Long-term outcomes in patients with single ventricle physiology

单心室生理学患者的长期预后 在先天性心脏病(CHD)中，单心室(SV)病变是最具挑战性的疾病 每3000名新生儿中就有1人受到影响。室性心动过速病变的治疗需要多阶段的姑息治疗，最终导致 方坦手术及其修改。尽管Fontan对提高生存和生活质量有好处，但 姑息治疗与多种疾病相关，导致过早死亡或需要移植。家庭 患有单脑室病变的儿童经常被咨询这种情况的不良预后 前提是在Fontan失败的情况下，可以进行心脏移植。然而，很少有数据是 可提供与Fontan过早衰竭和死亡风险相关的咨询，有机会成为 符合心脏移植名单，等待移植期间死亡的风险，甚至是过早移植的风险 如果他们成功地到达移植点，就会失败。在缺乏CHD纵向登记的情况下， 对我们来说，这些问题的答案是推测和评估这一弱势群体的长期结果 人口仍然不完整。 室上性心动过速患者接受一系列的干预，目的是最初提供稳定的循环，然后提供 “确定”修理。在每一步，都会做出不同的决定，以影响肺血流、容量的调节。 全身脑室负荷和暴露于紫紫的持续时间。这些决定可能对 整体手术策略的长期成功，主要由患者层面的因素驱动，但往往 也由中心特定的偏好决定。我们假设患者和中心层面的因素都可以影响 Fontan姑息疗法的长期成功，包括到达心脏移植并存活的能力。我们的 Project将通过利用现有数据集的使用来回答这些问题，该数据集结合了来自大型、 总部设在美国的CHDS临床登记机构--儿科心脏护理联盟(PCCC)，其关联数据来自 国家死亡指数(NDI)和器官采购和移植网络(OPTN)研究 注册处。这种联系使我们能够组成一个描述长期死亡率和移植的队列 接受手术的冠心病患者的状况。PCCC-NDI-OPTN数据集的深度和大小提供了 将患者水平的变量与Fontan途径的成功估计联系起来的前所未有的机会。在……里面 此外，我们可以利用SV病变的手术策略中的中心变化来进行长期比较 在存在职业不确定性的情况下，对替代管理策略进行有效性分析。 这项研究的完成将为SV患者的预期结果提供一个现实的视角，并将阐明 影响SV生理学患者远期预后的重要因素。这些信息不仅将是 对教育患者和护理提供者很重要，但也可能导致更好地选择候选人和 也许是管理策略上的修改。

Long-term outcomes in patients with single ventricle physiology Single ventricle (SV) lesions are the most challenging conditions among congenital heart defects (CHD) and affect 1 out of 3000 newborns. The management of SV lesions requires multistage palliation that culminates in the Fontan operation and its modifications. Despite its benefit to improve survival and quality of life, Fontan palliation is associated with multiple morbidities leading to premature mortality or need for transplant. Families of children with single ventricle lesions are often counseled on the poor prognosis of this condition with the premise that in case of Fontan failure, a heart transplant can be pursued. However, very little data are available to inform such counseling related to risk for premature Fontan failure and death, chances to become eligible for heart transplant listing, risk of death while waiting for transplant or even about premature transplant failure if they successfully reach the transplant point. In the absence of longitudinal registries for CHDs in the US, the answers to these questions are speculative and assessment of long-term outcomes of this vulnerable population remains incomplete. Patients with SV undergo a number of interventions aiming to provide initially a stable circulation and later a “definite” repair. At each step, various decisions are being made that affect regulation of pulmonary flow, volume load of the systemic ventricle and duration of exposure to cyanosis. These decisions may be important for the long-term success of the overall surgical strategy and are driven mostly by patient-level factors but frequently also by center-specific preferences. We hypothesize that both patient- and center-level factors can affect the long-term success of the Fontan palliation including ability to reach and survive heart transplantation. Our project will answer these questions by leveraging the use of an existing dataset combining data from a large, US-based, clinical registry for CHDs, the Pediatric Cardiac Care Consortium (PCCC), with linkage data from the National Death Index (NDI) and the Organ Procurement and Transplantation Network (OPTN) research registries. This linkage allowed us to constitute a cohort that describes the long-term mortality and transplant status of patients with an operated CHD. The depth and size of the PCCC-NDI-OPTN dataset offer an unprecedented opportunity to link patient-level variables with estimates for success in the Fontan pathway. In addition, we can leverage center variation in the surgical strategy of SV lesions to perform long-term comparative effectiveness analysis between alternative management strategies, where professional uncertainty exists. Completion of this study will provide a realistic view of expected outcomes for SV patients and will elucidate important factors affecting long-term outcomes of patients with SV physiology. This information will not only be important for educating patients and care providers but may also lead to better selection of candidates and perhaps modifications in management strategies.