利奥西呱治疗肺高血压有效性、安全性及经济性的快速卫生技术评估

Rapid health technology assessment of the efficacy,safety and economy of riociguat in the treatment of pulmonary hypertension

目的:快速评价利奥西呱治疗肺高血压(PH)的有效性、安全性和经济性,为临床治疗方案的选择提供循证参考。方法:计算机检索主要中英文数据库及各大卫生技术评估网站。研究人员独立筛选文献、提取资料。进行质量评价后,对提取的结果进行分类评价和描述性汇总分析。结果:共纳入文献12篇,其中HTA报告2篇、系统评价/Meta分析7篇、药物经济学研究4篇。有效性分析显示:对于肺动脉高压(PAH)患者,相比于安慰剂,利奥西呱可显著增加6分钟步行距离(6-MWD)、心脏指数(CI),降低Borg呼吸困难指数(BDI)、平均肺动脉压(mPAP)、肺动脉阻力(PVR)、临床恶化率、血清氨基末端B型脑利钠肽前体(NT-proBNP)、肺高血压评分(LPH评分),差异有统计学意义。对于慢性血栓栓塞性肺高血压(CTEPH)患者,相比于安慰剂,利奥西呱可显著延长6-MWD,降低mPAP、PVR、BDI、NT-proBNP,差异有统计学意义。对于左心疾病相关性肺高血压(PH-LHD)患者,相比于安慰剂,利奥西呱仅对PVR、CI有显著影响。安全性分析显示:与安慰剂相比,利奥西呱的严重不良事件无显著增加。经济性分析显示:国外研究表明利奥西呱具有较高的增量成本效用比。结论:利奥西呱对PAH、CTEPH患者具有良好的有效性和安全性,对PH-LHD的有效性尚需进一步研究。亟需开展其国内药物经济学研究。

OBJECTIVE To rapidly evaluate the effectiveness,safety and economics of riociguat in the treatment of pulmonary hypertension(PH)and provide evidence-based reference for selecting clinical treatment options.METHODS Major Chinese and English databases and some health technology assessment websites were searched.The researchers independently screened and extracted the relevant data.After a quality evaluation of the included literature,the extracted results were analyzed by descriptive summary.RESULTS There were 12 references,including 2 health technology assessment(HTA)reports,7 systematic evaluation/Meta-analysis and 4 pharmacoeconomic studies.Effectiveness analysis indicated that,for pulmonary arterial hypertension(PAH)patients,as compared to placebo,riociguat markedly extended 6-minute walking distance(6-MWD),cardiac index(CI),reduced Borg dyspnea index(BDI),mean pulmonary artery pressure(mPAP),pulmonary vascular resistance(PVR),clinical deterioration rate,serum amino terminal B type brain natriuretic peptide precursor(NT-proBNP)and pulmonary hypertension score.The differences were statistically significant.In patients with chronic thromboembolic pulmonary hypertension(CTEPH),6-MWD,PAP,PVR,BDI and NT-proBNP were markedly prolonged as compared with placebo.Patients with left heart disease-associated pulmonary hypertension(PH-LHD)had only significant effects on PVR/CI as compared to placebo.Security analysis revealed no significant increase in severe adverse events(SAEs)as compared with placebo.And economic analysis of foreign studies indicated that riociguat had a higher incremental cost-utility ratio.CONCLUSION Riociguat has excellent efficacy and safety for PAH/CTEPH patients.However,its effectiveness for PH-LHD requires further studies.And domestic pharmacoeconomic researches are urgently needed.