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）病变组是功能性单右心室（FSRV）畸形，包括左心发育不良综合征。早期婴儿期后的生存率，曾经不可能为这一患者群体，已显着改善，在过去的30年中，由于创新和完善的手术和围手术期护理。诺伍德手术是这些儿童3期手术姑息治疗的第一步，目前在选定的儿科心脏中心进行，住院生存率> 90%。然而，从诺伍德手术后出院到4至6个月大时开始II期手术之间的死亡率仍然高达20%，令人无法接受。来自两个小型、单中心病例系列的数据表明，从诺伍德出院到II期手术期间进行的全面家庭监测计划（CHMP）可显著降低期间死亡率。在CHMP中，在诺伍德出院时将数字秤和脉搏血氧仪分发给家庭，并指导父母护理人员进行日常测量。如果预先确定的氧饱和度或体重增加阈值被突破，则由医生在24小时内对患者进行评估。虽然CHMP直观上具有吸引力，但缺乏可靠的循证数据，并且尚未系统地评估与“假阳性数据”相关的行动（如侵入性检测或早期II期手术）的潜在负面影响。此外，缺乏第三方支付者对这种有前途但未经证实的治疗的报销仍然是先天性心脏病中心广泛实施的重大障碍。作为密歇根大学先天性心脏病中心向PHN申请的一部分，拟议的研究涉及一项多中心随机临床试验，以确定CHMP是否改善FSRV畸形儿童的期间生存率，并评估潜在的医疗后果和家庭监测计划的财务成本。 相关性：FSRV病变是最常见的严重先天性心脏畸形，在美国每年影响约1000名新生儿。诺伍德一期手术的住院死亡率近年来有了显著改善;出院和二期手术之间的时期现在是这些儿童的最高风险时期。拟议的试验针对患有FSRV的儿童的高风险时期。

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.