Thalassemia Clinic Research Network

地中海贫血临床研究网络

• 批准号: 6962127
• 负责人: SONJA M MCKINLAY
• 金额: $ 172.43万
• 依托单位: NEW ENGLAND RESEARCH INSTITUTES, INC.
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-07-01 至 2010-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6962127
• 关键词: biomedical registry /referral center; clinical research; clinical trials; cooperative study; data management; data quality /integrity; experimental designs; health care policy; human data; patient /disease registry; patient care management; thalassemia; tissue resource /registry

DESCRIPTION (provided by applicant): This is a five-year renewal application for the Thalassemia Clinical Research Network (TCRN). TCRN's mission is to accelerate research in the management of thalassemia. Syndromes encompassed by this mission include the ¿ thalassemias: homozygous or compound heterozygous plus Hemoglobin E/¿ thalassemia; and the alpha thalassemias: homozygous, hemoglobin H disease, and HbH/Constant Spring. The network is composed of four core U.S. sites, a data coordinating center, several large affiliated clinical sites in the U.S., Canada, and England, and several smaller regional "satellite" sites to the core centers, in a cooperative agreement with NHLBI. Key strengths of the TCRN include a demonstrated ability of the investigators and sites cooperatively to standardize patient assessment and care; to design, implement and conduct clinical trials with observational, interventional, and ancillary designs. The multicenter network has been crucial due to sample size considerations. A pivotal accomplishment of the initial grant period is the establishment and analysis of the TCRN Registry, from which subjects for trials are identified. The registry now includes over 800 patients from 22 sites, including ¿-thalassemia major (n=422), ¿ -thal intermedia (n=121), Hb E/ ¿ thal (n=103), transfusion-dependent a-thal major (n=9), HbH disease (n=l 05), and HbH/Constant Spring (n=44). The Specific Aims of the renewal grant are: (I) To perform interventional clinical trials in key areas of thalassemia care. Two trials are proposed. First, a randomized, controlled trial to examine the effect of deferoxamine alone v. deferoxamine plus deferriprone, on cardiac disease due to transfusional iron overload. Second, a randomized trial of arginine v. placebo for pulmonary hypertension, an important problem in thalassemia intermedia and other hemolytic states. (II) To provide an infrastructure for development, launch, and prompt completion of small, innovative trials in thalassemia. (Ill) To improve assessment of phenotype and clinical outcomes in thalassemia, in order to facilitate current and future clinical trials. This will be accomplished by two studies. The Thalassemia Longitudinal Cohort study, as well as a detailed study of iron-related organ damage, comparing measures of iron burden in the heart, liver and pancreas, to outcomes of iron-related organ dysfunction. Combined with the proposed clinical trials and the ability to perform detailed genotype/phenotype correlations, these improved phenotype and outcome measures are powerful tools to enhance knowledge about thalassemia clinical care, as envisioned by the NIH's 2003 Director's Roadmap.

描述（由申请人提供）： 这是Thalassexia临床研究网络（TCRN）的五年更新应用。 TCRN的使命是加快在丘脑贫血管理方面的研究。该任务所包含的综合症包括thalassemias：纯合或复合杂合子加血红蛋白E/¿和α丘脑菌素：纯合子，血红蛋白H疾病和HBH/恒定春季。该网络由美国，一个数据协调中心，美国，加拿大和英格兰的几个大型会员临床站点以及核心中心的几个较小的区域“卫星”站点组成。 TCRN的关键优势包括研究人员和站点协调一致的患者评估和护理的能力；设计，实施和进行临床试验，以观察性，介入性和辅助设计。由于样本量考虑，多中心网络至关重要。最初赠款期的关键成就是对TCRN注册中心的建立和分析，从中确定了试验对象。该注册表现在包括来自22个现场的800多名患者，包括�-丘陵症大（n = 422），€ - thal Intermedia（n = 121），HB E/¿续集授予的具体目的是：（i）在Thalassia Care的关键领域进行介入的临床试验。提出了两次试验。首先，一项随机，对照试验，用于检查单独的脱脂氧胺诉脱脂氧胺加脱氟替克隆对因输血过载而导致心脏病的影响。其次，精氨酸诉安慰剂的随机试验，用于肺动脉高压，这是thalassymia Intermedia和其他溶血态的重要问题。 （ii）提供用于开发，启动和迅速完成thalassexial的基础设施。 （ILL）为了促进当前和将来的临床试验，以改善对丘脑贫血的表型和临床结果的评估。这将通过两项研究来完成。 Thalalsia纵向队列研究以及对铁相关器官损伤的详细研究，比较了心脏，肝脏和胰腺中铁燃烧的测量与铁相关器官功能障碍的结果。结合了提出的临床试验以及执行详细的基因型/表型相关性的能力，这些改进的表型和结果度量是增强对Thalassia临床护理知识的强大工具，如NIH 2003年的2003年导演的路线图所示。