Newer Modalities and Updates in the Management of Sickle Cell Disease: A Systematic Review.

IF 2.1 Q3 HEMATOLOGY Journal of Blood Medicine Pub Date : 2024-09-12 eCollection Date: 2024-01-01 DOI:10.2147/JBM.S477507
Zeel Vishnubhai Patel, Priyadarshi Prajjwal, Lakshmi Deepak Bethineedi, Divyakshi J Patel, Kaarvi Khullar, Hinal Patel, Kanishka Khatri, Mohammed Dheyaa Marsool Marsool, Srikanth Gadam, Soumya Aleti, Omniat Amir
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Abstract

Sickle cell disease (SCD), the most common autosomal recessive genetic disorder, affects the hemoglobin (Hb) chains in human red blood cells. It is caused by mutations in the β-globin genes, leading to the production of hemoglobin S, which results in the formation of sickle-shaped red blood cells (RBCs). These abnormal cells cause hemolysis, endothelial damage, and small vessel occlusion, leading to both acute and long-term complications. According to the World Health Organization's 2008 estimates, SCD affects approximately 2.28 per 1000 individuals globally. Despite this high prevalence, therapeutic advancements have been slow. For many years, the only FDA-approved medications for managing SCD complications were hydroxyurea and deferiprone. However, recent years have seen the approval of several new therapies, including L-glutamine (2017), voxelotor and crizanlizumab (2019), as well as exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) (2023). These treatments have proven effective in managing both the acute and chronic effects of SCD, including hemolytic anemia, chronic pain, stroke, vaso-occlusive crises, and multiple organ damage syndromes. This review explores the mechanisms of action, practical considerations, and side effects of these emerging therapies, drawing from a comprehensive search of databases such as PubMed, Medline, and Cochrane.

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镰状细胞病治疗的新模式和最新进展:系统回顾。
镰状细胞病(SCD)是最常见的常染色体隐性遗传疾病,影响人类红细胞中的血红蛋白(Hb)链。它是由β-球蛋白基因突变引起的,导致产生血红蛋白S,从而形成镰刀形红细胞(RBC)。这些异常细胞会引起溶血、内皮损伤和小血管闭塞,导致急性和长期并发症。根据世界卫生组织 2008 年的估计,全球每 1000 人中约有 2.28 人患有 SCD。尽管发病率如此之高,但治疗进展却十分缓慢。多年来,FDA 批准用于控制 SCD 并发症的药物只有羟基脲和去铁酮。然而,近年来又有几种新疗法获得批准,包括 L-谷氨酰胺(2017 年)、voxelotor 和 crizanlizumab(2019 年),以及 exagamglogene autotemcel(Casgevy)和 lovotibeglogene autotemcel(Lyfgenia)(2023 年)。事实证明,这些疗法可有效控制 SCD 的急性和慢性影响,包括溶血性贫血、慢性疼痛、中风、血管闭塞性危象和多器官损伤综合征。本综述通过对 PubMed、Medline 和 Cochrane 等数据库的全面检索,探讨了这些新兴疗法的作用机制、实际注意事项和副作用。
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来源期刊
CiteScore
3.50
自引率
0.00%
发文量
94
审稿时长
16 weeks
期刊介绍: The Journal of Blood Medicine is an international, peer-reviewed, open access, online journal publishing laboratory, experimental and clinical aspects of all topics pertaining to blood based medicine including but not limited to: Transfusion Medicine (blood components, stem cell transplantation, apheresis, gene based therapeutics), Blood collection, Donor issues, Transmittable diseases, and Blood banking logistics, Immunohematology, Artificial and alternative blood based therapeutics, Hematology including disorders/pathology related to leukocytes/immunology, red cells, platelets and hemostasis, Biotechnology/nanotechnology of blood related medicine, Legal aspects of blood medicine, Historical perspectives. Original research, short reports, reviews, case reports and commentaries are invited.
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