Pediatric Heart Network Regents of the University of Michigan

描述(由申请人提供)： 先天性心脏畸形是最常见的出生缺陷，占所有活产婴儿的0.8%-0.9%。最常见的严重先天性心脏病(CHD)病变是功能性单右室(FSRV)畸形，包括左心发育不良综合征。由于外科和围手术期护理的创新和改进，这类患者的早期婴儿存活率曾经是不可能的，但在过去的30年里已经有了显著的改善。诺伍德手术是对这些儿童进行三阶段手术姑息的第一步，现在在选定的儿科心脏中心进行，住院存活率为90%。然而，从诺伍德手术后出院到4至6个月大的第二阶段手术开始之间的死亡率仍然高达20%，令人无法接受。来自两个小的、单中心的病例系列的数据表明，从诺伍德出院到第二阶段手术期间进行的全面家庭监测计划(CHMP)可以显著降低病死率。在CHMP中，数字秤和脉搏血氧仪被分发给诺伍德医院出院的家庭，父母照顾者被指导进行日常测量。如果超过预定的血氧饱和度或体重增加阈值，医生会在24小时内对患者进行评估。虽然CHMP在直觉上很有吸引力，但缺乏可靠的循证数据，而且还没有系统地评估与“假阳性数据”相关的潜在负面影响，如侵入性测试或早期II阶段手术。此外，这种前景看好但未经证实的治疗方法缺乏第三方支付者的报销，仍然是在先天性心脏中心广泛实施的一个重大障碍。作为密歇根大学先天性心脏中心应用于PHN的一部分，拟议的研究涉及一项多中心随机临床试验，以确定CHMP是否可以改善患有FSRV畸形的儿童的阶段间生存率，并评估这一家庭监测计划的潜在医疗后果和经济成本。 相关：FSRV病变是最常见的严重先天性心脏畸形，每年在美国影响1000-1000名新生儿。诺伍德手术I期的住院死亡率近年来有了显著的改善；出院到II期手术之间的时期现在是这些儿童的最高风险时期。拟议的试验针对的是FSRV儿童的这一高风险时间。

DESCRIPTION (provided by applicant): Congenital heart malformations are the most common birth defects, affecting 0.8-0.9% of all live births. The most frequently occurring group of serious congenital heart disease (CHD) lesions is the Functional Single Right Ventricle (FSRV) malformations, including hypoplastic left heart syndrome. Survival beyond early infancy, once impossible for this patient group, has improved markedly in the past 30 years due to innovations and refinements in surgical and perioperative care. The Norwood operation, the first step in the 3-stage surgical palliation for these children is now performed at selected pediatric cardiac centers with hospital survival rates >90%. However, the rate of death between hospital discharge following the Norwood operation and the initiation of the stage II operation at 4 to 6 months of age remains unacceptably high at up to 20%. Data from two small, single center, case series suggest that a comprehensive home monitoring program (CHMP) conducted from Norwood discharge until the stage II operation can significantly reduce interstage mortality. In a CHMP, digital scales and pulse oximeters are distributed to families at Norwood hospital discharge and parental caregivers are instructed on daily measurements. If pre-determined oxygen saturation or weight gain thresholds are breached, patients are evaluated by a physician within 24 hours. While a CHMP is intuitively attractive, sound evidence-based data are lacking, and the potential for negative implications related to actions on 'false positive data', such as invasive testing or earlier stage II operation, have not been systematically evaluated. In addition, lack of reimbursement from third party payers for this promising, but unproven, therapy remains a significant barrier to widespread implementation across congenital heart centers. As part of the University of Michigan Congenital Heart Center application to the PHN, the proposed study involves a multicenter randomized clinical trial to determine if a CHMP improves interstage survival for children with FSRV malformations, and to evaluate potential medical consequences and the financial costs of this home monitoring program. RELEVANCE: FSRV lesions are the most common group of severe congenital heart malformations, affecting - 1000 newborns in the US each year. Hospital mortality for the Stage I, Norwood operation has improved dramatically in recent years; the period between hospital discharge and the stage II operation is now the highest risk period for these children. The proposed trial targets this high risk time for children with FSRV.