Nangaku Masaomi
Division of Nephrology and Endocrinology, The University of Tokyo Graduate School of Medicine.
Nihon Yakurigaku Zasshi. 2024;159(3):157-159. doi: 10.1254/fpj.23112.
Anemia in chronic kidney disease (CKD) occurs due to insufficient production of erythropoietin to compensate for the decrease in hemoglobin. Anemia in CKD has traditionally been treated with periodic injections of erythropoiesis-stimulating agents (ESAs), which are recombinant human erythropoietin preparations. Although ESA improved anemia in CKD and dramatically improved the quality of life of patients, there are some patients who are hyporesponsive to ESA, and the use of large doses of ESA in these patients may have a negative impact on patient prognosis. Currently, HIF prolyl hydroxylase (HIF-PH) inhibitors have been approved in Japan as a new treatment for anemia in CKD. HIF-PH inhibitors activate HIF and promote the production of endogenous erythropoietin. The 2019 Nobel Prize in Physiology or Medicine was awarded for groundbreaking research that uncovered the HIF pathway. Because HIF-PH inhibitors improve both erythropoietin production and iron metabolism, they are expected to be effective in treating ESA hyporesponsiveness and solve the inconvenience of injectable preparations. On the other hand, its effects are systemic and multifaceted, and long-term effects must be closely monitored.
慢性肾脏病(CKD)中的贫血是由于促红细胞生成素生成不足,无法补偿血红蛋白的减少所致。传统上,CKD患者的贫血是通过定期注射促红细胞生成刺激剂(ESA)来治疗的,ESA是重组人促红细胞生成素制剂。尽管ESA改善了CKD患者的贫血状况,并显著提高了患者的生活质量,但仍有一些患者对ESA反应不佳,在这些患者中使用大剂量ESA可能会对患者预后产生负面影响。目前,低氧诱导因子脯氨酰羟化酶(HIF-PH)抑制剂已在日本获批,作为CKD贫血的一种新疗法。HIF-PH抑制剂可激活HIF并促进内源性促红细胞生成素的产生。2019年诺贝尔生理学或医学奖授予了揭示HIF通路的开创性研究。由于HIF-PH抑制剂既能改善促红细胞生成素的生成,又能改善铁代谢,因此有望有效治疗ESA反应不佳的情况,并解决注射制剂带来的不便。另一方面,其作用是全身性和多方面的,必须密切监测其长期影响。