美国加州锡达-西奈医疗中心Raj R Makkar团队比较了环腔内自膨胀瓣膜和市售经导管瓣膜治疗高危或极危严重主动脉瓣狭窄患者的效果。2020年6月25日出版的《柳叶刀》杂志发表了这项成果。

环腔内自膨胀经导管主动脉瓣膜系统与任何市售瓣膜相比，其安全性和有效性需通过随机试验来进行评估。

在这项前瞻性、多中心、非劣效性、随机对照试验中，2014年5月30日至9月12日，以及2015年8月21日至2017年10月10日，研究组在美国和澳大利亚的52个经导管主动脉瓣替换术经验丰富的医疗中心，招募了750例高危和极危严重主动脉瓣狭窄的患者，他们的年龄在21岁及以上，纽约心脏协会功能分级II级及以上，患有严重的主动脉狭窄。将其按1：1随机分组，其中381例接受第一代Portico瓣膜和输送系统治疗，369例接受市售瓣膜治疗。主要安全终点是30天时全因死亡率、致残性中风、需输血的致命性出血、需透析的急性肾损伤或主要血管并发症的复合结局。主要疗效终点为1年时的全因死亡率或致残性中风。

750名参与者的平均年龄为83岁，其中52.7%为女性。Portico瓣膜组有13.8%的患者发生30天主要安全终点，显著高于市售瓣膜组（9.6%）。Portico瓣膜组1年主要疗效终点发生率为14.8%，市售瓣膜组为13.4%，差异不显著。Portico瓣膜组2年时的死亡率为22.3%，中风致残率为3.1%，与市售瓣膜组（分别为20.2%和5.0%）相差不大。

总之，与市售瓣膜相比，Portico瓣膜在2年时的死亡率或中风致残率相差不大，但包括30天死亡在内的主要复合安全终点发生率更高。第一代Portico瓣膜和输送系统与市售瓣膜相比并无优势。

附：英文原文

Title: Self-expanding intra-annular versus commercially available transcatheter heart valves in high and extreme risk patients with severe aortic stenosis (PORTICO IDE): a randomised, controlled, non-inferiority trial

Author: Raj R Makkar, Wen Cheng, Ron Waksman, Lowell F Satler, Tarun Chakravarty, Mark Groh, William Abernethy, Mark J Russo, David Heimansohn, James Hermiller, Stephen Worthley, Bassem Chehab, Mark Cunningham, Ray Matthews, Ravi K Ramana, Gerald Yong, Carlos E Ruiz, Chunguang Chen, Federico M Asch, Mamoo Nakamura, Hasan Jilaihawi, Rahul Sharma, Sung-Han Yoon, Augusto D Pichard, Samir Kapadia, Michael J Reardon, Deepak L Bhatt, Gregory P Fontana

Issue&Volume: 2020-06-25

Abstract: Background

Randomised trial data assessing the safety and efficacy of the self-expanding intra-annular Portico transcatheter aortic valve system (Abbott Structural Heart, St Paul, MN, USA) compared with any commercially available valves are needed to compare performance among designs.

Methods

In this prospective, multicentre, non-inferiority, randomised controlled trial (the Portico Re-sheathable Transcatheter Aortic Valve System US Investigational Device Exemption trial [PORTICO IDE]), high and extreme risk patients with severe symptomatic aortic stenosis were recruited from 52 medical centres experienced in performing transcatheter aortic valve replacement in the USA and Australia. Patients were eligible if they were aged 21 years or older, in New York Heart Association functional class II or higher, and had severe native aortic stenosis. Eligible patients were randomly assigned (1:1) using permuted block randomisation (block sizes of 2 and 4) and stratified by clinical investigational site, surgical risk cohort, and vascular access method, to transcatheter aortic valve replacement with the first generation Portico valve and delivery system or a commercially available valve (either an intra-annular balloon-expandable Edwards-SAPIEN, SAPIEN XT, or SAPIEN 3 valve [Edwards LifeSciences, Irvine, CA, USA]; or a supra-annular self-expanding CoreValve, Evolut-R, or Evolut-PRO valve [Medtronic, Minneapolis, MN, USA]). Investigational site staff, implanting physician, and study participant were unmasked to treatment assignment. Core laboratories and clinical event assessors were masked to treatment allocation. The primary safety endpoint was a composite of all-cause mortality, disabling stroke, life-threatening bleeding requiring transfusion, acute kidney injury requiring dialysis, or major vascular complication at 30 days. The primary efficacy endpoint was all-cause mortality or disabling stroke at 1 year. Clinical outcomes and valve performance were assessed up to 2 years after the procedure. Primary analyses were by intention to treat and the Kaplan-Meier method to estimate event rates. The non-inferiority margin was 8·5% for primary safety and 8·0% for primary efficacy endpoints. This study is registered with ClinicalTrials.gov, NCT02000115, and is ongoing.

Findings

Between May 30 and Sept 12, 2014, and between Aug 21, 2015, and Oct 10, 2017, with recruitment paused for 11 months by the funder, we recruited 1034 patients, of whom 750 were eligible and randomly assigned to the Portico valve group (n=381) or commercially available valve group (n=369). Mean age was 83 years (SD 7) and 395 (52·7%) patients were female. For the primary safety endpoint at 30 days, the event rate was higher in the Portico valve group than in the commercial valve group (52 [13·8%] vs 35 [9·6%]; absolute difference 4·2, 95% CI 0·4 to 8·8 [upper confidence bound {UCB} 8·1%]; p non-inferiority=0·034, p superiority=0·071). At 1 year, the rates of the primary efficacy endpoint were similar between the groups (55 [14·8%] in the Portico group vs 48 [13·4%] in the commercial valve group; difference 1·5%, 95% CI 3·6 to 6·5 [UCB 5·7%]; p non-inferiority=0·0058, p superiority=0·50). At 2 years, rates of death (80 [22·3%] vs 70 [20·2%]; p=0·40) or disabling stroke (10 [3·1%] vs 16 [5·0%]; p=0·23) were similar between groups.

Interpretation

The Portico valve was associated with similar rates of death or disabling stroke at 2 years compared with commercial valves, but was associated with higher rates of the primary composite safety endpoint including death at 30 days. The first-generation Portico valve and delivery system did not offer advantages over other commercially available valves.

DOI: 10.1016/S0140-6736(20)31358-1

Source: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(20)31358-1/fulltext