Department of Medicine, Vanderbilt University Medical Center, Nashville, TN.
Department of Medicine, Vanderbilt University Medical Center, Nashville, TN; Department of Medical Cell Biology, Uppsala Universitet, Uppsala, Sweden; Department of Molecular Physiology & Biophysics and Program in Cancer Biology, Vanderbilt University School of Medicine, Nashville, TN.
Adv Chronic Kidney Dis. 2019 Jul;26(4):253-266. doi: 10.1053/j.ackd.2019.04.004.
Prolyl hydroxylase domain oxygen sensors are dioxygenases that regulate the activity of hypoxia-inducible factor (HIF), which controls renal and hepatic erythropoietin production and coordinates erythropoiesis with iron metabolism. Small molecule inhibitors of prolyl hydroxylase domain dioxygenases (HIF-PHI [prolyl hydroxylase inhibitor]) stimulate the production of endogenous erythropoietin and improve iron metabolism resulting in efficacious anemia management in patients with CKD. Three oral HIF-PHIs-daprodustat, roxadustat, and vadadustat-have now advanced to global phase III clinical development culminating in the recent licensing of roxadustat for oral anemia therapy in China. Here, we survey current clinical experience with HIF-PHIs, discuss potential therapeutic advantages, and deliberate over safety concerns regarding long-term administration in patients with renal anemia.
脯氨酰羟化酶结构域氧传感器是一种双加氧酶,可调节缺氧诱导因子 (HIF) 的活性,后者控制肾脏和肝脏促红细胞生成素的产生,并协调红细胞生成与铁代谢。脯氨酰羟化酶结构域双加氧酶(HIF-PHI[脯氨酰羟化酶抑制剂])的小分子抑制剂可刺激内源性促红细胞生成素的产生并改善铁代谢,从而有效地治疗 CKD 患者的贫血。目前,三种口服 HIF-PHIs——达普司他、罗沙司他和伐达司他——已进入全球 III 期临床开发阶段,最终罗沙司他在中国获得了口服贫血治疗的许可。在此,我们调查了 HIF-PHI 的临床应用经验,讨论了其潜在的治疗优势,并就长期治疗肾性贫血患者的安全性问题进行了审议。