safety continues to support its role as a key agent in disease management.
摘要:
镰状细胞病是一种遗传性疾病,其特征是血红蛋白S聚合导致血管闭塞和溶血性贫血。这些导致各种病理事件,引起急性和慢性并发症。全世界有数百万人受到镰状细胞疾病的影响,在撒哈拉以南非洲占主导地位。羟基脲是第一个被批准用于镰状细胞疾病的药物,以减少频繁患者的痛苦危机和输血的发生,中度到重度痛苦的危机。
随着新疗法的发展,羟基脲的作用正在演变。这篇叙述性综述旨在提供临床数据,安全信息,以及羟基脲在当前镰状细胞疾病时代的作用的补充证据。数据库的全面文献检索,包括PubMed和Cochrane图书馆,于1963-2024年进行。
尽管新的药物已被批准用于治疗镰状细胞病,羟基脲仍然是金标准。羟基脲不仅是疾病调节剂,但它有额外的临床益处,它是负担得起的,和它的寿命促使扩大研究领域,如利用不足和药物基因组学。随着治疗景观的发展,羟基脲的疗效和安全性的长期记录继续支持其作为疾病管理的关键药物的作用。
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