Pediatric Heart Network Medical University of South Carolina

描述(由申请人提供)： 南卡罗来纳州儿童心脏计划是一个全州范围的儿科心脏病专家联盟，他们为该州450万居民中的90%提供服务。该联盟具备PHN中心的所有关键要素：充足的患者数量、临床研究基础设施、受试者登记的记录以及致力于假设驱动的研究。这些因素使MUSC成为PHN的一个非常成功和积极的行政参与者。此外，MUSC的教职员工作为PI参与了广泛的多中心研究，这项提案的PI经常担任首席研究员。MUSC教员目前还指导许多当地的临床研究方案。患者数量、研究基础设施和奉献精神的综合资源使MUSC也成为PHN中最好的学科招生机构之一。 方案：主肺侧支(APC)线圈闭塞对Fontan结局的随机试验。在过去的25年中，管理功能性单心室的总体策略是从手术姑息到最终的Fontan手术。然而，具体的方法差别很大。PHN Fontan研究回顾表明，在Fontan手术前，APC闭塞表现出明显的实践变化，与后来的功能状态无关，但没有关于血流动力学影响的数据。建议进行一项多中心试验，主要目的是评估APC术前闭塞是否影响急性Fontan预后。假设：盘绕显著的APC不会与结果的改变相关。主要结果：Fontan手术后24小时内的Fontan压力。次要结果包括额外的血流动力学参数、胸腔积液持续时间和住院时间。总共将招募240名患者，并在42个月内对其进行评估，得出0.85的力量来检测p=0.05水平的显著性。研究结果将为APC盘绕的必要性提供证据基础，导致可能重新评估术前插管的必要性，以及或多或少积极地封堵这一重要患者组的APC。 相关性：单脑室患者所使用的资源非常高。因此，确定Fontan手术后改善预后和缩短住院时间的最佳管理方法应能降低发病率并提高资源利用率。这项研究将为这一重要患者群体的术前需要进行主肺卷曲提供证据依据。

DESCRIPTION (provided by applicant): The Children's Heart Program of South Carolina is a state wide consortium of pediatric cardiologists, who care for >90% of the 4.5 million residents in the state. This consortium has all the critical elements for a center in the PHN: adequate patient volume, clinical research infrastructure, a track record of subject enrollment, and a dedication to hypothesis driven research. These elements have allowed MUSC to be a highly successful and administratively active participant in the PHN. Further, MUSC faculty have participated as PI's in a wide variety of multicenter studies, with the PI's of this proposal often acting as the lead investigator. MUSC faculty also currently directs numerous local clinical research protocols. The combined resources of patient volume, research infrastructure and dedication have enabled MUSC to be among the top subject enrollers in the PHN as well. Protocol: Randomized Trial of Aortopulmonary Collateral (APC) Coil Occlusion on Fontan Outcome. The overall strategy for managing functional single ventricle over the past 25 years has been staged surgical palliation to an eventual Fontan procedure. However, the specific approaches vary widely. The PHN Fontan study has demonstrated retrospectively that APC occlusion prior to the Fontan procedure shows marked practice variation, and no relation to later functional status, but had no data on the hemodynamic effects. A multicenter trial is proposed with the Primary Aim of evaluating whether preoperative occlusion of APCs influences acute Fontan outcome. Hypothesis: Coiling significant APCs will not be associated with a change in outcome. Primary Outcome: Fontan pressure in the first 24 hours following Fontan surgery. Secondary outcomes include additional hemodynamic parameters, duration of pleural effusions and duration of hospitalization. A total of 240 patients will be recruited and evaluated over a period of 42 months, yielding a power of 0.85 to detect significance at the p=0.05 level. The study outcome will provide an evidence basis for the need for APC coiling, leading to potential reassessment of the need for preoperative catheterization, and either more or less aggressive efforts to occlude APCs in this important patient group. RELEVANCE: The resources utilized for single ventricle patients are extraordinarily high. Therefore, determining the optimum management for improving outcomes and reducing the duration of hospitalization after the Fontan procedure should reduce morbidity and improve resource utilization. This study will provide an evidence basis for the preoperative need for aortopulmonary coiling in this important patient group.