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Article(id=1193877802572279942, tenantId=1146029695717560320, journalId=1190317699101192196, issueId=1193877800143777917, articleNumber=1001-2494(2025)02-0194-07, orderNo=null, doi=10.11669/cpj.2025.02.013, pmid=null, cstr=null, oa=null, hot=null, price=null, onlineType=0, articleFormat=0, articleType=null, articleTypeStr=research-article, receivedDate=1711987200000, receivedDateStr=2024-04-02, revisedDate=null, revisedDateStr=null, acceptedDate=null, acceptedDateStr=null, onlineDate=1762572249310, onlineDateStr=2025-11-08, pubDate=1737475200000, pubDateStr=2025-01-22, doiRegisterDate=null, doiRegisterDateStr=null, onlineIssueDate=1762572249310, onlineIssueDateStr=2025-11-08, onlineJustAcceptDate=null, onlineJustAcceptDateStr=null, onlineFirstDate=null, onlineFirstDateStr=null, sourceXml=null, magXml=null, createTime=1762572249310, creator=13701087609, updateTime=1762572249310, updator=13701087609, issue=Issue{id=1193877800143777917, tenantId=1146029695717560320, journalId=1190317699101192196, year='2025', volume='60', issue='2', pageStart='109', pageEnd='206', issueExtLink='null', onlineDate='null', pubDate='null', beforeIssueId=null, nextIssueId=null, price=null, status=1, issueComplete=0, articleOrder=1, issueType=-1, specialIssue=null, createTime=1762572248731, creator=13701087609, updateTime=1762584852274, updator=13701087609, preIssue=null, nextIssue=null, ext={EN=IssueExt(id=1193930663289123481, tenantId=1146029695717560320, journalId=1190317699101192196, issueId=1193877800143777917, language=EN, specialIssueTitle=, coverIllustrator=null, specialIssueEditor=, specialIssueAbout=), CN=IssueExt(id=1193930663289123482, tenantId=1146029695717560320, journalId=1190317699101192196, issueId=1193877800143777917, language=CN, specialIssueTitle=, coverIllustrator=null, specialIssueEditor=, specialIssueAbout=)}, issueFiles=null}, startPage=194, endPage=200, ext={EN=ArticleExt(id=1193877802756829319, articleId=1193877802572279942, tenantId=1146029695717560320, journalId=1190317699101192196, language=EN, title=Indirect Efficacy Comparison and Cost-utility Analysis of Enarodustat in the Treatment of Renal Anemia in Patients with Non-Dialysis Chronic Kidney Disease, columnId=null, journalTitle=Chinese Pharmaceutical Journal, columnName=null, runingTitle=null, highlight=null, articleAbstract=

OBJECTIVE To evaluate the efficacy and cost-effectiveness of enarodustat in the treatment of renal anemia in non-dialysis patients. METHODS A matched-adjusted indirect comparison of the efficacy of enarodustat and roxadustat was conducted based on their respective phase Ⅲ clinical trials, and the results were used for cost-utility analysis of enarodustat in the treatment of renal anemia in non-dialysis patients. The robustness of the incremental cost-effectiveness ratio was verified through sensitivity analysis and scenario analysis. RESULTS After matching and adjusting the baseline characteristics of the trial population, it was shown that enarodustat was able to significantly increase the proportion of subjects with average hemoglobin ≥10.0 g·dL-1 at weeks 7-9 compared with roxadustat (73.5% vs 61.0%, MD=12.5%, P<0.001); the results of cost-utility analysis showed that compared with roxadustat, enarodustat increased patients' quality-adjusted life years and saved costs, making it an absolutely superior solution.CONCLUSION Compared with roxadustat, enarodustat is more effective and economical in treating renal anemia in patients with non-dialysis chronic kidney disease.

, correspAuthors=Xiaoning HE, authorNote=null, correspAuthorsNote=null, copyrightStatement=null, copyrightOwner=null, extLink=null, articleAbsUrl=null, sourceXml=null, magXml=null, pdfUrl=null, pdf=null, pdfFileSize=null, pdfExtLink=null, richHtmlUrl=null, mobilePdfUrl=null, reviewReport=null, pdfFirstPage=null, abstractGraph=null, abstractGraphContent=null, abstractVideo=null, citation=null, cebUrl=null, magXmlContent=null, mapNumber=null, authorCompany=null, fund=null, authors=null, authorsList=Jinsheng GAO, Lei GAN, Ying LIU, Xiaoning HE), CN=ArticleExt(id=1193877873397297385, articleId=1193877802572279942, tenantId=1146029695717560320, journalId=1190317699101192196, language=CN, title=恩那度司他治疗非透析慢性肾脏病患者肾性贫血的疗效间接比较与成本-效用分析, columnId=1190352405612040510, journalTitle=中国药学杂志, columnName=论著, runingTitle=null, highlight=null, articleAbstract=

目的 评价恩那度司他治疗非透析患者肾性贫血的疗效和经济性。方法 基于恩那度司他和罗沙司他各自的Ⅲ期临床试验开展了疗效的匹配调整间接比较,并将其结果用于恩那度司他治疗非透析患者肾性贫血的成本-效用分析。通过敏感性分析和情境分析,验证增量成本效果比的稳健性。结果 匹配调整试验人群的基线特征后,恩那度司他相比于罗沙司他能够显著提高第7~9周平均血红蛋白≥10.0 g·dL-1的受试者比例(73.5% vs 61.0%,MD=12.5%,P<0.001);成本-效用分析结果显示,与罗沙司他相比,恩那度司他增加患者质量调整生命年的同时节约了医疗成本,为绝对优势方案。结论 与罗沙司他相比,恩那度司他治疗非透析慢性肾脏病患者肾性贫血的疗效更好,且具有较好的经济性。

, correspAuthors=贺小宁, authorNote=null, correspAuthorsNote=
*贺小宁,女,博士,副教授 研究方向:卫生经济学与药物经济学 Tel: (022) 87401830
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高金生,男,硕士研究生 研究方向:药物经济学

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高金生,男,硕士研究生 研究方向:药物经济学

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高金生,男,硕士研究生 研究方向:药物经济学

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Keyword(id=1193928820542636546, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, orderNo=3, keyword=恩那度司他), Keyword(id=1193928820639105539, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, orderNo=4, keyword=匹配调整间接比较), Keyword(id=1193928820718797316, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, orderNo=5, keyword=成本-效用分析)], refs=[Reference(id=1193928822358770199, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, doi=null, pmid=null, pmcid=null, year=2021, volume=101, issue=20, pageStart=1463, pageEnd=1502, url=null, language=null, rfNumber=[1], rfOrder=0, authorNames=null, journalName=Natl Med J China(中华医学杂志), refType=null, unstructuredReference=Renal anemia Guideline Working Group of Nephrology Physicians Branch of Chinese Medical Doctor Association. 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Kidney Int Rep, 2021, 6(7): 1840-1849., articleTitle=A phase 3 study of enarodustat in anemic patients with CKD not requiring dialysis: the SYMPHONY ND study, refAbstract=null)], funds=null, companyList=[AuthorCompany(id=1193928818126717416, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, xref=null, ext=[AuthorCompanyExt(id=1193928818135106025, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, companyId=1193928818126717416, language=EN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=School of Pharmaceutical Science and Technology, Tianjin University, Tianjin 300072, China), AuthorCompanyExt(id=1193928818143494634, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, companyId=1193928818126717416, language=CN, country=null, province=null, city=null, postcode=null, companyName=null, departmentName=null, remark=天津大学药物科学与技术学院, 天津 300072)])], figs=[ArticleFig(id=1193928820928512517, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=cY6GqRZs7CNKplNgZZPzmg==, figureFileBig=3iEjufW8gp5j2RdclT0zyg==, tableContent=null), ArticleFig(id=1193928821020787206, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=图1, caption=3个互斥的健康状态Markov模型结构, figureFileSmall=cY6GqRZs7CNKplNgZZPzmg==, figureFileBig=3iEjufW8gp5j2RdclT0zyg==, tableContent=null), ArticleFig(id=1193928821104673287, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=3TApSH09nlOXnJeW2NT52Q==, figureFileBig=IpGd3zFt5rNtj1QiAU+Sig==, tableContent=null), ArticleFig(id=1193928821167587848, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=图2, caption=恩那度司他和于罗沙司他单因素敏感性分析龙卷风图, figureFileSmall=3TApSH09nlOXnJeW2NT52Q==, figureFileBig=IpGd3zFt5rNtj1QiAU+Sig==, tableContent=null), ArticleFig(id=1193928821293416969, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=null, figureFileBig=null, tableContent=
序号 试验简称 研究设计 研究时间 研究地域 治疗方案
干预组 对照组
1 OLYMPUS 安慰剂对照、随机、多中心 2014/06-2018/10 25个国家和地区 罗沙司他 安慰剂
2 ALPS 安慰剂对照、随机、多中心 2013/09-2017/11 22个国家 罗沙司他 安慰剂
3 ANDES 阳性对照、部分随机、多中心 2017/01-2019/09 全球 罗沙司他 达依伯汀α
4 DOLOMITES 阳性对照、随机、多中心 2014/03-2018/06 欧洲 罗沙司他 达依伯汀α
5 1517-CL-0314 非对照、随机、开放、多中心 2017/01-2018/08 日本 罗沙司他(50 mg) 罗沙司他(70 mg)
6 FGCL-4592-808 安慰剂对照、随机、多中心 2015/12-2017/01 中国 罗沙司他 安慰剂
), ArticleFig(id=1193928821360525834, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=表1, caption=

OLYMPUS,ALPS,ANDES,DOLOMITES,1517-CL-0314,FGCL-4592-808 6个罗沙司他试验的基本信息

, figureFileSmall=null, figureFileBig=null, tableContent=
序号 试验简称 研究设计 研究时间 研究地域 治疗方案
干预组 对照组
1 OLYMPUS 安慰剂对照、随机、多中心 2014/06-2018/10 25个国家和地区 罗沙司他 安慰剂
2 ALPS 安慰剂对照、随机、多中心 2013/09-2017/11 22个国家 罗沙司他 安慰剂
3 ANDES 阳性对照、部分随机、多中心 2017/01-2019/09 全球 罗沙司他 达依伯汀α
4 DOLOMITES 阳性对照、随机、多中心 2014/03-2018/06 欧洲 罗沙司他 达依伯汀α
5 1517-CL-0314 非对照、随机、开放、多中心 2017/01-2018/08 日本 罗沙司他(50 mg) 罗沙司他(70 mg)
6 FGCL-4592-808 安慰剂对照、随机、多中心 2015/12-2017/01 中国 罗沙司他 安慰剂
), ArticleFig(id=1193928821419246091, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=null, figureFileBig=null, tableContent=
参数名称 恩那度司他转移概率/% 罗沙司他转移概率/% 数据来源
转移概率
Hb<10 g·dL-1 45.15 57.32 计算
Hb≥10 g·dL-11) 54.72 42.55 FGCL-4592-808试验,MAIC研究
死亡 0.12 0.12 文献[11-12]
Hb≥10 g·dL-1 77.60 59.33 NCT06016036试验,文献[13-14]
Hb<10 g·dL-1 22.32 40.59 计算
死亡 0.08 0.08 文献[11-12]
不良事件发生率
代谢性酸中毒 0.25 6.13 FGCL-4592-808试验,MAIC研究
高钾血症 9.39 8.26
上呼吸道感染 7.26 2.51
), ArticleFig(id=1193928821494743564, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=表2, caption=

Markov模型状态转移概率、不良事件发生率及其来源

, figureFileSmall=null, figureFileBig=null, tableContent=
参数名称 恩那度司他转移概率/% 罗沙司他转移概率/% 数据来源
转移概率
Hb<10 g·dL-1 45.15 57.32 计算
Hb≥10 g·dL-11) 54.72 42.55 FGCL-4592-808试验,MAIC研究
死亡 0.12 0.12 文献[11-12]
Hb≥10 g·dL-1 77.60 59.33 NCT06016036试验,文献[13-14]
Hb<10 g·dL-1 22.32 40.59 计算
死亡 0.08 0.08 文献[11-12]
不良事件发生率
代谢性酸中毒 0.25 6.13 FGCL-4592-808试验,MAIC研究
高钾血症 9.39 8.26
上呼吸道感染 7.26 2.51
), ArticleFig(id=1193928821557658125, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=null, figureFileBig=null, tableContent=
模型参数 数值(范围) 分布
成本
恩那度司他价格/元(4 mg)1) 25.93(20.74~25.93) Gamma分布
罗沙司他价格/元(50 mg)1) 54.00(43.20~54.00) Gamma分布
疾病管理成本2)
Hb<10 g·dL-1状态 307.51(246.00~369.01) Gamma分布
Hb≥10 g·dL-1状态 118.35(94.68~142.02) Gamma分布
输血成本2) 867.07(693.65~1,040.48)
不良事件治疗成本3)
代谢性酸中毒[17] 200.00(160.00~240.00) Gamma分布
高钾血症[18] 239.33(191.46~287.20) Gamma分布
上呼吸道感染[19] 201.34(161.07~241.61) Gamma分布
效用
健康状态效用值
Hb<10 g·dL-1状态[2] 0.74(0.70~0.77) Beta分布
Hb≥10 g·dL-1状态[2] 0.82(0.78~0.86) Beta分布
不良事件负效用值3)
代谢性酸中毒[20] -0.0024(-0.002 2~-0.002 5) Beta分布
高钾血症[21] -0.003 4(-0.003 3~-0.003 6) Beta分布
上呼吸道感染[22] -0.000 2(-0.000 1~-0.000 2) Beta分布
), ArticleFig(id=1193928821624766990, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=表3, caption=

纳入直接医疗成本Markov模型的输入参数

, figureFileSmall=null, figureFileBig=null, tableContent=
模型参数 数值(范围) 分布
成本
恩那度司他价格/元(4 mg)1) 25.93(20.74~25.93) Gamma分布
罗沙司他价格/元(50 mg)1) 54.00(43.20~54.00) Gamma分布
疾病管理成本2)
Hb<10 g·dL-1状态 307.51(246.00~369.01) Gamma分布
Hb≥10 g·dL-1状态 118.35(94.68~142.02) Gamma分布
输血成本2) 867.07(693.65~1,040.48)
不良事件治疗成本3)
代谢性酸中毒[17] 200.00(160.00~240.00) Gamma分布
高钾血症[18] 239.33(191.46~287.20) Gamma分布
上呼吸道感染[19] 201.34(161.07~241.61) Gamma分布
效用
健康状态效用值
Hb<10 g·dL-1状态[2] 0.74(0.70~0.77) Beta分布
Hb≥10 g·dL-1状态[2] 0.82(0.78~0.86) Beta分布
不良事件负效用值3)
代谢性酸中毒[20] -0.0024(-0.002 2~-0.002 5) Beta分布
高钾血症[21] -0.003 4(-0.003 3~-0.003 6) Beta分布
上呼吸道感染[22] -0.000 2(-0.000 1~-0.000 2) Beta分布
), ArticleFig(id=1193928821687681551, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=null, figureFileBig=null, tableContent=
基线特征 罗沙司他试验
(n=152)		 恩那度司他试验
匹配前(n=155) 匹配后(ESS=51)
年龄/岁 54.2 54.3 54.2
男性比例/% 37.0 40.0 37.0
血红蛋白/mg·dL-1 8.9 9.4 8.9
估算肾小球滤过率/mL·min-1·1.73 m-2 15.8 19.2 15.8
收缩压/mm Hg 134.3 125.4 134.3
舒张压/mm Hg 80.2 76.2 80.2
总胆固醇/mg·dL-1 175.7 179.6 175.7
低密度脂蛋白胆固醇/mg·dL-1 100.3 102.6 100.3
高密度脂蛋白胆固醇/mg·dL-1 49.5 48.0 49.5
转铁蛋白饱和度/% 21.4 27.6 21.4
铁蛋白/μg·L-1 216.5 223.7 216.5
超敏C反应蛋白>4.9 mg·L-1比例/% 11.0 21.3 11.0
), ArticleFig(id=1193928821746401808, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=表4, caption=

分析并识别可直接作用于治疗结果或可能影响疗效的试验人群基线特征

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基线特征 罗沙司他试验
(n=152)		 恩那度司他试验
匹配前(n=155) 匹配后(ESS=51)
年龄/岁 54.2 54.3 54.2
男性比例/% 37.0 40.0 37.0
血红蛋白/mg·dL-1 8.9 9.4 8.9
估算肾小球滤过率/mL·min-1·1.73 m-2 15.8 19.2 15.8
收缩压/mm Hg 134.3 125.4 134.3
舒张压/mm Hg 80.2 76.2 80.2
总胆固醇/mg·dL-1 175.7 179.6 175.7
低密度脂蛋白胆固醇/mg·dL-1 100.3 102.6 100.3
高密度脂蛋白胆固醇/mg·dL-1 49.5 48.0 49.5
转铁蛋白饱和度/% 21.4 27.6 21.4
铁蛋白/μg·L-1 216.5 223.7 216.5
超敏C反应蛋白>4.9 mg·L-1比例/% 11.0 21.3 11.0
), ArticleFig(id=1193928821817704977, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=null, figureFileBig=null, tableContent=
疗效指标 罗沙司他试验 恩那度司他试验 间接疗效 P值
罗沙司他 安慰剂 组间差值 恩那度司他 安慰剂 组间差值
匹配前
第7~9周平均Hb浓度与基线期的差值/g·dL-1 1.90 -0.30 2.20 1.60 -0.01 1.61 -0.59 <0.001
第7~9周平均Hb≥10.0 g·dL-1的受试者比例 67.00% 6.00% 61.00% 85.29% 30.00% 55.29% -5.71% 0.109
匹配后
第7~9周平均Hb浓度与基线期的差值/g·dL-1 1.90 -0.30 2.20 1.89 -0.29 2.18 -0.02 0.845
第7~9周平均Hb≥10.0 g·dL-1的受试者比例 67.00% 6.00% 61.00% 86.70% 13.20% 73.50% 12.50% <0.001
), ArticleFig(id=1193928821922562578, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=表5, caption=

恩那度司他和罗沙司他的疗效指标比较统计学结果

, figureFileSmall=null, figureFileBig=null, tableContent=
疗效指标 罗沙司他试验 恩那度司他试验 间接疗效 P值
罗沙司他 安慰剂 组间差值 恩那度司他 安慰剂 组间差值
匹配前
第7~9周平均Hb浓度与基线期的差值/g·dL-1 1.90 -0.30 2.20 1.60 -0.01 1.61 -0.59 <0.001
第7~9周平均Hb≥10.0 g·dL-1的受试者比例 67.00% 6.00% 61.00% 85.29% 30.00% 55.29% -5.71% 0.109
匹配后
第7~9周平均Hb浓度与基线期的差值/g·dL-1 1.90 -0.30 2.20 1.89 -0.29 2.18 -0.02 0.845
第7~9周平均Hb≥10.0 g·dL-1的受试者比例 67.00% 6.00% 61.00% 86.70% 13.20% 73.50% 12.50% <0.001
), ArticleFig(id=1193928821993865747, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=null, figureFileBig=null, tableContent=
结果指标 恩那度司他 罗沙司他
总成本/元 43 450 72 751
药品成本/元 30 692 57 690
疾病管理成本/元 10 138 12 191
输血治疗成本/元 458 743
不良事件治疗成本/元 2 163 2 127
总QALYs 3.48 3.40
增量成本/元 -29 301
增量QALYs 0.08
ICER/元(QALY) 绝对优势
), ArticleFig(id=1193928822056780308, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=表6, caption=

5年的研究时限内恩那度司他和于罗沙司他成本效用分析结果

, figureFileSmall=null, figureFileBig=null, tableContent=
结果指标 恩那度司他 罗沙司他
总成本/元 43 450 72 751
药品成本/元 30 692 57 690
疾病管理成本/元 10 138 12 191
输血治疗成本/元 458 743
不良事件治疗成本/元 2 163 2 127
总QALYs 3.48 3.40
增量成本/元 -29 301
增量QALYs 0.08
ICER/元(QALY) 绝对优势
), ArticleFig(id=1193928822153249301, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=EN, label=null, caption=null, figureFileSmall=null, figureFileBig=null, tableContent=
结果指标 恩那度司他 罗沙司他
研究时限为8年
总成本/元 63 779 106 727
总QALYs 5.12 4.99
增量成本/元 -42 947
增量QALYs 0.13
研究时限为10年
总成本/元 75 424 126 129
总QALYs 6.06 5.90
增量成本/元 -50 705
增量QALYs 0.16
STC比较证据
总成本/元 42 313 72 751
总QALYs 3.49 3.40
增量成本/元 -30 437
增量QALYs 0.09
), ArticleFig(id=1193928822228746774, tenantId=1146029695717560320, journalId=1190317699101192196, articleId=1193877802572279942, language=CN, label=表7, caption=

Markov模型的模拟时限情境分析结果

, figureFileSmall=null, figureFileBig=null, tableContent=
结果指标 恩那度司他 罗沙司他
研究时限为8年
总成本/元 63 779 106 727
总QALYs 5.12 4.99
增量成本/元 -42 947
增量QALYs 0.13
研究时限为10年
总成本/元 75 424 126 129
总QALYs 6.06 5.90
增量成本/元 -50 705
增量QALYs 0.16
STC比较证据
总成本/元 42 313 72 751
总QALYs 3.49 3.40
增量成本/元 -30 437
增量QALYs 0.09
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恩那度司他治疗非透析慢性肾脏病患者肾性贫血的疗效间接比较与成本-效用分析
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高金生 , 甘磊 , 刘莹 , 贺小宁 *
中国药学杂志 | 论著 2025,60(2): 194-200
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中国药学杂志 | 论著 2025, 60(2): 194-200
恩那度司他治疗非透析慢性肾脏病患者肾性贫血的疗效间接比较与成本-效用分析
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高金生, 甘磊, 刘莹, 贺小宁*
作者信息
  • 天津大学药物科学与技术学院, 天津 300072

    • 高金生,男,硕士研究生 研究方向:药物经济学

通讯作者:

*贺小宁,女,博士,副教授 研究方向:卫生经济学与药物经济学 Tel: (022) 87401830
Indirect Efficacy Comparison and Cost-utility Analysis of Enarodustat in the Treatment of Renal Anemia in Patients with Non-Dialysis Chronic Kidney Disease
Jinsheng GAO, Lei GAN, Ying LIU, Xiaoning HE*
Affiliations
  • School of Pharmaceutical Science and Technology, Tianjin University, Tianjin 300072, China
出版时间: 2025-01-22 doi: 10.11669/cpj.2025.02.013
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摘要
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目的 评价恩那度司他治疗非透析患者肾性贫血的疗效和经济性。方法 基于恩那度司他和罗沙司他各自的Ⅲ期临床试验开展了疗效的匹配调整间接比较,并将其结果用于恩那度司他治疗非透析患者肾性贫血的成本-效用分析。通过敏感性分析和情境分析,验证增量成本效果比的稳健性。结果 匹配调整试验人群的基线特征后,恩那度司他相比于罗沙司他能够显著提高第7~9周平均血红蛋白≥10.0 g·dL-1的受试者比例(73.5% vs 61.0%,MD=12.5%,P<0.001);成本-效用分析结果显示,与罗沙司他相比,恩那度司他增加患者质量调整生命年的同时节约了医疗成本,为绝对优势方案。结论 与罗沙司他相比,恩那度司他治疗非透析慢性肾脏病患者肾性贫血的疗效更好,且具有较好的经济性。

非透析  /  肾性贫血  /  恩那度司他  /  匹配调整间接比较  /  成本-效用分析

OBJECTIVE To evaluate the efficacy and cost-effectiveness of enarodustat in the treatment of renal anemia in non-dialysis patients. METHODS A matched-adjusted indirect comparison of the efficacy of enarodustat and roxadustat was conducted based on their respective phase Ⅲ clinical trials, and the results were used for cost-utility analysis of enarodustat in the treatment of renal anemia in non-dialysis patients. The robustness of the incremental cost-effectiveness ratio was verified through sensitivity analysis and scenario analysis. RESULTS After matching and adjusting the baseline characteristics of the trial population, it was shown that enarodustat was able to significantly increase the proportion of subjects with average hemoglobin ≥10.0 g·dL-1 at weeks 7-9 compared with roxadustat (73.5% vs 61.0%, MD=12.5%, P<0.001); the results of cost-utility analysis showed that compared with roxadustat, enarodustat increased patients' quality-adjusted life years and saved costs, making it an absolutely superior solution.CONCLUSION Compared with roxadustat, enarodustat is more effective and economical in treating renal anemia in patients with non-dialysis chronic kidney disease.

non-dialysis  /  renal anemia  /  enarodustat  /  matching-adjusted indirect comparison  /  cost-utility analysis
高金生, 甘磊, 刘莹, 贺小宁. 恩那度司他治疗非透析慢性肾脏病患者肾性贫血的疗效间接比较与成本-效用分析. 中国药学杂志, 2025 , 60 (2) : 194 -200 . DOI: 10.11669/cpj.2025.02.013
Jinsheng GAO, Lei GAN, Ying LIU, Xiaoning HE. Indirect Efficacy Comparison and Cost-utility Analysis of Enarodustat in the Treatment of Renal Anemia in Patients with Non-Dialysis Chronic Kidney Disease[J]. Chinese Pharmaceutical Journal, 2025 , 60 (2) : 194 -200 . DOI: 10.11669/cpj.2025.02.013
正文
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肾性贫血是慢性肾脏病(chronic kidney disease,CKD)的常见并发症,是指各种肾脏病致肾功能下降时,红细胞生成素(erythropoietin,EPO)生成减少及血浆中一些毒性物质干扰红细胞生成并缩短其寿命而导致的贫血[1]。CKD非透析患者总体贫血患病率约为28.5%~72.5%,肾性贫血会增加非透析患者心脑血管事件和全因死亡风险,严重影响非透析患者的运动功能和生活质量[2]。
由于铁剂、红细胞生成刺激剂(erythropoiesis-stimulating agents,ESAs)等传统方案治疗贫血时存在诱导患者心血管并发症发生、可能导致患者机体铁超载等问题,低氧诱导因子脯氨酰羟化酶抑制剂(hypoxia inducible factor prolyl-hydroxylase inhibitors,HIF-PHI)类药物成为肾性贫血患者治疗的新选择。我国首个上市并纳入医保的HIF-PHI药物为罗沙司他,其在治疗肾性贫血方面具有很好的临床疗效证据与真实世界证据[3],但也有研究表明它可能会增加严重血栓事件的发生风险[4]。恩那度司他是2023年在中国上市的第2个HIF-PHI药物,适应证为非透析的成人CKD患者的贫血治疗;同年12月,被纳入国家医保药品目录。相较于罗沙司他,恩那度司他可以高选择性抑制脯氨酰羟化酶(prolyl hydroxylase,PHD),可以更好地促进EPO生成、改善铁代谢。该药的中国Ⅲ期临床试验(NCT06016036)显示,恩那度司他可稳定改善患者的贫血状态,而患者发生血栓事件的风险更低。目前缺乏恩那度司他与罗沙司他头对头疗效比较,本研究旨在比较恩那度司他与罗沙司他的疗效与经济性,通过开展疗效的匹配调整间接比较,并将其结果用于成本-效用分析,评估两药品的疗效指标与增量成本效果比。
1 资料与方法
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1.1 疗效间接比较
1.1.1 临床试验检索与数据提取
恩那度司他的患者个体数据(individual patient data,IPD)来自一项随机、安慰剂对照、双盲、开放标签、多中心的Ⅲ期临床试验(NCT06016036),旨在评估恩那度司他在中国CKD非透析患者人群中用于治疗肾性贫血的疗效。该临床试验的纳入标准和排除标准如下。主要纳入标准有:①估算肾小球滤过率(estimated glomerular filtration rate,eGFR)数值<60 mL·min·1.73 m-2(CKD3、4或5期)的未接受透析的确诊CKD贫血受试者;②在随机前至少8周未接受过ESAs治疗,且筛选期间最近2次(至少间隔6 d)的中心实验室血红蛋白(hemoglobin,Hb)值为≥8 g·dL-1且≤10.5 g·dL-1,2次访视绝对值的变化值≤1 g·dL-1;③研究者判断在整个试验过程中不需要透析或肾移植的替代治疗等。主要排除标准有:①筛选前12周内发生急性肾损伤;②筛选前3个月内进行过输血,或输注红细胞;③严重活动性感染(如活动性结核、真菌感染等),系统性血液疾病(例如骨髓异常增生综合征、再生障碍性贫血),或溶血性贫血,或失血性贫血;④可能影响红细胞生成的肾小球肾炎以外的慢性炎症性疾病,如系统性红斑狼疮、风湿性关节炎、乳糜泻等;⑤随机试验前8周内接受过HIF-PHI类药物治疗等。为获取对照方案(罗沙司他)的临床疗效数据,本研究进行了系统的文献检索以筛选出所有可获得的罗沙司他临床试验的汇总数据(aggregate data,AgD)。以“罗沙司他” “roxadustat”为关键词在中国知网、万方等中文数据库和PubMed、Embase等英文数据库进行检索,文献去重后检索文献数为600篇,经文献纳入与排除的标准筛选后,最终纳入6篇文献。本研究仅纳入研究人群为中国人群、安慰剂对照的罗沙司他试验,即与恩那度司他试验研究设计相似的试验。6个罗沙司他试验的基本信息,见表1。因此,本研究将FGCL-4592-808试验作为罗沙司他的数据来源。
1.1.2 研究指标与比较方法
比较恩那度司他和罗沙司他的疗效前,需对两临床试验疗效相关数据进行提取和比较,包括比较纳入试验中的疗效指标定义是否相同,是否纳入临床试验的主要疗效指标、指标是否可以用于成本-效用分析等。因此,间接比较研究纳入的疗效指标,分别是“第7~9周平均Hb浓度与基线期的差值”和“第7~9周平均Hb≥10.0 g·dL-1的患者比例”。
由于两试验有共同对照组(安慰剂组),本研究使用了有锚定的匹配调整间接比较(matching-adjusted indirect comparisons,MAIC)方法来比较恩那度司他与罗沙司他的疗效。MAIC方法是一种基于加权匹配的间接比较方法,在方法学上有较好的性能[5-6]。该方法基于逆概率倾向得分加权(inverse propensity score weighting,IPW)的原理,对拥有IPD的临床试验的每位患者赋予不同的权重比例,使得加权调整后IPD试验的患者基线特征与拥有AgD的试验差异无统计学意义[7]。在本研究中,恩那度司他试验的IPD被进行如上加权计算,从而使得恩那度司他试验中加权后的患者基线特征与罗沙司他试验的患者特征完全匹配,进而可以重新计算恩那度司他的疗效,并与罗沙司他比较。
1.2 成本-效用分析
1.2.1 模型构建
本研究的研究角度为中国医疗卫生体系角度,Markov模型的人群特征参数与上述疗效间接比较研究中的罗沙司他试验人群特征相同。本研究在设置循环周期和模拟时限时,参考了已发表同类文献[8-10],发现非透析患者一般会在5年后接受透析治疗。因此,Markov模型的循环周期为28 d,模拟时限为5年,该时间更符合临床实际。根据《中国肾性贫血诊治临床实践指南》与已发表文献,本研究将患者Hb水平的治疗靶目标设置为10 g·dL-1。本研究将患者Hb水平与靶目标的相对关系,作为疾病转归的状态,且Hb≥10 g·dL-1状态优于Hb<10 g·dL-1状态。因此,本研究的Markov模型设置了3个互斥的健康状态,即:Hb<10 g·dL-1状态、Hb≥10 g·dL-1状态和死亡状态,见图1。
本研究还采用单因素敏感性分析和概率敏感性分析验证Markov模型结果的稳健性。本研究同时开展了以下情境分析:①根据模拟治疗比较方法对恩那度司他与罗沙司他的疗效间接比较,并将其得到的比较结果也用于药物经济性评价中;②假设Markov模型的模拟时限为8和10年。
1.2.2 模型参数
患者刚进入模型时一部分患者处于Hb<10 g·dL-1状态,一部分患者处于Hb≥10 g·dL-1状态,之后患者将停留在这2种状态,或者在这两个状态间相互转移,或发生死亡事件进入死亡状态。因此,Markov模型需要6种转移概率。此外,每周期的患者均有可能发生不良事件。本研究中Markov模型需要的状态转移概率、不良事件发生率及其来源见表2。
由于模型的循环周期(4周)与某些转移概率来源所对应的研究时间不符,还需要把这些概率换算为4周的概率。转移概率的计算[15]公式1~2如下:
r=-[Ln(1-P1)]/t1
P2=1- e - r t 2
其中,P1为事件发生率,t1为事件发生的持续时间,t2为本模型周期,P2为状态转移概率。
本研究的研究角度为中国医疗卫生体系角度,因此,模型仅纳入直接医疗成本,具体包括药品成本、疾病管理成本、输血成本、不良事件治疗成本等,见表3。2个药品的价格均为医保谈判价格,其他医疗资源使用和费用数据来源于已发表文献与地方政府文件。本研究以质量调整生命年(quality adjusted life years,QALYs)作为效用指标计算健康结果。健康状态效用值和各不良事件的负效用值见表3。在参数范围设置方面,对于成本参数,2个药品的价格根据实际情况设置,即上限为维持原数值不变,下限为原数值基础上降低20%,其余成本参数均选择常用的±20%作为上下限;对于效用参数,选择±5%作为上下限。本研究中成本及效用的贴现率均采用《中国药物经济学评价指南(2020)》[16]推荐的5%。
2 结果
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2.1 疗效间接比较
2.1.1 人群基线特征匹配结果
不同的患者基线特征对结果的影响是存在差异的。因此在进行MAIC前,应先分析并识别可直接作用于治疗结果或可能影响疗效的患者基线特征。本部分根据MAIC方法原理相关综述和临床专家意见咨询将关键基线特征均纳入匹配,即年龄、性别、Hb、eGFR、收缩压、舒张压、总胆固醇、低密度脂蛋白胆固醇、高密度脂蛋白胆固醇、转铁蛋白饱和度、铁蛋白和超敏C反应蛋白(high sensitivity C-reactive protein,hs-CRP)指数共12个基线特征,采用R语言软件根据逆概率倾向得分加权原理匹配了拟比较的2个临床试验中的所有可以匹配的关键基线特征,且没有对基线特征进行删除。匹配前,两试验的基线特征存在着一定差异,而在对基线关键变量进行匹配调整后,不平衡的基线特征得到了很好的调整,2个试验人群的匹配特征完全相同(表4)。此外,匹配后恩那度司他试验的有效样本量(effective sample size,ESS)下降至51人,相比于原试验样本量减少了67.1%,表明2个试验的患者人群在匹配前确有较大的差异。
2.1.2 疗效比较结果
匹配前,与安慰剂相比,恩那度司他在“第7~9周平均Hb≥10.0 g·dL-1的患者比例”(指标2)上与罗沙司他无显著差异(55.29% vs 61.00%,P=0.109);而在“第7~9周平均Hb浓度与基线期的差值”(指标1)低于罗沙司他0.59 g·dL-1(1.61 g·dL-1vs 2.20 g·dL-1,P<0.001)。匹配后,与安慰剂相比,在“第7~9周平均Hb≥10.0 g·dL-1的受试者比例”上,恩那度司他高于罗沙司他12.5%,且有统计学意义(73.50% vs 61.00%,P<0.001);而在“第7~9周平均Hb浓度与基线期的差值”上,恩那度司他与罗沙司他无显著差异(2.18 g·dL-1vs 2.20 g·dL-1,P=0.845)(表5)。在提升血红蛋白值效能相同下的情况,恩那度司他相比于罗沙司他能显著提升患者血红蛋白达标率。
2.2 成本-效用分析
2.2.1 基础分析结果
在5年的研究时限内,恩那度司他相比于罗沙司他的增量QALYs为0.08(3.48 vs 3.40),增量成本为-29 301元(43 450元vs 72 751元),健康产出更高的同时总成本更低,恩那度司他具有绝对优势(表6)。
2.2.2 敏感性分析结果
单因素敏感性分析的龙卷风图显示,Hb≥10 g·dL-1状态效用值、Hb<10 g·dL-1状态效用值、罗沙司他的周给药剂量等参数是对ICER影响较大的参数(图2)。概率敏感性分析表明,恩那度司他具有成本-效果的概率为100%,恩那度司他始终为绝对优势方案。
2.2.3 情境分析结果
根据慢性肾脏病患者贫血的疾病转归的临床实际,非透析患者将会在平均5~10年后接受透析治疗。本研究对Markov模型的模拟时限进行了情境分析,以检验其对ICER的影响(表7)。当模拟时限分别为8年和10年时,恩那度司他均为绝对优势方案。本研究通过对研究时限开展情境分析,发现随着研究时限的增长,患者的增量QALY随之增加,说明恩那度司他对患者有更好的长期获益。本研究还对临床参数开展了情境分析,根据模拟治疗比较(simulated treatment comparison,STC)方法对两药品的疗效进行间接比较,并将其结果也用于成本效用分析中。当恩那度司他与罗沙司他疗效比较差值(12.5%)替换为STC方法得到的差值(10.4%)时,恩那度司他仍为绝对优势方案。综上所述,主分析的ICER是稳健的。
3 讨论
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肾性贫血会大大降低CKD患者的生活质量,增加额外的就医成本。近年来,HIF-PHI类药品作为一种新一代治疗肾性贫血的药物,克服了传统ESAs类药品的缺点。目前已有6种在研或上市的HIF-PHI类药物,但目前我国尚无比较不同HIF-PHI类药品疗效或安全性的研究。本研究的间接比较结果显示,恩那度司他与罗沙司他在“第7~9周平均Hb浓度与基线期的差值”上不存在显著差异,但是在“第7~9周平均Hb≥10.0 g·dL-1的患者比例”上优于罗沙司他,说明恩那度司他在升高Hb上与罗沙司他具有相同的效能,但是其升高速度较为温和,且能有更高比例的患者保持在非贫血状态。这可能是,由于恩那度司他高选择性地抑制PHD1,精准稳定低氧诱导因子(hypoxia-inducible factor-2,HIF-2),使患者的Hb变化更小,也能使更多患者贫血得到纠正。此外,两项分别评估了恩那度司他与罗沙司他长期疗效与安全性的日本临床试验[23-24]也证明了,恩那度司他比罗沙司他可以使患者维持在非贫血状态的比例更高(高约20.0%)。
本研究比较了不同HIF-PHI类药物治疗肾性贫血的经济性,而以往肾性贫血的成本-效用分析研究多是分析ESAs类药物治疗肾性贫血的经济性。同时,本研究还考察了模型的模拟时限与不同方法得到的疗效比较结果对增量成本效果比(incremental cost effectiveness ratio,ICER)的影响。成本-效用分析显示,与罗沙司他相比,恩那度司他使患者增加了0.08个QALYs,而恩那度司他在使患者Hb<10 g·dL-1状态至Hb≥10 g·dL-1状态的比例与使患者维持在Hb≥10 g·dL-1状态的比例均高于罗沙司他,这表明使用恩那度司他的患者有更好的健康获益。在成本方面,恩那度司他的药品成本更低,而药品成本在总治疗成本中占很大的比例,因此,恩那度司他的治疗总成本也更低。此外,在挽救治疗(输血)成本和不良事件成本方面,恩那度司他也能够降低患者此类经济负担。考虑到CKD患者一般会在5年进展至终末期肾病(接受透析治疗),以往研究对研究时限设置主要是5年,而循环周期通常设置为1或3个月[8-10]。本研究也设置了5年的研究时限,并对研究时限开展了情境分析,发现当用药时间更长时,患者使用恩那度司他在长期上会有更高的健康获益。本研究根据STC方法也对两药品的疗效进行了间接比较,同样得到恩那度司他优于罗沙司他,将该疗效数据用于成本-效用分析后,恩那度司他仍为绝对优势方案。
本研究也存在一定的局限性。①在成本效用分析中对Markov模型的模拟时限开展了情境分析,但是未充分考虑恩那度司他在提高患者生命质量的长期优势,可能会对恩那度司他在增加患者的健康获益上有所低估;②由于目前恩那度司他在中国获批的适应证仅为非透析的成人慢性肾脏病(CKD)患者的贫血治疗,在成本效用分析中只模拟了非透析患者肾性贫血的疾病转归,没有在模型中考虑透析治疗的患者;③有研究表明罗沙司他会增加患者主要心血管不良事件(major adverse cardiovascular events,MACE)以及血栓的发生风险[4],但是在疗效间接比较和成本效用分析中没有考虑此类事件,可能会对恩那度司他在安全性方面的优势有所低估。
综上所述,相较于罗沙司他,恩那度司他治疗CKD非透析患者肾性贫血能有效提高患者血红蛋白水平,且不良反应更少,可有效提高患者贫血的治疗效果,并减轻其治疗的经济负担。
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doi: 10.11669/cpj.2025.02.013
  • 接收时间：2024-04-02
  • 首发时间：2025-11-08
  • 出版时间：2025-01-22
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  • 收稿日期：2024-04-02
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    天津大学药物科学与技术学院, 天津 300072

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*贺小宁,女,博士,副教授 研究方向:卫生经济学与药物经济学 Tel: (022) 87401830
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2种不同金属材料的力学参数
科
Family		 属数
Number of
genus		 种数
Number of
species		 占总种数比例
Percentage of
total species (%)		 属
Genus		 种数
Number of
species		 占总种数比例
Percentage of total
species (%)
鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
红菇属 Russula 17 8.13
栓菌属 Trametes 5 2.39
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