Long-term safety and effectiveness of roxadustat in Chinese patients with chronic kidney disease-anemia: The ROXSTAR registry.

IF 7.5 3区医学 Q1 MEDICINE, GENERAL & INTERNAL

Chinese Medical Journal Pub Date : 2025-05-29 DOI:10.1097/CM9.0000000000003672

Xiaoying Du, Yaomin Wang, Haifeng Yu, Jurong Yang, Weiming He, Zunsong Wang, Dongwen Zheng, Xiaowei Li, Shuijuan Shen, Dong Sun, Weimin Yu, Detian Li, Changyun Qian, Yiqing Wu, Shuting Pan, Jianghua Chen

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引用次数: 0

Abstract

Background: Chronic kidney disease (CKD)-associated anemia (CKD-anemia) is associated with poor survival, and hemoglobin targets are often not achieved with current therapies. Phase 3 trials have demonstrated the treatment efficacy of roxadustat for CKD-anemia. This phase four study aims to evaluate the long-term (52-week) safety and effectiveness of roxadustat in a broad real-world patient population with CKD-anemia with and without dialysis in China.

Methods: This Phase 4 multicenter, open-label, prospective study, conducted from 24 November 2020 to 11 November 2022, evaluated the long-term safety and effectiveness of roxadustat for CKD-anemia in China. Patients aged ≥18 years with CKD-anemia with or without dialysis were included. The initial oral dose was 70-120 mg (weight-based followed by dose adjustment) over 52 weeks. The primary endpoint was safety based on adverse events (AEs). The secondary endpoints were hemoglobin changes from baseline and the proportion of patients who achieved mean hemoglobin ≥100 g/L. Effectiveness evaluable populations 1 (EE1) and EE2 included roxadustat-naïve and previously roxadustat-treated patients, respectively. The safety analysis set (SAF) included all patients who received ≥1 dose.

Results: The EE1, EE2, and SAF populations included 1804, 193, and 2021 patients, respectively. In the SAF, the mean age was 50 ± 14 years, and 1087 patients (53.8%) were male. Mean baseline hemoglobin was 96.9 ± 14.0 g/L in EE1 and 100.3 ± 12.9 g/L in EE2. In EE1, the mean (95% confidence interval) hemoglobin changes from baseline over weeks 24-36 and 36-52 were 14.2 (13.5-14.9) g/L and 14.3 (13.5-15.0) g/L, respectively. Over weeks 24-36 and 36-52, 83.3% and 86.1% of patients in EE1 and 82.7% and 84.7% in EE2 achieved mean hemoglobin ≥100 g/L, respectively. In the SAF, 1643 patients (81.3%) experienced treatment-emergent AEs (TEAEs). Overall, 219 patients (10.8%) experienced drug-related TEAEs. Thirty-eight patients (1.9%) died of TEAEs (unrelated to the study drug). Vascular access thrombosis was uncommon.

Conclusions: Roxadustat (52 weeks) increased hemoglobin and maintained the treatment target in Chinese patients with CKD-anemia with acceptable safety, supporting its use in real-world settings.

Registration: Chinese Clinical Trial Registry (www.chictr.org.cn) ChiCTR2100046322 CDE (www.chinadrugtrials.org.cn) CTR20201568.

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罗沙他在中国慢性肾病-贫血患者中的长期安全性和有效性：ROXSTAR注册

背景：慢性肾脏疾病（CKD）相关性贫血（CKD-anemia）与较差的生存率相关，目前的治疗方法通常无法达到血红蛋白目标。3期试验已经证明了罗沙司他治疗慢性肾病贫血的疗效。这项4期研究旨在评估罗沙司他在中国广泛的现实世界ckd -贫血伴透析和不伴透析患者群体中的长期（52周）安全性和有效性。方法：这项4期多中心、开放标签、前瞻性研究于2020年11月24日至2022年11月11日在中国进行，评估罗昔他特治疗ckd -贫血的长期安全性和有效性。年龄≥18岁伴有或不伴有透析的ckd贫血患者纳入研究。初始口服剂量为70- 120mg（以体重为基础，随后调整剂量），持续52周。主要终点是基于不良事件（ae）的安全性。次要终点是血红蛋白较基线的变化和达到平均血红蛋白≥100 g/L的患者比例。有效性可评估人群1 （EE1）和EE2分别包括roxadustat-naïve和先前接受罗沙司他治疗的患者。安全性分析集（SAF）包括所有接受≥1剂量的患者。结果：EE1、EE2和SAF人群分别包括1804、193和2021例患者。SAF患者平均年龄50±14岁，男性1087例（53.8%）。EE1的平均基线血红蛋白为96.9±14.0 g/L， EE2为100.3±12.9 g/L。在EE1中，24-36周和36-52周血红蛋白与基线相比的平均（95%置信区间）变化分别为14.2 (13.5-14.9)g/L和14.3 (13.5-15.0)g/L。在24-36周和36-52周，EE1组83.3%和86.1%的患者平均血红蛋白≥100 g/L， EE2组82.7%和84.7%的患者平均血红蛋白≥100 g/L。在SAF中，1643名患者（81.3%）经历了治疗突发事件（teae）。总体而言，219名患者（10.8%）经历了与药物相关的teae。38例（1.9%）患者死于teae（与研究药物无关）。血管通路血栓不常见。结论：罗沙司他（52周）提高了中国ckd贫血患者的血红蛋白并维持了治疗目标，安全性可接受，支持其在现实环境中的使用。注册：中国临床试验注册中心（www.chictr.org.cn） ChiCTR2100046322 CDE (www.chinadrugtrials.org.cn) CTR20201568。

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来源期刊

Chinese Medical Journal 医学-医学：内科

CiteScore

9.80

自引率

4.90%

发文量

19245

审稿时长

6 months

期刊介绍： The Chinese Medical Journal (CMJ) is published semimonthly in English by the Chinese Medical Association, and is a peer reviewed general medical journal for all doctors, researchers, and health workers regardless of their medical specialty or type of employment. Established in 1887, it is the oldest medical periodical in China and is distributed worldwide. The journal functions as a window into China’s medical sciences and reflects the advances and progress in China’s medical sciences and technology. It serves the objective of international academic exchange. The journal includes Original Articles, Editorial, Review Articles, Medical Progress, Brief Reports, Case Reports, Viewpoint, Clinical Exchange, Letter,and News,etc. CMJ is abstracted or indexed in many databases including Biological Abstracts, Chemical Abstracts, Index Medicus/Medline, Science Citation Index (SCI), Current Contents, Cancerlit, Health Plan & Administration, Embase, Social Scisearch, Aidsline, Toxline, Biocommercial Abstracts, Arts and Humanities Search, Nuclear Science Abstracts, Water Resources Abstracts, Cab Abstracts, Occupation Safety & Health, etc. In 2007, the impact factor of the journal by SCI is 0.636, and the total citation is 2315.