罗沙司他在慢性肾脏病贫血患者中的真实世界安全性和有效性：来自日本一项上市后监测研究的中期结果。
Real-world safety and effectiveness of roxadustat in patients with anemia of chronic kidney disease: interim results from a post-marketing surveillance study in Japan.
作者信息
Tsuruya Kazuhiko, Sugamori Haruko, Tanaka Yusuke, Wakasugi Naoko, Ito Yuichiro
机构信息
Department of Nephrology, Nara Medical University, Nara, Japan.
Astellas Pharma, Inc, Chuo-Ku, Japan.
出版信息
Expert Opin Pharmacother. 2025 Mar;26(4):503-517. doi: 10.1080/14656566.2025.2462181. Epub 2025 Feb 7.
BACKGROUND
This planned interim analysis of a mandatory post-marketing surveillance study in Japan evaluated the safety and effectiveness of roxadustat, the first approved hypoxia-inducible factor prolyl hydroxylase inhibitor in the world, in real-world clinical use.
RESEARCH DESIGN AND METHODS
This prospective observational study has a planned 104-week observation period. We report data obtained through 16 December 2023, as a planned interim analysis. Adverse drug reactions (ADRs), mean hemoglobin level change from baseline to 12 weeks of roxadustat treatment, and subgroup analyses stratified by either baseline C-reactive protein or albumin levels were reported.
RESULTS
Overall, 2084 patients were treated with roxadustat (total patient-years of exposure: 1579.2). In the non-dialysis-dependent (NDD) group ( = 1075), ADRs and serious ADRs occurred in 209 (19.4%) and 109 (10.1%) patients, respectively. In patients receiving hemodialysis (HD; = 856), ADRs and serious ADRs occurred in 224 (26.2%) and 142 (16.6%) patients, respectively. In patients receiving peritoneal dialysis (PD; = 146), ADRs and serious ADRs occurred in 46 (31.5%) and 26 (17.8%) patients, respectively. Mean hemoglobin levels reached target levels at 12 weeks in most patients (NDD: 54.6%; HD: 56.3%; PD: 45.4%).
CONCLUSIONS
Roxadustat safety was demonstrated in real-world clinical settings. No new safety concerns were identified. NCT04408820.
背景
这项针对日本一项强制性上市后监测研究的计划中期分析评估了全球首个获批的缺氧诱导因子脯氨酰羟化酶抑制剂罗沙司他在实际临床应用中的安全性和有效性。
研究设计与方法
这项前瞻性观察性研究计划观察期为104周。我们报告截至2023年12月16日获得的数据，作为计划中的中期分析。报告了药物不良反应（ADR）、罗沙司他治疗12周时血红蛋白水平相对于基线的平均变化，以及按基线C反应蛋白或白蛋白水平分层的亚组分析结果。
结果
总体而言，2084例患者接受了罗沙司他治疗（总暴露患者年数：1579.2）。在非透析依赖（NDD）组（n = 1075）中，分别有209例（19.4%）和109例（10.1%）患者发生了药物不良反应和严重药物不良反应。在接受血液透析（HD；n = 856）的患者中，分别有224例（26.2%）和142例（16.6%）患者发生了药物不良反应和严重药物不良反应。在接受腹膜透析（PD；n = 146）的患者中，分别有46例（31.5%）和26例（17.8%）患者发生了药物不良反应和严重药物不良反应。大多数患者在12周时血红蛋白水平达到目标水平（NDD组：54.6%；HD组：56.3%；PD组：45.4%）。
结论
罗沙司他在实际临床环境中的安全性得到了证实。未发现新的安全问题。NCT04408820。
Zotero 插件