镰状细胞病的疾病调整疗法--概述。

IF 1.1 4区医学 Q3 PEDIATRICS

Pediatric Annals Pub Date : 2024-02-01 DOI:10.3928/19382359-20231205-05

Radhika Peddinti

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引用次数: 0

摘要

镰状细胞病是一组遗传性血液疾病，血红蛋白聚合会导致溶血和血管闭塞。镰状细胞病会在患者的一生中引起各种并发症，包括贫血、感染、急性和慢性疼痛、中风和多器官功能障碍。虽然由于青霉素预防、免疫接种和输血实践等支持性护理的改善，儿童患者的存活率有了显著提高，但改变病情的疗法却十分匮乏，羟基脲是 20 多年来唯一的药物。在此，我们将讨论近年来新出现的镰状细胞病疗法。[2024;53(2):e52-e55.].

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Disease-Modifying Therapies for Sickle Cell Disease-An Overview.

Sickle cell disease refers to a group of inherited blood disorders in which hemoglobin polymerization leads to hemolysis and vaso-occlusion. This causes a myriad of complications during a patient's life span, ranging from anemia, infections, and acute and chronic pain to stroke and multiorgan dysfunction. Although there have been dramatic improvements in childhood survival thanks to improved supportive care with penicillin prophylaxis, immunizations, and improved transfusion practices, there was a dearth of disease-modifying therapies, with hydroxyurea being the only medication for >20 years. Here, we discuss the newer therapies for sickle cell disease that have emerged in recent years. [Pediatr Ann. 2024;53(2):e52-e55.].

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来源期刊

Pediatric Annals 医学-小儿科

CiteScore

1.80

自引率

0.00%

发文量

104

审稿时长

2 months

期刊介绍： Published for more than 40 years, Pediatric Annals is an online-only, monthly medical review journal dedicated to providing pediatricians and other clinicians with the latest practical information on the diagnosis and treatment of pediatric diseases and disorders. Begin to explore the Journal and all of its great benefits such as: -Single-topic summary reviews of important trends in pediatric medicine -Access to current articles, as well as several years of archived content -Columns including Healthy Baby/Healthy Child and Case Challenges