罗沙司他治疗慢性肾病患者纯红细胞发育不全1例。

IF 1.7 4区 生物学 Q3 BIOLOGY
Open Life Sciences Pub Date : 2025-10-04 eCollection Date: 2025-01-01 DOI:10.1515/biol-2025-1155
Shanlin Liu, Bonan Yan, Yuhua He
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引用次数: 0

摘要

纯红细胞发育不全(PRCA)是一种罕见的血液疾病,其特征是严重的低红细胞骨髓发育不全,导致严重的贫血,通常对标准治疗无效。慢性肾脏疾病(CKD)患者,特别是对促红细胞生成素(ESA)有耐药性的患者,治疗相当困难。在本病例报告中,我们将介绍一位67岁的CKD女性(未透析),她表现为难治性贫血(血红蛋白45-65 g/L),并在骨髓抽吸后最终被诊断为PRCA。该患者先前接受过治疗(esa、补铁和多次输血),但其血细胞计数没有改善。开始口服罗沙司他100mg t.i.w后,患者血红蛋白逐渐升高,稳定在100 ~ 106 g/L之间,不再需要输血。本病例报告强调了罗沙司他作为PRCA和esa耐药CKD的新治疗选择的潜在作用。虽然这些数据令人鼓舞,但需要更大规模的对照研究来评估该人群的疗效、剂量和长期安全性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Treatment of pure red cell aplasia in a chronic kidney disease patient with roxadustat: A case report.

Pure red cell aplasia (PRCA) is a rare blood disorder that is characterized by severe hypo-erythroid bone marrow hypoplasia leading to severe anemia that usually does not respond to standard treatment. In patients with chronic kidney disease (CKD), especially in those patients who are erythropoiesis-stimulating agent (ESA) resistant, management can be quite difficult. In this case report, we will present a 67-year-old woman with CKD (not on dialysis) who presented with refractory anemia (hemoglobin 45-65 g/L) and was ultimately diagnosed with PRCA after bone marrow aspirate. This patient had previously received treatment (ESAs, iron supplementation, and multiple blood transfusions) without improvement in her blood counts. After initiation of oral Roxadustat 100 mg t.i.w., her hemoglobin increased gradually and stabilized between 100 and 106 g/L, and she no longer required blood transfusions. This case report highlights the potential role of Roxadustat as a new therapeutic option for PRCA and ESA-resistant CKD. While these data are encouraging, larger controlled studies are going to be required to evaluate measures of efficacy, dosing, and long-term safety in this population.

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来源期刊
CiteScore
2.50
自引率
4.50%
发文量
131
审稿时长
43 weeks
期刊介绍: Open Life Sciences (previously Central European Journal of Biology) is a fast growing peer-reviewed journal, devoted to scholarly research in all areas of life sciences, such as molecular biology, plant science, biotechnology, cell biology, biochemistry, biophysics, microbiology and virology, ecology, differentiation and development, genetics and many others. Open Life Sciences assures top quality of published data through critical peer review and editorial involvement throughout the whole publication process. Thanks to the Open Access model of publishing, it also offers unrestricted access to published articles for all users.
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