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达泊西汀成功治疗慢性肾脏病合并低危骨髓增生异常综合征相关性贫血:两例报告

Successful treatment of low-risk myelodysplastic syndrome-related anemia in patients with chronic kidney disease with daprodustat: A report of two cases.

作者信息

Kunimoto Hiroyoshi, Sakuma Takayuki, Ohashi Takuma, Shirafuta Mayoko, Teranaka Hiroshi, Nakajima Hideaki

机构信息

Department of Stem Cell and Immune Regulation Yokohama City University Graduate School of Medicine Yokohama Japan.

出版信息

EJHaem. 2024 Nov 25;5(6):1335-1339. doi: 10.1002/jha2.1057. eCollection 2024 Dec.

DOI:10.1002/jha2.1057
PMID:39691241
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11647689/
Abstract

Anemia is a major clinical manifestation seen in myelodysplastic syndromes (MDS). Treatment options for anemia in low-risk MDS are limited. Especially, oral medication which is uniformly effective for anemia in low-risk MDS is required. Hypoxia-inducible factor (HIF) prolyl hydroxylase (HIF-PH) inhibitors, such as daprodustat, are oral tablets effective for renal anemia. Pharmacological restoration of HIF activity by HIF-PH inhibitors may be beneficial for MDS-related anemia as well. Yet, their efficacy and safety against low-risk MDS are unclear. Here, we report two cases of low-risk MDS complicated with chronic kidney disease whose anemia responded to daprodustat treatment.

摘要

贫血是骨髓增生异常综合征(MDS)的主要临床表现。低危MDS贫血的治疗选择有限。特别是,需要一种对低危MDS贫血普遍有效的口服药物。缺氧诱导因子(HIF)脯氨酰羟化酶(HIF-PH)抑制剂,如达普司他,是治疗肾性贫血有效的口服片剂。HIF-PH抑制剂对HIF活性的药理恢复可能对MDS相关贫血也有益。然而,它们对低危MDS的疗效和安全性尚不清楚。在此,我们报告两例合并慢性肾脏病的低危MDS患者,其贫血对达普司他治疗有反应。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b32f/11647689/b2784bbfc79e/JHA2-5-1335-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b32f/11647689/7a0849faa713/JHA2-5-1335-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b32f/11647689/b2784bbfc79e/JHA2-5-1335-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b32f/11647689/7a0849faa713/JHA2-5-1335-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b32f/11647689/b2784bbfc79e/JHA2-5-1335-g002.jpg

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本文引用的文献

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Roxadustat versus placebo for patients with lower-risk myelodysplastic syndrome: MATTERHORN phase 3, double-blind, randomized controlled trial.罗沙司他与安慰剂治疗低危骨髓增生异常综合征患者的比较:MATTERHORN 期 3 双盲随机对照试验。
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Hypoxia-Inducible Factor-Prolyl-Hydroxylase and Sodium-Glucose Cotransporter 2 Inhibitors for Low-Risk Myelodysplastic Syndrome-Related Anemia in Patients with Chronic Kidney Disease: A Report of Three Cases.缺氧诱导因子脯氨酰羟化酶与钠-葡萄糖协同转运蛋白2抑制剂治疗慢性肾脏病患者低危骨髓增生异常综合征相关贫血:三例报告
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Daprodustat for the Treatment of Anemia in Patients Undergoing Dialysis.达普司他用于治疗透析患者的贫血
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