Long-term outcomes in patients with single ventricle physiology

单心室生理学患者的长期结果

• 批准号: 9883836
• 负责人: Lazaros K. Kochilas
• 金额: $ 11.7万
• 依托单位: EMORY UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-07-01 至 2021-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9883836
• 关键词: Affect; Anatomy; Blood Circulation; Cardiac; Cardiac Catheterization Procedures; Caring; Cessation of life; Child; Childhood; Clinical; Cohort Studies; Common Ventricle; Congenital Heart Defects; Counseling; Cyanosis; Data; Data Linkages; Data Set; Diagnosis; Event; Exposure to; Failure; Family; Fontan Procedure; Frequencies; Geographic Locations; Goals; Healthcare; Healthcare Systems; Heart; Heart Transplantation; Heart failure; Hospital Mortality; Hospitalization; Intervention; Lead; Length; Lesion; Life Expectancy; Link; Lung; Medical; Modification; Morbidity - disease rate; Newborn Infant; Operative Surgical Procedures; Organ Transplantation; Outcome; Pathway interactions; Patient Care; Patient-Focused Outcomes; Patients; Pattern; Physiology; Premature Mortality; Procedures; Prospective Studies; Pulmonary Circulation; Pulmonary Vascular Resistance; Quality of life; Registries; Regulation; Reporting; Research; Resources; Risk; Societies; Source; Time; Transplantation; Uncertainty; Variant; Ventricular Remodeling; Vulnerable Populations; Waiting Lists; base; candidate selection; care providers; cohort; comorbidity; comparative effectiveness; experience; family burden; hemodynamics; improved; indexing; mortality; mortality risk; negative affect; organ procurement transplantation network; outcome forecast; palliation; patient oriented; patient population; post-transplant; preference; premature; repaired; success; trend; ventricular assist device

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.

单心室生理学患者的长期结果 单心室（SV）病变是先天性心脏病（CHD）和先天性心脏病中最具挑战性的病症。 影响 3000 名新生儿中的 1 名。 SV 病变的治疗需要多阶段的姑息治疗，最终导致。 Fontan 手术及其修改 尽管 Fontan 手术有利于改善生存和生活质量。 姑息治疗与导致过早死亡或需要移植的多种发病有关。 患有单心室病变的儿童经常被告知这种情况的不良预后 前提是如果发生 Fontan 衰竭，可以进行心脏移植，但数据很少。 可以提供与 Fontan 过早衰竭和死亡风险相关的此类咨询，成为 符合心脏移植清单的资格、等待移植期间的死亡风险甚至过早移植的风险 如果他们成功到达移植点，则失败。 美国，这些问题的答案是推测性的，是对这一弱势群体的长期结果的评估 人口仍然不完整。 SV 患者接受了多种干预措施，最初旨在提供稳定的循环，随后提供稳定的循环。 “确定”修复的每一步都会做出各种影响肺流量和容量调节的决定。 全身心室负荷和紫绀暴露时间这些决定可能很重要。 整体手术策略的长期成功主要由患者层面的因素驱动，但经常 我们也承认患者和中心层面的因素都会影响。 Fontan 姑息治疗的长期成功，包括实现心脏移植并存活的能力。 项目将通过利用现有数据集来回答这些问题，该数据集结合了来自大型、 总部位于美国的儿科心脏病护理联盟 (PCCC) 的先心病临床登记处，其关联数据来自 国家死亡指数 (NDI) 和器官获取和移植网络 (OPTN) 研究 这种联系使我们能够构建一个描述长期死亡率和移植的队列。 PCCC-NDI-OPTN 数据集的深度和大小提供了接受手术的 CHD 患者的状态。 将患者水平变量与 Fontan 途径成功估计联系起来的前所未有的机会。 此外，我们可以利用SV病变手术策略的中心变异来进行长期比较 在存在职业不确定性的情况下，对替代管理策略之间的有效性进行分析。 这项研究的完成将为 SV 患者的预期结果提供现实的视角，并将阐明 影响 SV 患者长期预后的重要因素不仅是生理学。 对于教育患者和护理人员很重要，但也可能导致更好地选择候选人和 也许是管理策略的修改。