New Investigation Initiatives for the Prevention of Complications of Thalassemia

预防地中海贫血并发症的新研究举措

• 批准号: 7427229
• 负责人: ELLIS J NEUFELD
• 金额: $ 17.5万
• 依托单位: BOSTON CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-09-30 至 2012-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7427229
• 关键词: New; Investigation; Initiatives; Prevention; Complications

DESCRIPTION (provided by applicant): This is a new application for a comprehensive Thalassemia care center, at Children's Hospital Boston (CHB), with off-site collaboration with the Thalassemia program of Children's Hospital of Atlanta. Both organizations have been participants as subcontractors in the first five year period of the CDC-led Thalassemia project. The objectives of this proposal are to provide assessment for, and prevention and management of complications of Thalassemia. The target populations include individuals in New England and in the Atlanta region, with transfusion-dependent beta Thalassemia (Thal), alpha Thal (homozygous and hemoglobin H disease), Thal intermedia, and related disorders. The burden of disease in Thal is high. No other single group of humans receives as many fresh red cell transfusions in a lifetime as patients with transfusion-dependent Thal (essentially monthly transfusion for life). Therefore, screening for blood borne diseases is a crucial part of management. In addition, with transfusions comes obligatory iron overload, and iron-related heart disease, liver disease, and endocrinopathies are major causes of morbidity and mortality in this patient group. Good compliance with oral iron chelators is highly correlated with improved survival, but even apparently well-chelated patients, plus non-transfused Thal intermedia patients, can develop over decades, life-threatening iron-related complications including heart failure, arrhythmia, cirrhosis, diabetes, as well as infertility and other reproductive endocrine disorders. Faced with these challenges, the keys to management, surveillance and prevention of complications include regular comprehensive visits at expert centers with specialists and tests skilled in Thal care, ongoing blood safety surveillance and tests for iron and its sequelae, as well as community outreach, collaboration with patient support groups, family and patient education, and continuing psychosocial assessment and support. The Thalassemia program at CHB has been active for more than 40 years, and has led many of the developments in the field, particularly in chelation therapy. The specific aims of this project are: (I) to provide comprehensive care in Thal, screen for potential complications, and implement strategies to prevent complications; (II) to initiate a patient/family education program modeled on the Do the Five strategy used by hemophilia centers and CDC to promote comprehensive care, vaccines for blood-borne viruses, adequate chelation, and blood safety monitoring. (III) To establish research collaboration with the NIH-sponsored Thalassemia Clinical Research Network (to which CHB and CHOA belong; and principal investigator Dr. Neufeld is chair of the TCRN steering committee.) Each aim contains a research component for assessment of clinical outcomes, aimed at generating hypothesis for further research, and providing opportunities to improve care and reduce complications.Narrative: The purpose of this project is to improve care and long-term health outcomes in patients with Thalassemia (Cooley's Anemia). This is a new application to join an ongoing cooperative endeavor of several large US Thalassemia programs, with Children's Hospital, Boston as the primary site, and Emory/Children's Healthcare of Atlanta as a collaborating site. The goals will be achieved by providing comprehensive medical services to patients with transfusion-dependent Thalassemia, and screening patients for blood-borne infections and for complications of the disorder, which are related to transfusional iron overload and to treatment of iron problems. We propose a nationwide educational strategy analogous to the efforts launched in the hemophilia community, with CDC support, for comprehensive care. We propose to pilot a new database under development at Children's Memorial Hospital in Chicago, to improve data abstraction from the electronic medical records. Finally, we propose formal collaboration with the Thalassemia clinical research network of the National Heart, Blood, and Lung Institute, to leverage the ongoing longitudinal screening in both networks.

描述（由申请人提供）：这是波士顿儿童医院（CHB）的全面Thalassemia护理中心的新申请，并与亚特兰大儿童医院的Thalassemia计划异地合作。在CDC领导的Thalassia项目的第一五年中，两个组织都是参与者作为分包商。该提案的目标是为地中海贫血的并发症提供评估，预防和管理。目标人群包括新英格兰和亚特兰大地区的个体，依赖输血的β地中海贫血（thal），alpha thal（纯合子和血红蛋白H病），thal Intermedia和相关疾病。 Thal的疾病负担很高。没有其他单一的人类在一生中接受许多新鲜的红细胞输血，而不是依赖输血的患者（本质上是每月每月输血的生命）。因此，筛查血液传播疾病是管理的关键部分。此外，输血带有强制性的铁超负荷，与铁相关的心脏病，肝病和内分泌病是该患者组发病和死亡率的主要原因。良好对口服铁螯合剂的良好性与改善的生存率高度相关，但显然，即使是卫生良好的患者，以及非转移的THAL中间媒体患者，也可以发展多十年来，具有威胁生命的铁相关并发症，包括心律失常，心律失常，心律失常，cirrhosis，cirrhosis，糖尿病，糖尿病，糖尿病以及其他生殖和其他复制性和其他复制性杂物。 面对这些挑战，管理，监视和预防并发症的关键包括在专家中心进行定期全面访问，并具有专家和熟练的THAL护理，持续的血液安全监测以及对铁及其后遗症的测试以及社区外展，与患者支持小组，家庭和患者教育以及继续心理社会的评估和支持和支持和支持和支持。 CHB的Thalassia计划已经活跃了40多年，并领导了该领域的许多发展，尤其是在螯合疗法方面。 该项目的具体目的是：（i）在Thal中提供全面的护理，筛选潜在的并发症，并实施防止并发症的策略； （ii）启动一项以血友病中心和CDC促进综合护理的五种策略为模型的患者/家庭教育计划，以促进血液传播病毒的疫苗，适当的螯合和血液安全监测。 （iii）与NIH赞助的Thalassedyclia Research网络（CHB和Choa属于其；以及Neufeld博士是TCRN指导委员会主席。）每个目标都包含评估临床成果的研究组成部分，旨在为进一步的研究和提供降低复杂性的假设。 该项目的目的是改善丘脑贫血患者（Cooley的贫血）患者的护理和长期健康状况。这是一项新的申请，以加入几个美国大型Thalassymia计划的持续合作努力，其中包括儿童医院，波士顿为主要地点，以及亚特兰大的埃默里/儿童医疗保健作为合作网站。这些目标将通过为依赖输血依赖性thal症的患者提供全面的医疗服务，并筛查患者的血传播感染和疾病并发症，这与输血铁超负荷和治疗铁问题有关。我们提出了一项类似于CDC支持在全国范围内的全国教育策略，类似于血友病社区的努力。 我们建议在芝加哥儿童纪念医院开发一个新的数据库，以改善电子病历的数据抽象。 最后，我们建议与国家心脏，血液和肺研究所的Thalassemia临床研究网络进行正式合作，以利用两个网络中正在进行的纵向筛查。