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曲前列尼尔治疗肺动脉高压有效性、安全性和经济性的快速卫生技术评估
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| 篇名： | 曲前列尼尔治疗肺动脉高压有效性、安全性和经济性的快速卫生技术评估 |
| TITLE： | Effectiveness，Safety and Economy of Treprostinil in the Treatment of Pulmonary Arterial Hypertension ：A Rapid Health Technology Assessment |
| 摘要： | 目的：快速评价曲前列尼尔治疗肺动脉高压（PAH）的有效性、安全性和经济性，为临床药物选择和决策提供循证参考。方法：系统检索PubMed、Embase、WebofScience、Cochrane图书馆、Epistemonikos、英国约克大学卫生技术评估（HTA）数据库、中国知网和万方数据等中英文数据库，纳入曲前列尼尔对比安慰剂或其他药物治疗PAH的HTA报告、系统评价/Meta分析和药物经济研究，检索时限均为建库起至2020年5月1日。分别采用HTAchecklist、AMSTA、卫生经济学评价报告标准共识量表评价纳入HTA、系统评价/Meta分析、药物经济学研究的文献质量，并进行描述性汇总分析。结果：共纳入文献18篇，其中HTA报告1篇、系统评价/Meta分析12篇、药物经济学研究5篇。有效性分析结果显示，相比于安慰剂，曲前列尼尔可显著延长PAH患者的6分钟步行距离（6-MWD）、降低其Borg呼吸困难评分（P＜0.05），而对死亡率、临床恶化率、世界卫生组织（WHO）功能分级、入院治疗率和平均肺动脉压、肺血管阻力、心脏指数和平均右动脉压等指标无显著影响（P＞0.05）；此外，与安慰剂联合内皮素受体拮抗剂和/或磷酸二酯酶抑制剂比较，曲前列尼尔口服给药联合内皮素受体拮抗剂和/或磷酸二酯酶抑制剂能显著延长患者的6-MWD；与利奥西呱比较，曲前列尼尔可显著降低患者Borg呼吸困难评分。安全性分析结果表明，虽然接受曲前列尼尔治疗的患者因不能耐受不良反应（ADR）而停药的发生率有所增加（P＜0.05），但严重不良事件（SAEs）的发生率与安慰剂和其他治疗药物比较均无显著性差异（P＞0.05），且经吸入或静脉注射途径给药时患者的耐受性更好。经济性分析结果表明，虽然不同国家不同支付方的意愿支付阈值不同，但曲前列尼尔的增量成本-效果比高于意愿支付阈值。结论：曲前列尼尔治疗PAH具有良好的有效性，患者虽可能因不能耐受ADR而停药，但不会增加SAEs的发生风险；该药虽然价格偏高，但仍是临床治疗进展期或预后不良PAH患者的重要备选方案。 |
| ABSTRACT： | OBJECTIVE：To provide evidence-based reference for c linical drug selection and decision by rapidly evaluating the effectiveness，safety and economy of treprostinil in the treatment of pulmonary arterial hypertension （PAH）. METHODS ：Retrieved from Chinese and English database such as PubMed ，Embase，Web of Science ，the Cochrane Library ，Epistemonikos，HTA database（University of York ），CNKI and Wanfang databases ，included the health technology assessment （HTA）report，systematic/ Meta-analysis and pharmacoeconomic evaluation of treprostinil compared with placebo or other drugs in the treatment of PAH. The search time limit is from the construction of the database to May 1st，2020. HTA checklist ，AMSTA and CHEERS were applied to evaluate the quali ty of the literatures about HTA ，systematic review/Meta-analysis analysis a nd pharmacoecono mic evaluation and the inclusion studies was analyazed by descriptive summary. RESULTS：A total of 18 literatures were included ，involving 1 HTA report ，12 systematic review/Meta-analysis ，5 pharma- coeconomic studies. The analysis results of effectiven- ess mail： showed that compared with placebo ，treprostinil could signifi- cantly increase 6-MWD while decrease Borg dyspnea score of PAH p atients（P＜0.05），but had no significant effect on mortality，the rate of clinical deterioration ，WHO functional grading ，the rate of hospitalization ，mPAP，PVR，cardiac index and mRAP（P＞0.05）. In addition ，compared with placebo combined with endothelin receptor antagonist and/or phosphodiesterase inhibitors，oral administration of treprostinil combined with endothelin receptor antagonist and/or phosphodiesterase inhibitors could extend 6-MWD significantly. Compared with riociguat ，treprostinil could significantly reduce Borg dyspnea score of patients. The analysis results of safety displayed that ，although the incidence of drug withdrawal due to can ’t tolerate ADR increased in patients receiving treprostinil ，there was no significant difference in the incidence of serious adverse events compared with placebo or other treatments，and it was better tolerated when administered by inhalation or intravenous injection. The analysis results of pharmacoeconomic studies showed that ICER of treprostinil was higher than the willingness payment threshold ，although the willingness payment threshold was different in different countries and different payers. CONCLUSIONS ：Treprostinil treatment is effective for PAH. Patients may stop taking it due to can ’t tolerate ADR ，but the risk of serious adverse events are not increase. Although the price is high ，it is still an important alternative for PAH patients with clinical progress or poor prognosis. |
| 期刊： | 2020年第31卷第14期 |
| 作者： | 肖晓，周婧，杨敏，赖伟华，钟诗龙，曾英彤 |
| AUTHORS： | XIAO Xiao，ZHOU Jing，YANG Min，LAI Weihua ，ZHONG Shilong ，ZENG Yingtong |
| 关键字： | 曲前列尼尔；有效性；安全性；经济性；快速卫生技术评估 |
| KEYWORDS： | Treprostinil；Effectiveness；Safety；Economy；Rapid healt h technology assessment |
| 阅读数： | 945 次 |
| 本月下载数： | 13 次 |
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