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: 7901156
• 负责人: ELLIS J NEUFELD
• 金额: $ 15万
• 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/7901156
• 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地中海贫血综合护理中心的新申请 (CHB)，与亚特兰大儿童医院地中海贫血项目进行非现场合作。两者都有 各组织作为分包商参加了疾控中心领导的头五年 地中海贫血项目。这项提案的目标是提供评估和预防 地中海贫血并发症的处理。目标人群包括新英格兰的个人 在亚特兰大地区，患有输血依赖型β地中海贫血(Thal)，阿尔法地中海贫血(纯合子 和血红蛋白H病)、Thal中间体和相关疾病。THAL的疾病负担是 很高。没有任何一组人在一生中接受的新鲜红细胞输注数量 输血依赖Thal的患者(基本上是终身每月输血)。因此，筛选 血液传播疾病是管理的关键部分。此外，输血还伴随着必需的铁元素。 超负荷，以及与铁有关的心脏病、肝病和内分泌疾病是导致 该患者组的发病率和死亡率。良好的依从性与口服铁络合剂高度相关 存活率提高，但即使是明显螯合良好的患者，加上未输血的Thal中间介质 患者，可能会在几十年内发展为危及生命的铁相关并发症，包括心力衰竭， 心律失常、肝硬变、糖尿病以及不孕不育等生殖内分泌紊乱。面对面 面对这些挑战，管理、监测和预防并发症的关键包括 定期对专家中心进行全面访问，由精通Thal护理的专家和测试人员进行，目前正在进行 血液安全监测和铁及其后遗症检测，以及社区外展、合作 通过患者支持小组、家庭和患者教育以及持续的心理社会评估和 支持。CHB的地中海贫血项目已经活跃了40多年，并领导了许多 该领域的发展，特别是在螯合疗法方面。这项计划的具体目标是：(I) 在Thal中提供全面的护理，筛查潜在的并发症，并实施战略以 预防并发症；(Ii)仿效Do the Five发起病人/家庭教育计划 血友病中心和疾控中心用来促进全面护理的战略，血源性疫苗 病毒、充分的螯合作用和血液安全监测。(Iii)与该委员会建立研究合作 NIH赞助的地中海贫血临床研究网络(CHB和CHEA所属；和负责人 研究员Neufeld博士是TCRN指导委员会主席。)每个目标都包含一项研究 用于评估临床结果的组成部分，旨在为进一步研究产生假设；以及 提供改善护理和减少并发症的机会。叙述： 该项目的目的是改善保健和长期健康结果。 地中海贫血(库利氏贫血)患者。这是一个新的加入 正在进行的几个美国大型地中海贫血项目的合作努力 儿童医院，波士顿为主要地点，埃默里/儿童医疗保健 作为合作地点的亚特兰大。这些目标将通过提供 为输血依赖患者提供全面的医疗服务 地中海贫血，以及筛查血液传播感染和 这种疾病的并发症与输血铁超载和 来治疗铁的问题。我们提出了一项全国性的教育战略 类似于在血友病社区发起的努力，CDC 支持，全面照顾。我们建议在以下情况下试行一个新的数据库 芝加哥儿童纪念医院的发展，以改善数据 从电子病历中提取。最后，我们提出了形式化的 与国家地中海贫血临床研究网络合作 心脏、血液和肺脏研究所，利用正在进行的纵向筛查 在两个网络中。