Pediatric Heart Network Medical University of South Carolina

描述（由申请人提供）： 南卡罗来纳州的儿童心脏项目是一个全州范围的儿科心脏病专家联盟，他们照顾该州450万居民中的90%以上。该联盟具有PHN中心的所有关键要素：足够的患者数量，临床研究基础设施，受试者招募的跟踪记录以及对假设驱动研究的奉献精神。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教师作为PI参与了各种多中心研究，该提案的PI通常担任首席研究员。MUSC教师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成为PHN的顶级受试者之一。 方案：主动脉-肺动脉侧支（APC）弹簧圈闭塞对Fontan结局的随机试验。在过去的25年里，治疗功能性单心室的总体策略是分期手术姑息治疗，最终进行Fontan手术。然而，具体做法差别很大。PHN Fontan研究回顾性证明，Fontan手术前的APC闭塞显示出明显的实践差异，与后期功能状态无关，但没有关于血流动力学影响的数据。提出了一项多中心试验，主要目的是评价术前APC闭塞是否影响急性Fontan结局。假设：盘绕重要APC与结局变化无关。主要结局：Fontan手术后最初24小时内的Fontan压力。次要结局包括其他血流动力学参数、胸腔积液持续时间和住院时间。在42个月的时间内，将招募并评价总计240例患者，在p=0.05水平下检测显著性的把握度为0.85。研究结果将为APC弹簧圈栓塞术的必要性提供证据基础，从而可能重新评估术前导管插入术的必要性，并在这一重要患者组中采取或多或少的积极措施来闭塞APC。 相关性：用于单心室患者的资源非常高。因此，确定最佳的管理，以改善结果和减少住院时间后Fontan程序应减少发病率和提高资源利用率。本研究将为这一重要患者组术前需要行腰椎间盘弹簧圈栓塞术提供证据基础。

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.