目的评价司来帕格治疗肺动脉高压(PAH)的有效性、安全性和经济性。方法计算机检索PubMed、Embase、Web of Science、Cochrane Library、Epistemonikos、SinoMed、中国知网、维普网、万方数据、中国生物医学文献数据库、国内外卫生技术...目的评价司来帕格治疗肺动脉高压(PAH)的有效性、安全性和经济性。方法计算机检索PubMed、Embase、Web of Science、Cochrane Library、Epistemonikos、SinoMed、中国知网、维普网、万方数据、中国生物医学文献数据库、国内外卫生技术评估机构官方网站及相关数据库,经文献筛选、资料提取、质量评价后,对纳入研究的结果进行描述性分析。结果与结论共纳入7篇文献,包括6篇Meta分析和1篇药物经济学研究。有效性方面,司来帕格可改善患者运动耐量、血流动力学指标,并可降低临床恶化事件发生率和住院治疗率,但静脉/皮下途径的前列环素类似物在改善WHO心功能分级和降低全因死亡率方面的效果更佳(P<0.05)。司来帕格与其他靶向药物对各项结局指标影响的差异均无统计学意义(P>0.05)。安全性方面,司来帕格可增加一般不良反应事件的发生率和因无法耐受不良反应停药的发生率(P<0.05),而与各类靶向药物比较的差异均无统计学意义(P>0.05)。经济性方面,基于加拿大卫生体系背景,司来帕格治疗PAH不具有经济性;目前缺少基于我国国情的经济学研究。尚需进一步开展司来帕格和前列环素类药物在真实世界中疗效和经济性的对比研究。展开更多
Purpose:This study examined the efficacy and safety of selexipag in treating pulmonary arterial hypertension(PAH)associated with congenital heart disease(CHD).Materials and Methods:We conducted a retrospective study o...Purpose:This study examined the efficacy and safety of selexipag in treating pulmonary arterial hypertension(PAH)associated with congenital heart disease(CHD).Materials and Methods:We conducted a retrospective study of patients with CHD-associated PAH,treated with selexipag since December 2017.Thirteen adult patients(mean age,45.4 years;women,77%)were treated with selexipag as add-on therapy.Baseline characteristics,World Health Organization functional class,6-minute walking distance(6MWD)test results,N-terminal pro-B-type natriuretic peptide levels,echocardiographic data,and incidence of side effects were assessed.Results:The majority of patients(12/13,92.3%)experienced more than one treatment-associated complication;one patient dropped out of the study due to intolerable myalgia.The results of 6MWD test(from 299.2±56.2 m to 363.8±86.5 m,p=0.039)and tricuspid regurgitation(TR)pressure gradient(from 84.7±20.5 mmHg to 61.6±24.0 mmHg,p=0.018)improved and remained improved after selexipag treatment in 12 patients.Based on the results of a non-invasive risk assessment,8(66.7%)patients showed improvement,3(25.0%)showed no interval change,and the status of one patient(8.3%)deteriorated.Moreover,compared to patients treated with a low dosage,patients treated with a medium-to-high dosage showed a greater increase in 6MWD results(88.3±26.4 m vs.55.3±27.6 m,p=0.043)and a greater reduction in the TR pressure gradient(-33.7±10.9 mmHg vs.-12.5±12.0 mmHg,p=0.015).Conclusion:Selexipag is an efficient pulmonary vasodilator as add-on therapy in treating CHD-associated PAH.展开更多
文摘目的评价司来帕格治疗肺动脉高压(PAH)的有效性、安全性和经济性。方法计算机检索PubMed、Embase、Web of Science、Cochrane Library、Epistemonikos、SinoMed、中国知网、维普网、万方数据、中国生物医学文献数据库、国内外卫生技术评估机构官方网站及相关数据库,经文献筛选、资料提取、质量评价后,对纳入研究的结果进行描述性分析。结果与结论共纳入7篇文献,包括6篇Meta分析和1篇药物经济学研究。有效性方面,司来帕格可改善患者运动耐量、血流动力学指标,并可降低临床恶化事件发生率和住院治疗率,但静脉/皮下途径的前列环素类似物在改善WHO心功能分级和降低全因死亡率方面的效果更佳(P<0.05)。司来帕格与其他靶向药物对各项结局指标影响的差异均无统计学意义(P>0.05)。安全性方面,司来帕格可增加一般不良反应事件的发生率和因无法耐受不良反应停药的发生率(P<0.05),而与各类靶向药物比较的差异均无统计学意义(P>0.05)。经济性方面,基于加拿大卫生体系背景,司来帕格治疗PAH不具有经济性;目前缺少基于我国国情的经济学研究。尚需进一步开展司来帕格和前列环素类药物在真实世界中疗效和经济性的对比研究。
文摘Purpose:This study examined the efficacy and safety of selexipag in treating pulmonary arterial hypertension(PAH)associated with congenital heart disease(CHD).Materials and Methods:We conducted a retrospective study of patients with CHD-associated PAH,treated with selexipag since December 2017.Thirteen adult patients(mean age,45.4 years;women,77%)were treated with selexipag as add-on therapy.Baseline characteristics,World Health Organization functional class,6-minute walking distance(6MWD)test results,N-terminal pro-B-type natriuretic peptide levels,echocardiographic data,and incidence of side effects were assessed.Results:The majority of patients(12/13,92.3%)experienced more than one treatment-associated complication;one patient dropped out of the study due to intolerable myalgia.The results of 6MWD test(from 299.2±56.2 m to 363.8±86.5 m,p=0.039)and tricuspid regurgitation(TR)pressure gradient(from 84.7±20.5 mmHg to 61.6±24.0 mmHg,p=0.018)improved and remained improved after selexipag treatment in 12 patients.Based on the results of a non-invasive risk assessment,8(66.7%)patients showed improvement,3(25.0%)showed no interval change,and the status of one patient(8.3%)deteriorated.Moreover,compared to patients treated with a low dosage,patients treated with a medium-to-high dosage showed a greater increase in 6MWD results(88.3±26.4 m vs.55.3±27.6 m,p=0.043)and a greater reduction in the TR pressure gradient(-33.7±10.9 mmHg vs.-12.5±12.0 mmHg,p=0.015).Conclusion:Selexipag is an efficient pulmonary vasodilator as add-on therapy in treating CHD-associated PAH.
