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: 7492148
• 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/7492148
• 关键词: New; Investigation; Initiatives; Prevention; Complications

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.

这是波士顿儿童医院的全面thalassemia护理中心的新申请 （CHB），与亚特兰大儿童医院的Thalassemia计划的现场合作。两个都 在CDC领导的第一五年中，组织一直是分包商的参与者 Thalassia Project。该提案的目标是为预防和预防和 丘脑贫血并发症的管理。目标人群包括新英格兰的个人 在亚特兰大地区，依赖输血的beta thalassya（thal），alpha thal（纯合子 和血红蛋白H疾病），thal中间和相关疾病。塔尔的疾病负担是 高的。没有其他单一人类在一生中会接受许多新鲜的红细胞输血 依赖输血的患者（基本上是每月输血的生命）。因此，筛选 血液传播疾病是管理的关键部分。此外，输血还具有强制性铁 超负荷和与铁相关的心脏病，肝病和内分泌病是主要原因 该患者组的发病率和死亡率。良好符合口服铁螯合剂高度相关 生存率提高，但显然是友善的患者，以及未转移的thal Intermedia 患者可以发育多十年来，威胁生命的铁相关并发症，包括心力衰竭， 心律失常，肝硬化，糖尿病以及不育症和其他生殖内分泌疾病。面对 面对这些挑战，管理，监视和预防并发症的关键包括 定期在专家中心进行专家和专家的全面访问 血液安全监视和对铁及其后遗症的测试以及社区外展，协作 与患者支持小组，家庭和患者教育以及持续的社会心理评估以及 支持。 CHB的Thalassia计划已经活跃了40多年，并且领导了许多 该领域的发展，特别是在螯合疗法方面。该项目的具体目的是：（i） 在Thal中提供综合护理，筛选潜在的并发症，并实施策略 预防并发症； （ii）启动以DO五个模型的患者/家庭教育计划 血友病中心和CDC使用的策略来促进综合护理，血源疫苗 病毒，足够的螯合和血液安全监测。 （iii）与 由NIH赞助的Thalassia临床研究网络（CHB和Choa属于哪个； 研究人员Neufeld博士是TCRN指导委员会主席。）每个目标都包含一项研究 评估临床结果的组成部分，旨在为进一步研究产生假设，并 提供改善护理并减少并发症的机会。叙述： 该项目的目的是改善护理和长期健康状况 丘脑血症患者（Cooley的贫血）。这是加入的新应用 正在进行的几个美国大型thalassymia计划的合作努力， 儿童医院，波士顿作为主要地点，以及埃默里/儿童医疗保健 亚特兰大的合作网站。目标将通过提供 依赖输血的患者的全面医疗服务 地中海贫血，并筛查患者血传播感染和 该疾病的并发症与输血铁超负荷有关 治疗铁问题。我们提出了一项全国性的教育策略 类似于血友病社区发起的努力， 支持，全面护理。我们建议在下面驾驶一个新数据库 在芝加哥儿童纪念医院开发，以改善数据 从电子病历中抽象。最后，我们提出正式 与国家的Thalassexia临床研究网络合作 心脏，血液和肺部研究所，以利用正在进行的纵向筛查 在两个网络中。