SINGLE VENTRICLE RECONSTRUCTION TRIAL

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。左心发育不良综合征（HLHS）和相关的单右心室（RV）异常是高危的先天性心血管畸形（CCVM）。这些患者的手术缓和包括新生儿期的Norwood手术，4-6个月时的II期手术和大约18 - 36个月时的改良Fontan手术。诺伍德手术仍然是先天性心脏手术中风险最高的手术之一。最近，一些小的、非随机的研究报道了一种新的诺伍德手术方法的改善结果，该方法使用右心室到肺动脉（rv - pa）分流来提供肺血流，而不是标准的改良Blalock-Taussig分流（MBTS）。儿科心脏网络提出的多机构随机临床试验将评估接受Norwood手术的患者的早期和中期结果，无论是RV-to-PA分流术还是MBTS。诊断为单一形态右心室异常的婴儿将有资格纳入本研究。参与者将被随机分配接受MBTS或rv - pa分流术，随机分组根据主动脉闭锁（存在或不存在）和肺静脉回流阻塞（存在或不存在）分层。动态分配将用于确保外科医生之间的治疗臂平衡。数据将在Norwood手术结束时收集，在II期手术之前（大约6个月大），然后在随机化后12个月和14个月再次收集。该研究的主要目的是比较随机化后12个月MBTS与rv - pa分流术对死亡率或心脏移植率的影响。次要目标包括Norwood和II期缓解手术后的术后发病率，II期缓解时的右心室功能和肺动脉生长，以及14个月时的神经发育结果。不良事件的发生率也将在治疗组之间进行比较。总样本量目标为466名参与者，将在26个月内招募。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Hypoplastic left heart syndrome (HLHS) and related single right ventricle (RV) anomalies are the highest-risk congenital cardiovascular malformations (CCVM). Surgical palliation for these patients consists of the Norwood procedure during the newborn period, a stage II procedure at 4-6 months and the modified Fontan procedure at approximately 18 to 36 months. The Norwood procedure remains one of the highest risk procedures in congenital heart surgery. Recently, improved outcomes have been reported in a few small, non-randomized studies of a novel approach to the Norwood procedure, which uses a right ventricle to pulmonary artery (RV-to-PA) shunt to provide pulmonary blood flow rather than the standard modified Blalock-Taussig shunt (MBTS). The Pediatric Heart Network's proposed multi-institutional, randomized clinical trial will evaluate early and intermediate-term outcomes for patients undergoing a Norwood procedure with either the RV-to-PA shunt or MBTS. Infants with a diagnosis of single, morphologic right ventricle anomaly will be eligible for inclusion in this study. Participants will be randomly assigned to receive either a MBTS or RV-to-PA shunt, with randomization stratified by aortic atresia (presence or absence) and obstructed pulmonary venous return (presence or absence). Dynamic allocation will be used to ensure treatment arms are balanced across surgeons. Data will be collected at discharge from the Norwood procedure, prior to the stage II procedure (at approximately 6 months of age), and then again at 12 and 14 months post-randomization. The primary aim of the study will be to compare the effect of the MBTS to that of the RV-to-PA shunt on the rate of death or cardiac transplantation 12 months after randomization. Secondary aims include post-operative morbidity following the Norwood and stage II palliation procedures, RV function and pulmonary artery growth at the time of the stage II palliation, and neurodevelopmental outcome at 14 months. The incidence of adverse events will also be compared between the treatment groups. The total sample size target of 466 participants will be recruited over 26 months.