Pediatric Heart Network: Follow-up of the Single Ventricle Reconstruction Trial

描述（由申请人提供）： 单右心室（RV）新生儿姑息性手术，即诺伍德1手术，在婴儿期进行的所有心脏外科手术中死亡率和发病率风险最高。单心室重建（SVR）试验是一项正在进行的儿科心脏网络（PHN）研究，旨在比较诺伍德1手术的两种手术改良之间的1年结局。这两种改良对RV心肌的潜在影响不同：改良的Blalock-Taussig分流（MBTS）导致血液从主动脉到肺动脉的舒张期流出，可能影响冠状动脉血流，而RV-肺动脉（RV-PA）分流避免了这种潜在的冠状动脉盗血现象，但存在右心室切开术造成右心室损伤的长期风险。SVR试验的主要假设是RV至PA分流术在1岁时的死亡或移植发生率较低。在该患者人群中，持续存在死亡或移植超过一年的风险，特别是在18个月至3岁之间进行计划的心脏直视手术。这项多中心前瞻性随访研究旨在将RV至PA分流术和MBTS存活者的临床结局和RV性能的比较延长至4岁。主要目的是比较两组之间的死亡或移植的中期联合终点，假设RV至PA分流术在4岁时的死亡或移植发生率低于MBTS。次要目的将包括比较神经发育结局、健康相关生活质量、RV功能、躯体生长、肺动脉生长、心力衰竭、心律失常的发生率以及Fontan完成后的手术和术后病程。所有入组SVR试验的受试者，如果在14个月大时存活至试验完成，将有资格入选。将在入组时、Fontan手术时（预计发生在18个月至3岁之间）和4岁时收集数据。支持临床研究技能发展核心的应用程序也包括在本提案中。与公共卫生的相关性：在所有先天性心脏病病变中，左心发育不良综合征的手术死亡率或移植率最高。这项多中心、前瞻性研究的结果将提供关于此病变的最佳新生儿外科干预的重要信息。(End摘要）

DESCRIPTION (provided by applicant): Palliative surgery in neonates with a single right ventricle (RV), the Norwood 1 procedure, carries the highest risk for mortality and morbidity of all cardiac surgical procedures performed in infancy. The Single Ventricle Reconstruction (SVR) Trial is an ongoing Pediatric Heart Network (PHN) study designed to compare one year outcomes between two surgical modifications of the Norwood 1 procedure. The two modifications differ in their potential effect on RV myocardium: the modified Blalock-Taussig shunt (MBTS) results in a diastolic run-off of blood from the aorta to the pulmonary arteries that may compromise coronary blood flow, while the RV-to-pulmonary artery (RV-to-PA) shunt avoids this potential coronary artery steal phenomenon, but has a long term risk of right ventricular damage from a right ventriculotomy. The primary hypothesis of the SVR Trial is that the RV-to-PA shunt will have a lower incidence of death or transplant at one year of age. There is an ongoing risk of death or transplant extending beyond one year in this patient population, especially as planned open heart surgery is performed between 18 months and 3 years of age. This multicenter prospective follow-up study is designed to extend comparison of clinical outcome and RV performance between survivors of the RV-to-PA shunt and MBTS up to age 4 years. The primary aim is to compare the intermediate-term combined endpoint of death or transplant between the two groups with the hypothesis that the RV-to-PA shunt will have a lower incidence of death or transplantation compared the MBTS at 4 years of age. Secondary aims will include comparison of neurodelopmental outcome, health-related quality of life, RV function, somatic growth, pulmonary artery growth, the incidence heart failure, arrhythmias and the operative and post-operative course following Fontan completion. All subjects enrolled in the SVR Trial who survive to completion at 14 months of age will be eligible for inclusion. Data will be collected at the time of enrollment, at the time of the Fontan surgery (expected to occur between 18 months-3 years of age) and at age 4 years. An application to support a Clinical Research Skills Development Core is also included in this proposal. Relevance to public health: Surgery for hypoplastic left heart syndrome has the highest incidence of death or transplantation of all congenital heart disease lesions. The results of this multicenter, prospective study will provide important information regarding the optimal neonatal surgical intervention for this lesion. (End of Abstract)