Novel Methods to Grow the Impact of Pediatric Thoracic Transplantation

项目摘要/摘要： 需要胸部(心脏或肺)移植的儿童患者的死亡率高得令人无法接受。 等待名单，即使有相当数量的捐赠者没有得到利用。许多捐赠者决心 高风险和浪费可能会使高风险接受者受益，他们更有可能在等待中死亡。我们建议(目标1) 开发一种创新的基于风险的捐赠者和接受者配对方法。这将通过开发 心脏和肺移植的儿科特定、供体和受者风险评分模型。风险分层 从分数中得出的结果将被用于基于模拟的模型中，以确定最佳匹配策略 可以为移植社区提供最佳的总体结果(更多的患者被移植，等待时间更短 时间，减少等待名单上的死亡率和整个社区移植后更长的寿命)。此外， 儿科胸部移植社区仍在使用一种古老的方法，即以体重/身高为基础的捐赠者和受者 体型匹配，导致大量捐献者因体型不匹配而被拒绝。我们还提议 (目标2)开发创新和普遍可用的虚拟器官大小匹配工具，而不是 患者大小，以改善儿童胸部移植的大小匹配。这将包括开发 经过验证的心肺容量估计工具和一项临床研究，以证明 该工具适用于儿科心脏和肺移植患者。我们的团队已经收集了初步数据来支持 这个项目的前提和可行性，成员代表不同的观点和长处 这是这个项目需要的。在这个项目结束时，我们将能够展示捐赠者的潜在好处- 受体风险匹配和器官大小匹配可以提高捐赠者的利用率并减少大小不匹配 并最终提高小儿胸部移植的效果。

Project Summary/Abstract: Pediatric patients in need of thoracic (heart or lung) transplantation suffer unacceptably high mortality on the waitlist, even when there are significant number of donors that go unutilized. Many donors that are determined high-risk and wasted could benefit high-risk recipients that are more likely to die waiting. We propose (Aim 1) development of an innovative risk-based matching of donor and recipient. This will be achieved by developing pediatric specific, donor and recipient risk scoring models for heart and lung transplantation. Risk stratification derived from the scores will then be used in simulation-based models to identify best matching strategy that could deliver optimal overall outcomes for the transplant community (more patients transplanted, shorter waiting time, reduce wait-list mortality and more years of life post-transplant for the community as a whole). Furthermore, pediatric thoracic transplant community is still using an age-old method of weight/height-based donor to recipient size matching which leads to significant number of donors being refused for size mismatch. We also propose (Aim 2) development of in innovative and universally accessible tool for virtual matching of organ size rather than patient size to improve size matching for pediatric thoracic transplantation. This will include development of validated tools for cardiac and lung volume estimation and a clinical study to demonstrate potential benefits of the tool in pediatric heart and lung transplant candidates. Our team has gathered preliminary data to support the premise and feasibility of this project and has the members representing diverse perspective and strengths needed for this project. At the end of this project, we will be able to demonstrate potential benefits of donor- recipient risk matching and organ sizing matching that could improve donor utilization and reduce size mismatch and ultimately improve the impact of pediatric thoracic transplantation.