The Johns Hopkins Hospital, Baltimore, MD, USA.
Am Heart J. 2012 Jul;164(1):7-13. doi: 10.1016/j.ahj.2012.04.008. Epub 2012 Jun 20.
Coarctation of the aorta (CoA) accounts for 4% to 5% of congenital cardiac abnormalities. Stent therapy has become an accepted alternative to surgery for older children and adults, although there are no balloon-expandable stents approved by the Food and Drug Administration for use in the aorta. The Cheatham-Platinum (CP) stent was designed for CoA therapy and is widely used outside the United States. We have designed the first prospective trial of stent therapy for CoA to serve as the pivotal trial for Food and Drug Administration approval of the CP stent.
The COAST study is a prospective, multicenter, single-arm clinical study. The population includes patients with native or recurrent CoA. Four primary outcome variables were defined. For each variable, the stent will be compared to performance guidelines derived from surgical experience. The first efficacy outcome is reduction in arm-leg systolic blood pressure gradient, and the second is reduction in hospital length of stay. Safety outcomes include the following: the occurrence of any serious or somewhat serious adverse event attributed to the stent or implantation procedure and the occurrence of postprocedure paradoxical hypertension. A total of 105 patients treated with the CP stent will be enrolled.
To ascertain the effectiveness and safety of an interventional device, randomized controlled trials have been offered as the criterion standard. However, these trials are not well suited to study rare conditions such as CoA, especially once the therapy in question has received acceptance within the medical community. New clinical trial and statistical approaches are needed to evaluate such therapies. The COAST study is an example of this kind of innovative trial design.
主动脉缩窄（CoA）占先天性心脏异常的 4%至 5%。支架治疗已成为大龄儿童和成人的一种可接受的替代手术方法，尽管食品和药物管理局尚未批准用于主动脉的球囊扩张支架。Cheatham-Platinum（CP）支架是专为 CoA 治疗设计的，在美国以外广泛使用。我们设计了首例 CoA 支架治疗的前瞻性试验，作为 CP 支架获得食品和药物管理局批准的关键试验。
COAST 研究是一项前瞻性、多中心、单臂临床研究。该人群包括患有原生或复发性 CoA 的患者。定义了四个主要的结局变量。对于每个变量，支架将与源自手术经验的性能指南进行比较。第一个疗效结局是减少手臂-腿部收缩压梯度，第二个是减少住院时间。安全性结局包括以下内容：任何归因于支架或植入程序的严重或有些严重的不良事件的发生，以及术后出现矛盾性高血压的发生。将招募总共 105 名接受 CP 支架治疗的患者。
为了确定介入治疗装置的有效性和安全性，已经提供了随机对照试验作为标准。然而，这些试验并不适合研究罕见疾病，如 CoA，特别是在有关治疗方法已经在医学界得到认可之后。需要新的临床试验和统计方法来评估此类治疗方法。COAST 研究就是这种创新试验设计的一个例子。
