Sickle Cell Disease Clinical Research Network

镰状细胞病临床研究网络

• 批准号: 7060588
• 负责人: Kwaku Ohene-Frempong
• 金额: $ 18.26万
• 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/7060588
• 关键词: asthma; blood disorder chemotherapy; blood transfusion; chelation therapy; clinical research; clinical trials; combination therapy; cooperative study; drug screening /evaluation; heart metabolism; human subject; human therapy evaluation; hydroxyurea; iron metabolism; iron poisoning; liver metabolism; magnetic resonance imaging; medical complication; patient oriented research; sickle cell anemia; sickle cell crisis; sickling inhibitor

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)： 费城儿童医院的镰刀细胞中心(SCC-CHOP)由国家资助的临床镰刀细胞疾病项目和NIH支持的综合镰刀细胞中心组成。这项在镰状细胞病临床研究网络(SCO-CRN)内建立临床中心的建议结合了SCC-CHOP的两个组成部分的优势--SCC-CHOP对21岁以下儿童的经验丰富的临床管理和护理协调，以及对SCO和其他血红蛋白疾病的长期临床研究记录。SCC-CHOP参与多中心协作临床研究以及本地单一机构研究的历史由来已久。拟建的临床中心将获得CHOP在临床护理协调和临床研究方面的广泛资源。我们的临床中心将从CHOP的705名患者的镰状细胞病临床计划和费城仁慈医院110名患者的镰状细胞计划中招募成年人。 对于患有严重临床并发症的SCD患者，输血治疗是最常用的干预措施。然而，只有在中风的治疗中，这种疗法才在组织良好的对照研究中得到测试。此外，SCD中越来越多的输血治疗预示着未来铁超载的问题。我们提出了两个临床试验，这两个试验都利用了我们在输血治疗和输血铁超载治疗血红蛋白疾病方面的丰富经验。研究1试图比较部分交换输血(PET)和单纯输血加螯合治疗对SCD患者铁超载程度的影响。此外，本研究还试图确定长期输血的SCD患者的全身铁负荷与肝脏和心脏铁负荷的关系，以及SCD的器官特异性铁负荷与器官功能障碍的关系。研究2将比较羟基脲(HU)疗法和慢性红细胞疗法的效果。 在无哮喘的镰状细胞病SS(SCD-SS)患者和有SCD-SS和哮喘的患者中，一项类似的研究表明，输血(CRCT)疗法可降低镰状细胞病SS(SCD-SS)患者的急性胸部综合征(ACS)复发频率。在随附的申请中，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病之一。这些研究有可能改善最严重疾病患者的健康并延长他们的预期寿命 疾病的并发症。(摘要结束)