SINGLE VENTRICLE RECONSTRUCTION TRIAL

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。左心发育不良综合征（HLHS）和相关的单右心室（RV）畸形是最高风险的先天性心血管畸形（CCVM）。这些患者的手术姑息治疗包括新生儿期的诺伍德手术、4-6个月时的II期手术和约18 - 36个月时的改良Fontan手术。诺伍德手术仍然是先天性心脏病手术中风险最高的手术之一。最近，一些小型非随机研究报告了诺伍德手术新方法的结局改善，该方法使用右心室至肺动脉（RV至PA）分流术而不是标准改良Blalock-Taussig分流术（MBTS）提供肺血流。儿科心脏网络提出的多机构随机临床试验将评估接受诺伍德手术的患者的早期和中期结局，无论是RV至PA分流术还是MBTS。 诊断为单个形态学右心室异常的婴儿将有资格入选本研究。受试者将被随机分配接受MBTS或RV至PA分流术，随机化按主动脉闭锁（存在或不存在）和阻塞性肺静脉回流（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组平衡。将在诺伍德手术出院时、II期手术前（约6个月大时）以及随机化后12个月和14个月时收集数据。本研究的主要目的是比较MBTS与RV-PA分流术对随机化后12个月死亡率或心脏移植率的影响。次要目的包括诺伍德和II期姑息手术后的术后发病率、II期姑息手术时的RV功能和肺动脉生长以及14个月时的神经发育结局。还将比较治疗组之间不良事件的发生率。将在26个月内招募466名参与者的总样本量目标。

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.