罗沙司他治疗慢性肾脏疾病患者肾性贫血的快速卫生技术评估

Rapid health technology assessment of roxadustat for renal anemia in patients with chronic kidney disease

目的:通过快速卫生技术评估(HTA)的方法,评价罗沙司他治疗慢性肾脏疾病(CKD)患者肾性贫血的有效性、安全性和经济性,为临床提供高效、便捷的循证依据。方法:检索PubMed、Cochrane Library、万方和中国知网等数据库。由2位研究者根据纳入与排除标准独立筛选文献、提取资料和评价质量后,对结果进行定性分析。结果:纳入系统评价/Meta分析11篇以及药物经济学研究1篇。结果显示罗沙司他与安慰剂或促红细胞生成素(EPO)相比,能够显著增加CKD伴肾性贫血患者的血红蛋白水平,提高血红蛋白的达标率,升高血清铁水平、转铁蛋白、总铁结合力水平,同时能够下调铁调素(P<0.05)。在非透析患者中与安慰剂相比能够显著提高铁蛋白和转铁蛋白饱和度(P<0.05);在透析患者中与EPO相比铁蛋白和转铁蛋白饱和度水平变化无显著差异(P>0.05)。罗沙司他与安慰剂之间安全性无显著差异;总体不良事件与EPO之间无显著差异,但严重不良事件发生率稍高于EPO(P<0.05)。药物经济学研究表明,与安慰剂相比,罗沙司他能够显著延长非透析患者的质量调整生命年(QALY),增量成本效果比为25563美元·QALY^(-1),低于3倍的人均GDP。结论:罗沙司他在慢性CKD伴肾性贫血患者中具有较好的有效性和安全性,与安慰剂相比显示出较好的经济性,未来有必要开展与EPO之间的经济学评估。

Objective:To evaluate the efficacy,safety and economy of roxadustat for renal anemia in patients with chronic kidney disease(CKD)by rapid health technology assessment(HTA),and provide efficient and convenient evidence-based evidence for clinic.Methods:PubMed,Cochrane Library,Wanfang,CNKI and other databases were searched,two researchers independently screened the literature,extracted data and evaluated the quality according to the inclusion and exclusion criteria,and conducted qualitative analysis of the results.Results:A total of 11 systematic reviews/meta-analysis and one pharmacoeconomic study were included.Results showed that roxadustat significantly increased the level of hemoglobin and the compliance rate of hemoglobin in patients with CKD and renal anemia and downregulated iron modulator compared with placebo or erythropoietin(EPO),P<0.05.In non-dialysis patients,roxadustat significantly increased ferritin and transferrin saturation compared with placebo(P<0.05).And there were no significant differences in ferritin and transferrin saturation levels in dialysis patients between roxadustat and EPO(P>0.05).In terms of safety,there was no significant difference between roxadustat and placebo.And the total adverse events of roxadustat were also similar to EPO while the severe adverse events induced by roxadustat were slightly higher than those of EPO(P<0.05).Pharmacoeconomic study showed that compared with placebo,roxadustat significantly extended quality-adjusted life year(QALY)in patients with CKD.Patients with an incremental cost-effectiveness ratio of 25563 dollars·QALY^(-1) which was less than 3 times per capita GDP.Conclusion:Roxadustat showed good efficacy and safety in CKD patients with renal anemia,and showed better economy compared with placebo.It was necessary to carry out economic evaluation between roxadustat and EPO in the future.