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镰状细胞病(SCD)治疗进展:新型药物疗法及其对患者预后影响的综述

Advancements in Sickle Cell Disease (SCD) Treatment: A Review of Novel Pharmacotherapies and Their Impact on Patient Outcomes.

作者信息

Khan Salman J, Zaidi Syed Asjad Tauheed, Murtaza Syeda Fatima, Asif Muhammad, Kumar Vinod

机构信息

Medicine, Mayo Clinic, Jacksonville, USA.

Hematology and Oncology, Mayo Clinic, Jacksonville, USA.

出版信息

Cureus. 2023 Aug 2;15(8):e42847. doi: 10.7759/cureus.42847. eCollection 2023 Aug.

Abstract

Sickle cell disease (SCD) is a genetic hereditary blood disease that disrupts normal beta-globin production. Patients with SCD experience a broad range of symptoms ranging from anemia, pain crises, and jaundice to acute coronary syndrome and stroke. SCD has been treated with hydroxyurea since 1998. Three important pharmacotherapies have been approved by the Food and Drug Administration (FDA) in the past few years. L-Glutamine has shown efficacy in reducing vaso-occlusive pain crises and hospitalization. Crizanlizumab has also shown positive outcomes in patients with SCD. Voxelotor has been studied to be effective in improving hemolytic anemia and the quality of life in SCD patients. These drugs can be used alone or in conjunction with hydroxyurea. Trials have shown that these therapies have significant efficacy. The events of pain, hemolytic anemia, vaso-occlusive crises, and hospitalizations have been reduced by using these agents. In this editorial, we will discuss these advanced treatment options for patients with SCD.

摘要

镰状细胞病(SCD)是一种遗传性血液疾病,会干扰正常的β-珠蛋白生成。SCD患者会出现广泛的症状,从贫血、疼痛危象、黄疸到急性冠状动脉综合征和中风。自1998年以来,SCD一直用羟基脲治疗。在过去几年中,美国食品药品监督管理局(FDA)批准了三种重要的药物疗法。L-谷氨酰胺已显示出在减少血管阻塞性疼痛危象和住院方面的疗效。crizanlizumab在SCD患者中也显示出积极的结果。voxelotor已被研究证明对改善SCD患者的溶血性贫血和生活质量有效。这些药物可以单独使用或与羟基脲联合使用。试验表明,这些疗法具有显著疗效。使用这些药物可减少疼痛、溶血性贫血、血管阻塞性危象和住院等情况。在这篇社论中,我们将讨论SCD患者的这些先进治疗选择。

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