Research Initiatives for the Preventions of Complications of Thalassemia

描述（由申请人提供）：目前的项目将开发和维护南加州综合地中海贫血中心（SCCTC）地中海贫血患者的管理系统。 这将通过以下方式实现：（1）通过评估、监测、外展、教育、咨询和管理提供全面的医疗服务，以预防并发症;（2）参与血液安全监测和监测工作;（3）维持预防评估监测系统，以评估预防服务的效果;（4）与非专业组织合作，提供一致的预防信息，以预防并发症;（5）开拓研究思路和方法，并修改治疗和随访方案，以最大限度地实现最佳的医疗驱动结果。 南加州地中海贫血综合中心将通过以下方式实现这些目标：1）与其他CDC地中海贫血中心密切合作，参与国家一级的血液安全监测和研究项目，2）继续积极参与地中海贫血临床研究网络，3）在该中心制定外展和教育计划，并与北方加州地中海贫血中心合作，以及4）根据本地多学科综合护理模式的数据，维持和发展新的研究计划。 南加州综合地中海贫血中心的地中海贫血研究计划一般涉及输血铁过载的评估和管理以及通过磁共振成像测量组织铁。 基于从CDC综合护理模式收集的数据但由CDC项目外部资助的具体项目包括：1）心脏铁与心脏功能之间的关系，2）MRI用于测量肝脏铁的效用，3）地拉罗司去除心脏铁的功效，以及4）胰腺铁与胰腺功能之间的关系。 这些研究利用了南加州地中海贫血综合中心的独特专业知识，该中心的共同主任伍德博士是一名心脏病学家和生物医学工程师。 南加州地中海贫血综合中心将继续采用以下模式：对在地中海贫血患者多学科综合临床管理方面收集的数据进行分析，以修改治疗方法，并提出假设，以便对治疗和管理方法进行更正式的研究，从而减少地中海贫血患者的并发症，改善他们的健康。在洛杉矶儿童医院实施血液安全监测系统，以造福地中海贫血患者。疾病预防控制中心血液病分支将利用该系统提供的信息评估血液供应的安全性，并制定和监测旨在减少或预防地中海贫血并发症的干预措施的有效性。

DESCRIPTION (provided by applicant): The current project will develop and maintain systems for the management of patients with thalassemia at the Southern California Comprehensive Thalassemia Center (SCCTC). This will be accomplished by 1) providing comprehensive healthcare services to prevent complications through assessment, surveillance, outreach, education, consultation, and management; (2) participating in blood safety monitoring and surveillance efforts; (3) maintaining a prevention evaluation monitoring system to assess the efficacy of prevention services; (4) collaborating with lay organizations to deliver consistent prevention messages aimed at preventing complications; and (5) develop research ideas and methods to determine, measure, and modify treatment and follow-up protocols to maximize the achievement of optimal medical-driven outcomes. The Southern California Comprehensive Thalassemia Center will address these goals by 1) working closely with the other CDC thalassemia centers to participate in blood safety monitoring and research projects at a national level, 2) continuing active participation in the Thalassemia Clinical Research Network, 3) developing outreach and education programs at this center and by working collaboratively with the Northern California Thalassemia center, and 4) maintaining and developing new research initiatives based on data from our local multidisciplinary comprehensive care model. The thalassemia research initiatives from the Southern California Comprehensive Thalassemia Center in general are related to the assessment and management of transfusional iron overload and measurement of tissue iron by magnetic resonance imaging. Specific projects based on data collected from the CDC comprehensive care model but funded externally to the CDC program include 1) the relationship between cardiac iron and cardiac function, 2) the utility of MRI for the measurement of liver iron, 3) the efficacy of the deferasirox for the removal of cardiac iron, and 4) the relation between pancreatic iron and pancreatic function. These studies leverage the unique expertise of the Southern California Comprehensive Thalassemia Center of the Center co-director, Dr. Wood, who is a cardiologist and biomedical engineer. The Southern California Comprehensive Thalassemia Center will continue the model of using the analysis of data collected in the context of providing multidisciplinary comprehensive clinical management of patients with thalassemia to modify approaches to treatment and to generate hypotheses for more formal study of treatments and management approaches that will reduce the complications of patients with thalassemia and improve their health. Project Narrative To implement a blood safety monitoring surveillance system at Childrens Hospital Los Angeles to benefit persons with thalassemia. Information from this system will be used by the Hematologic Diseases Branch of CDC to assess the safety of the blood supply and to develop and monitor the effectiveness of interventions designed to reduce or prevent the complications of thalassemia.