Prevention of the Complications of Thalassemia

预防地中海贫血并发症

• 批准号: 7682874
• 负责人: Alan R Cohen
• 金额: $ 20万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 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/7682874
• 关键词: Prevention; Complications; Thalassemia

Project Summary Abstract Patients with thalassemia major require regular red cell transfusions to circumvent their deficient or absent production of globin. As a consequence of these lifelong transfusion programs, affected patients have an intense and sustained exposure to blood-borne pathogens and may be a sentinel population for the recognition of new infectious agents in the blood supply. Regular transfusion programs also lead to other transfusion-related complications including red cell alloimmunization and iron overload. The latter is a particularly devastating complication of therapy and requires regular chelation therapy to prevent widespread organ damage and early death from cardiac failure or arrhythmias. Previously, the CDC- sponsored ¿Prevention of Complications of Iron Overload¿ developed a multi-center study of transfusion- related and other complications of thalassemia. The present study offers an opportunity to continue the regular collection of relevant clinical, demographic and genetic data to address the major goals of characterizing and preventing the complications of thalassemia. Using a variety of strategies and tools, The Thalassemia Program of the Children's Hospital of Philadelphia will continue to enroll patients in the surveillance projects, establish methods for consumer input and assess and meet the needs of unserved or underserved populations, provide multi-disciplinary, comprehensive care, identify patients with newly acquired transfusion-transmitted infections and other disease-related and treatment-related complications, and disseminate the study findings. In addition, our Thalassemia Program has identified two areas of new investigation: (1) the use of the longitudinal cohort to characterize the current mortality of patients with thalassemia major and differences in mortality related to treatment in or outside of a treatment center and (2) defining the current risk of postsplenectomy events, in particular sepsis and thrombosis. Overall, these studies will better define the optimal therapy of thalassemia and will also contribute to improvements in the treatment of other disorders requiring regular red cell transfusions. Project Narrative By characterizing in detail the complications of thalassemia, the proposed studies will not only improve the management of this inherited blood disorder but will also influence the treatment of all patients requiring short-term and long-term red cell transfusion programs.

项目摘要 重型地中海贫血患者需要定期输注红细胞，以避免其缺乏或缺乏。 生产珠蛋白。由于这些终身输血计划，受影响的患者有一个 强烈和持续暴露于血液传播的病原体，可能是一个哨兵人口的 在血液供应中识别新的感染因子。定期输血计划还会导致其他问题 输血相关并发症，包括红细胞同种异体免疫和铁超载。后者是一 特别是治疗的破坏性并发症，需要定期螯合治疗，以防止 广泛的器官损伤和心力衰竭或心律失常导致的早期死亡。此前，CDC- 赞助的预防铁超载并发症开展了一项输血多中心研究- 地中海贫血的相关和其他并发症。本研究报告提供了一个机会， 定期收集相关的临床、人口和遗传数据，以实现 地中海贫血的特征和预防并发症。使用各种策略和工具， 费城儿童医院地中海贫血项目将继续招募患者， 监督项目，建立消费者投入的方法，评估和满足得不到服务或 服务不足的人群，提供多学科，全面的护理，确定新的患者 获得性输血传播感染和其他疾病相关和治疗相关并发症， 并传播研究结果。此外，我们的地中海贫血项目还确定了两个新的领域， 研究：（1）使用纵向队列来描述患者的当前死亡率， 重型地中海贫血和与治疗中心内外治疗相关的死亡率差异， (2)确定脾切除术后事件的当前风险，特别是败血症和血栓形成。总的来说，这些 研究将更好地确定地中海贫血的最佳治疗方法，也将有助于改善地中海贫血的治疗效果。 治疗其他需要定期输注红细胞的疾病。项目叙述 通过详细描述地中海贫血的并发症，拟议的研究不仅将改善 这种遗传性血液疾病的管理，但也将影响所有需要治疗的患者的治疗。 短期和长期红细胞输注计划。