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病例报告:伐昔洛韦对一名镰状细胞病合并IV期慢性肾脏病患者的安全性和有效性

Case report: Safety and efficacy of voxelotor in a patient with sickle cell disease and stage IV chronic kidney disease.

作者信息

Alshurafa Awni, Yassin Mohamed A

机构信息

Hamad Medical Corporation, Doha, Qatar.

出版信息

Front Med (Lausanne). 2022 Sep 15;9:931924. doi: 10.3389/fmed.2022.931924. eCollection 2022.

Abstract

Sickle cell disease (SCD) is a heterogeneous group of inherited disorders characterized by the production of sickle hemoglobin which is less soluble than an adult or fetal hemoglobin. Voxelotor is a hemoglobin S polymerization inhibitor that has been approved for sickle cell disease treatment in the adult and adolescent populations. It acts as a hemoglobin modulator by increasing its affinity to oxygen which prevents red blood cells from sickling. Chronic kidney disease is a common but under-reported complication of SCD and it is a leading cause of morbidity and mortality. The data about the safety and efficacy of voxelotor use in chronic kidney disease is limited. Herein we report a 49-year-old man, with sickle cell disease and stage IV chronic kidney disease, who was managed successfully with voxelotor and resulted in decreasing transfusion requirement and vaso-occlusive painful crisis without affecting kidney function.

摘要

镰状细胞病(SCD)是一组遗传性疾病的异质性群体,其特征是产生比成人或胎儿血红蛋白溶解度更低的镰状血红蛋白。伏克索洛是一种血红蛋白S聚合抑制剂,已被批准用于治疗成人和青少年镰状细胞病。它通过增加对氧气的亲和力来充当血红蛋白调节剂,从而防止红细胞镰变。慢性肾脏病是SCD常见但报告不足的并发症,是发病和死亡的主要原因。关于伏克索洛在慢性肾脏病中使用的安全性和有效性的数据有限。在此,我们报告一名49岁患有镰状细胞病和IV期慢性肾脏病的男性,他使用伏克索洛治疗成功,减少了输血需求和血管闭塞性疼痛危机,且未影响肾功能。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/89ad/9520303/9a3744707a4a/fmed-09-931924-g001.jpg

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