Randomized Clinical Trials for Pediatric Heart Disease

小儿心脏病的随机临床试验

• 批准号: 7902074
• 负责人: J Philip SAUL
• 金额: $ 28.35万
• 依托单位: MEDICAL UNIVERSITY OF SOUTH CAROLINA
• 依托单位国家: 美国
• 财政年份: 2001
• 资助国家: 美国
• 起止时间: 2001-09-01 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7902074
• 关键词: Admission activity; Adrenal Cortex Hormones; Adverse event; Blood capillaries; Bypass; Cardiac; Cardiopulmonary Bypass; Caring; Cessation of life; Child; Childhood; Clinical; Clinical Research; Clinical Research Protocols; Data; Dedications; Development; Dose; EFRAC; Elements; Enrollment; Faculty; Fluid Balance; Heart; Heart Diseases; Hour; Incidence; Individual; Inflammatory; Inflammatory Response; Intake; Intravenous; Lead; Left ventricular structure; Length of Stay; Liquid substance; Low Cardiac Output; Matrix Metalloproteinases; Measures; Mechanical ventilation; Methylprednisolone; Multicenter Trials; Neonatal; Operative Surgical Procedures; Output; Participant; Patients; Peroxidases; Placebos; Play; Process; Production; Protocols documentation; Randomized Clinical Trials; Recovery; Registries; Research Infrastructure; Research Personnel; Research Support; Resources; Right ventricular structure; Role; Running; Safety; Sampling; Schedule; Serious Adverse Event; South Carolina; Steroids; Support Groups; Techniques; Technology; Three-Dimensional Echocardiography; Time; Urine; Ventricular; Ventricular Function; Whole Blood; abstracting; capillary; cytokine; indexing; milliliter; neonate; novel; programs; randomized trial

DESCRIPTION (provided by applicant): The Children's Heart Program of South Carolina, with MUSC as the tertiary referral center, is a statewide consortium of pediatric cardiologists, who care for >90% of the 4.0 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 clinical research. Indeed, these elements have allowed MUSC to be a highly successful participant in the PHN. Further, MUSC faculty have participated as Pi's in a large number and wide variety of multicenter trials or registries, and the Pi's of this proposal have often been the lead investigator. MUSC faculty also currently run numerous local clinical research protocols. Participation in all of these protocols is supported by a dedicated pediatric cardiac research support group with RN coordinators and an administrative FTE. The combined resources of patient volume and strong research infrastructure have enabled MUSC to be among the top subject enrollers as well. Protocol: Randomized Trial of Preoperative Corticosteroid Therapy in Neonates Undergoing Cardiopulmonary Bypass (CPB). CPB is critical to corrective intracardiac surgery, but the pathophysiologic processes engendered by CPB, particularly in neonates, play an important role in post- CPB recovery. The use, doses and schedule of glucortocoid administration to ameliorate these CPB induced processes appears to be highly variable among pediatric cardiac surgical centers, without clear data to provide direction. The Primary Aim of this study is to compare the effects of preoperative intravenous methypredisolone (IVMP) to placebo on the Primary Endpoint of low cardiac output state (LCOS) or death at 36 hours following neonatal CPB. We hypothesize that neonates receiving preoperative IVMP will have a decreased incidence of the Primary Endpoint. Secondary Endpoints include: inotropic score at 36 hours, fluid balance, ICU stay parameters, echocardiographic measures, levels of inflammatory molecules, and safety parameters. One unique aspect of the protocol is the use of a novel multiplexer technology which allows analysis of a broad array of cytokines, matrix metalloproteinases and myeloperoxidase from a sample of one milliliter or less of whole blood. The results of this study should clearly define the clinical utility of preoperative steroids and help determine the mechanisms for any observed effect. (End of Abstract).

描述(由申请人提供)： 南卡罗来纳州儿童心脏计划，以南卡罗来纳州为第三转诊中心，是全州范围内的儿科心脏病专家联盟，他们照顾该州400万居民中的90%。该联盟拥有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临床研究。 事实上，这些因素使MUSC成为PHN的一个非常成功的参与者。此外，MUSC的教职员工作为PI参与了大量和种类繁多的多中心试验或登记，而这项提案的PI通常是主要的调查者。密歇根州立大学的教职员工目前还运行着许多当地的临床研究方案。所有这些方案的参与都得到了一个专门的儿科心脏研究支持小组的支持，该小组有RN协调员和一名行政FTE。患者数量和强大的研究基础设施相结合的资源使MUSC也成为顶级学科招生机构之一。方案：新生儿体外循环(CPB)术前糖皮质激素治疗的随机试验。体外循环对矫正性心内直视手术至关重要，但体外循环所产生的病理生理过程，尤其是新生儿，在体外循环后的恢复中起着重要作用。在儿科心脏外科中心，糖皮质激素用于改善体外循环诱导的这些过程的使用、剂量和时间安排似乎有很大的差异，没有明确的数据提供指导。本研究的主要目的是比较术前静脉注射甲基强的松龙(IVMP)和安慰剂对新生儿CPB后36小时低心排状态(LCOS)或死亡的主要终点的影响。我们假设，术前接受IVMP治疗的新生儿主要终点的发生率将会降低。次要终点包括：36小时的肌力评分、液体平衡、ICU停留参数、超声心动图测量、炎症分子水平和安全参数。该协议的一个独特方面是使用了一种新的多路复用器技术，该技术允许从一毫升或更少的全血样本中分析一系列广泛的细胞因子、基质金属蛋白酶和髓过氧化物酶。这项研究的结果应该清楚地定义术前类固醇的临床效用，并有助于确定任何观察到的效果的机制。(摘要结束)。

项目成果

Autonomic heart rate control at rest and during unloading of the right ventricle in repaired tetralogy of Fallot in adolescents.

• DOI: 10.1016/j.amjcard.2008.05.065
• 发表时间: 2008-10-15
• 期刊: AMERICAN JOURNAL OF CARDIOLOGY
• 影响因子: 2.8
• 作者: Wyller, Vegard Bruun;Saul, J. Philip;Barbieri, Riccardo;de Lange, Charlotte;Hopp, Einar;Norum, Ingvild B.;Thaulow, Erik
• 通讯作者: Thaulow, Erik

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• DOI: 10.1002/art.40923
• 发表时间: 2019
• 期刊: Arthritis & rheumatology (Hoboken, N.J.)
• 作者: Kim,AlfredHJ;Strand,Vibeke;Atkinson,JohnP
• 通讯作者: Atkinson,JohnP