伏氯托品：镰状细胞病的一线希望。
Voxelotor: A Ray of Hope for Sickle Disease.
作者信息
AlDallal Salma M
机构信息
Hematology Laboratory Specialist, Amiri Hospital, Kuwait City, KWT.
出版信息
Cureus. 2020 Feb 26;12(2):e7105. doi: 10.7759/cureus.7105.
Abstract
Sickle cell disease is one challenging blood disorder, affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Several clinical trials and case series have documented the benefits and safety of voxelotor therapy in sickle cell disease. Currently, the US FDA has approved the drug for treatment of sickle cell disease and also granted the status of orphan drug.
摘要
镰状细胞病是一种具有挑战性的血液疾病，仅在美国就影响着约10万人。目前获批的药物均无法改变该疾病的潜在病理状况。伏索利度（Voxelotor）是同类药物中的首款，是一种口服药物，可改变潜在的疾病病理状况（通过增加血红蛋白与氧气之间的亲和力）并抑制红细胞镰变。多项临床试验和病例系列记录了伏索利度治疗镰状细胞病的益处和安全性。目前，美国食品药品监督管理局已批准该药物用于治疗镰状细胞病，并授予其孤儿药地位。
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本文引用的文献
1
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.一项针对镰状细胞病患者的 voxotor 的 3 期随机试验。
N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.
2
Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease.在健康成年人和镰状细胞病患者中 voxotor（GBT440）的药代动力学和药效学。
Br J Clin Pharmacol. 2019 Jun;85(6):1290-1302. doi: 10.1111/bcp.13896. Epub 2019 Mar 31.
3
A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease.一项评估 voxeloTOR 在镰状细胞病患者中的 1/2 期递增剂量和开放标签扩展研究。
Blood. 2019 Apr 25;133(17):1865-1875. doi: 10.1182/blood-2018-08-868893. Epub 2019 Jan 17.
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Emerging pharmacotherapeutic approaches for the management of sickle cell disease.新兴的镰状细胞病治疗策略。
Expert Opin Pharmacother. 2019 Feb;20(2):173-186. doi: 10.1080/14656566.2018.1548610. Epub 2018 Nov 30.
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Impact of voxelotor (GBT440) on unconjugated bilirubin and jaundice in sickle cell disease.伐昔洛韦（GBT440）对镰状细胞病中非结合胆红素和黄疸的影响。
Hematol Rep. 2018 May 22;10(2):7643. doi: 10.4081/hr.2018.7643. eCollection 2018 May 14.
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A Phase 3 Trial of l-Glutamine in Sickle Cell Disease.一项关于 l-谷氨酰胺在镰状细胞病中应用的 3 期临床试验。
N Engl J Med. 2018 Jul 19;379(3):226-235. doi: 10.1056/NEJMoa1715971.
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Development and validation of an oxygen dissociation assay, a screening platform for discovering, and characterizing hemoglobin-oxygen affinity modifiers.氧解离分析方法的开发与验证：一种用于发现和表征血红蛋白-氧亲和力调节剂的筛选平台。
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Case series of patients with severe sickle cell disease treated with voxelotor (GBT440) by compassionate access.通过同情用药途径使用voxelotor（GBT440）治疗的重症镰状细胞病患者病例系列
Am J Hematol. 2018 May 12. doi: 10.1002/ajh.25139.
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