Niu Junqi, Gao Yanhang, Wang Guiqiang, Qin Zhijie, Wu Cuisong, Yu Zujiang, Wang Lichun, Hu Zhongjie, Li Xing, Zhang Zong, Chen Yue, Yao Lvfeng, Yang Jinhui, Li Guang-Ming, Yang Yida, Lu Xiaobo, Gu Ye, Wu Xiaofeng, Mao Xiaorong, Zhou Zhongyin, Shang Jia, Lin Bingliang, Jia Ji-Dong, Wang Fengmei, Zhang Jiming, Ma Hongyan, Wang Xinrui, Yang Cliff Y, Yang Daichang
Department of Hepatology, Center of Infectious Diseases and Pathogen Biology, The First Hospital of Jilin University, Changchun, Jilin, China
China-Singapore Belt and Road Joint Laboratory on Liver Disease Research, Changchun, China.
Gut. 2025 Aug 7;74(9):1476-1485. doi: 10.1136/gutjnl-2025-335577.
Despite inadequate supply and potential contamination risk, human plasma has remained the only source for human serum albumin (pHSA) intravenous administration since the 1940s.
We sought to establish the safety and efficacy of OsrHSA, a recombinant HSA from bioengineered (rice).
In this multicentre, randomised, double-blind and positive-controlled study, patients with decompensated liver cirrhosis and serum albumin ≤30 g/L were recruited from 22 centres in China. The patients were randomly assigned to OsrHSA or pHSA (4:1) to once-daily intravenous injection (10 g or 20 g) until their serum albumin level reached 35 g/L, for a maximum of 2 weeks, with 2 weeks of follow-up. The primary outcome was the proportion of patients to reach a serum albumin level of 35 g/L (non-inferiority margin <-0.20). Outcomes were evaluated in patients who received the study drug and had at least one post-baseline serum albumin value (full analysis set, FAS). Safety was evaluated in all patients who received the study drug.
Between 22 March 2021 and 2 June 2022, 220 patients received OsrHSA (n=175) or pHSA (n=45). 216 patients were included in the FAS (OsrHSA, n=171; pHSA, n=45). Primary outcome of OsrHSA (130/171, 76%) was non-inferior to pHSA (34/45, 75.6%) (difference=0.5%; lower limit of 97.5% CI=-0.119). There was no significant difference between all secondary outcomes of OsrHSA and pHSA. There were no drug-related serious adverse events.
Rice-derived HSA is non-inferior to plasma-derived HSA in efficacy and safety. This finding should be confirmed in phase 3 trial.
NCT04835480.
尽管存在供应不足和潜在污染风险，但自20世纪40年代以来，人血浆一直是静脉注射人血清白蛋白（pHSA）的唯一来源。
我们试图确定来自生物工程（水稻）的重组人血清白蛋白（OsrHSA）的安全性和有效性。
在这项多中心、随机、双盲和阳性对照研究中，从中国22个中心招募了失代偿期肝硬化且血清白蛋白≤30g/L的患者。患者被随机分配至OsrHSA组或pHSA组（4:1），每日静脉注射一次（10g或20g），直至血清白蛋白水平达到35g/L，最长持续2周，并进行2周的随访。主要结局是血清白蛋白水平达到35g/L的患者比例（非劣效界值<-0.20）。在接受研究药物且至少有一个基线后血清白蛋白值的患者中评估结局（全分析集，FAS）。在所有接受研究药物的患者中评估安全性。
在2021年3月22日至2022年6月2日期间，220例患者接受了OsrHSA（n=175）或pHSA（n=45）治疗。216例患者纳入FAS（OsrHSA组，n=171；pHSA组，n=45）。OsrHSA组的主要结局（130/171，76%）不劣于pHSA组（34/45，75.6%）（差异=0.5%；97.5%CI下限=-0.119）。OsrHSA组和pHSA组的所有次要结局之间无显著差异。未发生与药物相关的严重不良事件。
水稻来源的人血清白蛋白在疗效和安全性方面不劣于血浆来源的人血清白蛋白。这一发现应在3期试验中得到证实。
NCT04835480。
