Updating clinical endpoint definitions.

Updating clinical endpoint definitions.
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DOI:
10.4103/2045-8932.109920
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发表时间:
2013-01
影响因子:
2.6
通讯作者:
Oudiz RJ
Oudiz RJ
中科院分区:
医学4区
文献类型:
--
作者:
Hassoun PM;Nikkho S;Rosenzweig EB;Moreschi G;Lawrence J;Teeter J;Meier C;Ghofrani AH;Minai O;Rinaldi P;Michelakis E;Oudiz RJ

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在过去20年进行的临床试验中,6分钟步行距离(6-MWD)是判断肺动脉高压(PAH)治疗疗效的最常用终点。尽管6-MWD简单,在最近的试验中广泛使用,具有总体预后价值,但在过去几年中,6-MWD经常受到批评,文献中出现了几位PAH专家的请求,希望找到更可靠的替代终点,以反映肺血管阻力以及PAH的心脏状态及其对治疗的反应。2012年初召开了PAH专家和监管机构及制药公司代表会议,讨论当前和新兴有价值终点的有效性。目前的工作是会议的议事录。
The 6-Minute Walk Distance (6-MWD) has been the most utilized endpoint for judging the efficacy of pulmonary arterial hypertension (PAH) therapy in clinical trials conducted over the past two decades. Despite its simplicity, widespread use in recent trials and overall prognostic value, the 6-MWD has often been criticized over the past several years and pleas from several PAH experts have emerged from the literature to find alternative endpoints that would be more reliable in reflecting the pulmonary vascular resistance as well as cardiac status in PAH and their response to therapy. A meeting of PAH experts and representatives from regulatory agencies and pharmaceutical companies was convened in early 2012 to discuss the validity of current as well as emerging valuable endpoints. The current work represents the proceedings of the conference.
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