Sickle Cell Disease Clinical Research Network

镰状细胞病临床研究网络

• 批准号: 7224235
• 负责人: Kwaku Ohene-Frempong
• 金额: $ 17.73万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2011-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7224235
• 关键词: Adult; Adverse event; Age; Age-Years; Americas; Asthma; Cardiac; Caring; Chelation Therapy; Child; Chronic; Clinical; Clinical Management; Clinical Research; Clinical Trials; Controlled Study; Cross-Sectional Studies; Disease; Erythrocytes; Frequencies; Functional disorder; Funding; Future; Health; Hemoglobinopathies; Hepatic; Hospitals; Inherited; Institution; Intervention; Iron; Iron Chelation; Iron Overload; Life Expectancy; Liver; Magnetic Resonance Imaging; Measures; Methods; Oral; Organ; Outcome; Outcome Measure; Patients; Pediatric Hospitals; Philadelphia; Randomized Controlled Trials; Recording of previous events; Recruitment Activity; Recurrence; Resources; Sickle Cell; Sickle Cell Anemia; Testing; Transfusion; Weight; abstracting; acute chest syndrome; base; clinical effect; experience; hydroxyurea; improved; programs; stroke therapy

DESCRIPTION (provided by applicant): The Sickle Cell Center at the Children's Hospital of Philadelphia (SCC-CHOP) comprises the state-funded Clinical Sickle Cell Disease Program and the NIH-supported Comprehensive Sickle Cell Center. This proposal for the establishment of a Clinical Center within the Sickle Cell Disease Clinical Research Network (SCO-CRN) combines the strengths of both components of the SCC-CHOP - the experienced clinical management and care coordination of SCO in children up to 21 years of age and long record of clinical research in SCO and other hemoglobinopathies. The SCC-CHOP has a long history of participation in multicenter collaborative clinical studies as well as local single institution studies. The proposed Clinical Center will have access to the extensive resources at CHOP for both clinical care coordination and clinical research. Our Clinical Center will recruit from the 705-patient Sickle Cell Disease Clinical Program at CHOP and adults from the 110-patient Sickle Cell Program at Mercy Hospital of Philadelphia. Transfusion therapy is the most commonly used intervention in people with SCD who have developed major clinical complications. However, it is in only the management of stroke that this therapy has been tested in well-organized controlled studies. In addition, the increasing use of transfusion therapy in SCD predicts future problems with iron overload. We propose two Clinical Trials both of which take advantage of our extensive experience in transfusion therapy and transfusional iron overload in hemoglobinopathies. Study 1 seeks to compare the effect of partial exchange transfusion (PET) with that of simple transfusion plus chelation therapy on the degree of iron overload in patients with SCD. In addition, the study seeks to determine the relationship between total body iron burden and liver and cardiac iron loading in chronically transfused patients with SCD, and the relationship between organ-specific iron overload and organ dysfunction in SCD. Study 2 will compare the effect of hydroxyurea (HU) therapy to that of chronic red cell transfusion (CRCT) therapy in reducing the frequency of recurrent acute chest syndrome (ACS) in patients with sickle cell disease SS (SCD-SS) without asthma and a similar study in those with SCD-SS and asthma. In an accompanying application, This center is also applying for a Patient Outcomes Core for the SCO-CRN. Sickle cell disease is one of the most common inherited diseases in America. These studies have the potential to improve the health and prolong the life expectancy of patients with the most severe complications of the disease. (End of Abstract)

描述（由申请人提供）： 费城儿童医院（SCC-Chop）的镰状细胞中心包括国家资助的临床镰状细胞疾病计划和NIH支持的综合镰状细胞中心。这项关于在镰状细胞疾病临床研究网络（SCO-CRN）中建立临床中心的建议结合了SCC-Chop的两种组成部分 - 经验丰富的临床管理和SCO的经验丰富的临床管理和护理协调，最高21岁的儿童以及SCO和其他血红蛋白病的临床研究的长期记录。 SCC-Chop在参与多中心协作临床研究以及本地单一机构研究的历史悠久。拟议的临床中心将可以在CHOP中获得广泛的资源，以进行临床护理协调和临床研究。我们的临床中心将从Chop的705名患者镰状细胞疾病临床计划中招募，以及费城Mercy Hospital的110名患者镰状细胞计划的成年人。 输血疗法是患有主要临床并发症患者的最常用的干预措施。但是，只有中风的管理才在组织良好的对照研究中进行了测试。此外，在SCD中输血疗法的使用越来越多可预测铁超载的未来问题。我们提出了两项​​临床试验，这两者都利用了我们在输血疗法方面的丰富经验和血红蛋白病中的铁超负荷。研究1旨在将部分交换输血（PET）与简单输血以及螯合治疗对SCD患者铁超载程度的影响进行比较。此外，该研究旨在确定长期输注的SCD患者的总体铁负担和肝脏的总体铁负荷与心脏铁负荷之间的关系，以及SCD中器官特异性的铁超载与器官功能障碍之间的关系。研究2将比较羟基脲（HU）疗法的作用与慢性红细胞的疗法 在没有哮喘的镰状细胞病SS（SCD-SS）的患者中，输血（CRCT）治疗降低了复发性急性胸部综合征（AC）的频率，并且在患有SCD-SS和哮喘患者中进行了类似的研究。在随附的应用中，该中心还申请了SCO-CRN的患者结果核心。 镰状细胞病是美国最常见的遗传疾病之一。这些研究有可能改善健康状况并延长最严重的患者的预期寿命 疾病的并发症。 （抽象的结尾）