Studies of Pediatric Liver Transplantation

小儿肝移植研究

• 批准号: 7121023
• 负责人: ANNE S LINDBLAD
• 金额: $ 9.24万
• 依托单位: THE EMMES COMPANY, LLC
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-05-15 至 2009-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7121023
• 关键词: Epstein Barr virus; age difference; children; clinical research; cooperative study; diagnosis design /evaluation; disease /disorder onset; disease /disorder proneness /risk; human morbidity; human subject; human therapy evaluation; immunosuppression; liver function; liver transplantation; longitudinal human study; outcomes research; patient oriented research; pediatrics; postoperative complications; postoperative state; preoperative state; prognosis; renal failure

DESCRIPTION (provided by applicant): The Studies of Pediatric Liver Transplantation (SPLIT) Registry, founded in 1995, is designed to characterize and follow trends and indications for transplant, transplantation techniques, and outcomes such as patient and graft survival, rejection incidence, growth parameters, immunosuppressive therapy and complications. Registry membership includes a central statistical and data Coordinating Center located at The EMMES Corporation in Rockville, Maryland, and 39 Transplant Centers from the US and Canada. Data is submitted over a secure password protected Internet data entry system. The activities of the registry are governed by a Scientific Advisory Board. This application seeks to secure 5-year funding in order to assess factors predicting both short and long-term (5 years or more) outcomes overall and within primary disease subgroups (biliary atresia, metabolic disease, tumor, autoimmune hepatitis, etc.). This funding will permit SPLIT to continue registration and close follow-up of patients. Registration of children in the database as of the 2002 Annual Report is 1,721 listed children with 1,187 receiving a transplant. Five-year follow-up is available for 64 children from 11 centers. If this grant were funded, by the end of the grant period it is estimated that 5-year data would be available from over 900 children from 36 centers. This would be the largest cohort of pediatric liver recipients with long term followup ever studied in the depth. This application focuses on two specific aims. The first is to develop and test models which predict short and long-term patient and graft survival and graft function of children after liver transplantation, as well as from the time of listing. Baseline factors including status at transplantation, operative factors and post-transplant complications will be evaluated and are expected to vary based on primary disease diagnosis. The second aim is to study long term consequences of liver transplantation and immunosuppression in children including: renal function, post transplant lymphoproliferative disease (PTLD), cardiovascular risk factors and growth failure. SPLIT's unique sample will allow determination of the prevalence of morbidities associated with long term immunosuppression and identification of the predictors of those morbidities. SPLIT provides an established organizational framework with a track record of success. No other research database exists that can address these questions that have been mandated by the Children's Health Care Act of 2000 (HR4365), and recognized by the Department of Health and Human Services and the Institute of Medicine (Federal Register 98-8191 ,IOM report, 1999) as priority research areas

描述（由申请人提供）： 儿科肝移植研究 (SPLIT) 注册中心成立于 1995 年，旨在描述和跟踪移植、移植技术以及患者和移植物存活、排斥发生率、生长参数、免疫抑制治疗和并发症等结果的趋势和适应症。注册会员包括位于马里兰州罗克维尔的 EMMES Corporation 的中央统计和数据协调中心以及来自美国和加拿大的 39 个移植中心。数据通过安全密码保护的互联网数据输入系统提交。登记处的活动由科学顾问委员会管理。该申请旨在获得 5 年的资助，以评估预测短期和长期（5 年或更长）总体结果和原发疾病亚组（胆道闭锁、代谢性疾病、肿瘤、自身免疫性肝炎等）结果的因素。这笔资金将使 SPLIT 能够继续登记并密切跟踪患者。截至 2002 年年度报告，数据库中登记的儿童有 1,721 名，其中 1,187 名接受了移植。来自 11 个中心的 64 名儿童可进行五年随访。如果这笔赠款得到资助，预计到赠款期结束时，将可以获得来自 36 个中心的 900 多名儿童的 5 年数据。这将是有史以来规模最大的长期随访儿童肝脏接受者研究队列。该应用程序侧重于两个特定目标。第一个是开发和测试模型，预测肝移植后以及从上市时起的短期和长期患者和移植物存活率以及儿童的移植物功能。将评估基线因素，包括移植时的状态、手术因素和移植后并发症，并预计会根据原发疾病的诊断而有所不同。第二个目标是研究儿童肝移植和免疫抑制的长期后果，包括：肾功能、移植后淋巴增殖性疾病（PTLD）、心血管危险因素和生长障碍。 SPLIT 独特的样本将能够确定与长期免疫抑制相关的发病率，并确定这些发病的预测因子。 SPLIT 提供了一个既定的组织框架，并具有成功的记录。没有其他研究数据库可以解决 2000 年儿童保健法案 (HR4365) 规定的这些问题，并得到卫生与公共服务部和医学研究所的认可（联邦公报 98-8191，IOM 报告） ，1999）作为优先研究领域