Du Xiaoying, Wang Yaomin, Yu Haifeng, Yang Jurong, He Weiming, Wang Zunsong, Zheng Dongwen, Li Xiaowei, Shen Shuijuan, Sun Dong, Yu Weimin, Li Detian, Qian Changyun, Wu Yiqing, Pan Shuting, Chen Jianghua
Kidney Disease Center, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310003, China.
Department of Nephrology, Taizhou Central Hospital, Taizhou, Zhejiang 318001, China.
Chin Med J (Engl). 2025 Jun 20;138(12):1465-1476. doi: 10.1097/CM9.0000000000003672. Epub 2025 May 29.
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 4 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.
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 occasion.
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 (81.3%) patients experienced treatment-emergent AEs (TEAEs). Overall, 219 (10.8%) patients experienced drug-related TEAEs. Thirty-eight (1.9%) patients died of TEAEs (unrelated to the study drug). Vascular access thrombosis was uncommon.
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.
Chinese Clinical Trial Registry ( www.chictr.org.cn ) ChiCTR2100046322; CDE ( www.chinadrugtrials.org.cn ) CTR20201568.
慢性肾脏病（CKD）相关贫血（CKD-贫血）与生存率低相关，且目前的治疗方法往往无法达到血红蛋白目标。3期试验已证明罗沙司他对CKD-贫血的治疗效果。这项4期研究旨在评估罗沙司他在中国广泛的伴有或不伴有透析的CKD-贫血真实世界患者群体中的长期（52周）安全性和有效性。
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日进行，评估了罗沙司他在中国CKD-贫血患者中的长期安全性和有效性。纳入了年龄≥18岁的伴有或不伴有透析的CKD-贫血患者。初始口服剂量为70-120毫克（基于体重，随后进行剂量调整），疗程为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相对于基线的变化以及达到平均血红蛋白≥100克/升的患者比例。有效性可评估人群1（EE1）和EE2分别包括既往未使用过罗沙司他和既往使用过罗沙司他的患者。安全性分析集（SAF）包括所有接受过≥1次治疗的患者。
EE1、EE2和SAF人群分别包括1804例、193例和2021例患者。在SAF中，平均年龄为50±14岁，1087例患者（53.8%）为男性。EE1组的平均基线血红蛋白为96.9±14.0克/升，EE2组为100.3±12.9克/升。在EE1中，第24至36周和第36至52周相对于基线的平均（95%置信区间）血红蛋白变化分别为14.2（13.5-14.9）克/升和14.3（13.5-15.0）克/升。在第24至36周和第36至52周，EE1组分别有83.3%和86.1%的患者、EE2组分别有82.7%和84.7%的患者达到平均血红蛋白≥100克/升。在SAF中，1643例（81.3%）患者发生治疗中出现的不良事件（TEAE）。总体而言，219例（10.8%）患者发生与药物相关的TEAE。38例（1.9%）患者死于TEAE（与研究药物无关）。血管通路血栓形成并不常见。
罗沙司他（52周）可提高血红蛋白水平，并在中国CKD-贫血患者中维持治疗目标，安全性可接受，支持其在真实世界中的应用。
中国临床试验注册中心（www.chictr.org.cn）ChiCTR2100046322；国家药品监督管理局药品审评中心（www.chinadrugtrials.org.cn）CTR20201568。
