经导管植入式主动脉人工心脏瓣膜临床试验初步建议

中国药物警戒 ›› 2014, Vol. 11 ›› Issue (10): 628-631.

经导管植入式主动脉人工心脏瓣膜临床试验初步建议

程茂波, 史新立, 苗晶晶, 刘威, 聂飞龙

国家食品药品监督管理总局医疗器械技术审评中心,北京 100044

收稿日期:2014-09-09 修回日期:2016-02-03 出版日期:2014-10-08 发布日期:2016-03-02
作者简介:程茂波,男,博士,助理研究员,无源植入性医疗器械产品技术审评。

Considerations on Clinical Study of Transcatheter Aortic Valve Implantation

CHENG Mao-bo, SHI Xin-li, MIAO Jing-jing, LIU Wei, NIE Fei-long

Center for Medical Device Evaluation, CFDA, Beijing 100044, China

Received:2014-09-09 Revised:2016-02-03 Online:2014-10-08 Published:2016-03-02

摘要/Abstract

摘要： 用于治疗有症状的重度主动脉瓣狭窄的经导管植入式主动脉人工心脏瓣膜的研发日益增多,但并未有规范性临床试验指导意见,缺少安全性和有效性评价的统一原则,为了更好地引导研究者完成上市前临床试验研究,通过结合该类产品已完成的临床试验研究情况、心脏病治疗指南及国内心血管内科、心血管外科和临床统计学专家的意见,总结了目前对于该类产品临床试验的初步建议。在中国境内开展临床试验时,建议首先完成可行性研究;确证性研究按照产品应用人群风险收益分析,分别对开展安全性和有效性评价,建议优先选择不适合常规外科手术有症状的重度主动脉瓣狭窄患者作为受试人群。

关键词: 经导管植入式主动脉人工心脏瓣膜, 临床试验, 可行性研究, 确证性研究

Abstract: Transcatheter aortic valve implantation for the patients suffering from severe aortic stenosis has emerged as a promising technique alternative to conventional surgical aortic valve replacement, the guidances of clinical study to evaluate the safety and efficacy of the products have not been drafted as so far. The recommendations which were drafted according to the existing clinical data and guideline for valvular heart disease and expert advice were introduced in the article in order to provide a guidance to manufacturers. Feasibility study to see if a larger pivotal study is practical and to refine the study protocol for the pivotal study was recommended firstly, the hypothesis of pivotal study was built in accordance with benefit-risk analysis, the aortic stenosis patients who cannot undergo surgery was preferentially treated in clinical trial.

Key words: transcatheter aortic valve implantation, clinical trial, feasibility study, pivotal study

中图分类号:

R197.39

程茂波, 史新立, 苗晶晶, 刘威, 聂飞龙. 经导管植入式主动脉人工心脏瓣膜临床试验初步建议[J]. 中国药物警戒, 2014, 11(10): 628-631.

CHENG Mao-bo, SHI Xin-li, MIAO Jing-jing, LIU Wei, NIE Fei-long. Considerations on Clinical Study of Transcatheter Aortic Valve Implantation[J]. Chinese Journal of Pharmacovigilance, 2014, 11(10): 628-631.

参考文献

[1] Leon M B, Smith C R, Mack M, et al. Transcatheter aortic-valve implantation for aortic stenosis in patients who cannot undergo surgery[J]. N Engl J Med, 2010, 363(17): 1597-1607.
[2] Andersen H R, Knudsen L L, Hasenkam J M. Transluminal implan-tation of artificial heart valves: Description of a new expand-able aortic valve and initial results with implantation by catheter technique in closed chest pigs[J]. Eur Heart J, 1992, 13(5): 704-708.
[3] Cribier A, Eltchaninoff H, Bash A, et al. Percutaneous transcatheter implantation of an aortic valve prosthesis for calcific aortic stenosis: first human case description[J]. Circulation, 2002, 106(24): 3006-3008.
[4] Grube E, Laborde J C, Gerckens U, et al. Percutaneous implantation of the CoreValve self-expanding valve prosthesis in high-risk patients with aortic valve disease: the Siegburg first-in-man study[J]. Circulation, 2006, 114(15): 1616-1624.
[5] FDA. The Edwards SAPIEN THV transcatheter heart valve system for patients with severe aortic stenosis who are not candidates for conventional open-heart aortic valve replacement surgery [EB/OL]. (2011-07)[2014-06-20]. http://www.fda.gov/downloads/advisoryco-mmittees/committeesmeetingmaterials/medicaldevices/medicaldev-icesadvisorycommittee/circulatorysystemdevicespanel/ucm262935.pdf.
[6] FDA. Summary of safety and effectiveness data [EB/OL]. (2014-01)[2014-06-20]. http://www.accessdata.fda.gov/cdrh_docs/pdf13/P130021b.pdf.
[7] Boston Scientific announces first commercial implants of the Lotus valve system in Europe [EB/OL]. (2013-11-15)[2014-06-20]. http://news.bostonscientific.com/2013-11-15-Boston-Scientific-Announces-First-Commercial-Implants-Of-T-he-Lotus-Valve-System-in-Europe.
[8] Vahanian A, Alfieri O, Andreotti F, et al. Guidelines on the management of valvular heart disease (version 2012)[J]. Eur Heart J, 2012, 33(19): 2451-2496.
[9] Leon M B, Piazza N, Nikolsky E, et al. Standardized endpoint definitions for transcatheter aortic valve implantation clinical trials: a consensus report from the Valve Academic Research Consortium[J]. Eur Heart J, 2011, 32(2): 205-217.
[10] NICE. Transcatheter aortic valve implantation for aortic stenosis [EB/OL].(2012-03)[2014-06-20]. http://guidance.nice.org.uk/IPG421/PublicInfo/pdf/English.
[11] Vahanian A, Alfieri O, Al-Attar N, et al. Transcatheter valve implantation for patients with aortic stenosis: a position statement from the European association of cardio-thoracic surgery (EACTS) and the European Society of Cardiology (ESC), in collaboration with the European Association of Percutaneous Cardiovascular Interventions (EAPCI)[J]. EuroIntervention, 2008, 4(2): 193-199.
[12] Kappetein A P, Head S J, Genereux P, et al. Updated standardized endpoint definitions for transcatheter aortic valve implantation: the Valve Academic Research Consortium-2 consensus document[J]. EuroIntervention, 2012, 8(7): 782-795.

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