Sickle Cell Disease Clinical Research Network

镰状细胞病临床研究网络

基本信息

  • 批准号:
    7407380
  • 负责人:
  • 金额:
    $ 19.19万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 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

项目摘要

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 multi- center 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-patientSickle 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.
费城儿童医院镰状细胞中心 (SCC-CHOP) 由国家资助的 临床镰状细胞病计划和 NIH 支持的综合镰状细胞中心。这 关于在镰状细胞病临床研究网络内建立临床中心的提案 (SCO-CRN) 结合了 SCC-CHOP 两个组成部分的优势 - 经验丰富的临床 21 岁以下儿童 SCO 的管理和护理协调以及长期临床记录 SCO 和其他血红蛋白病的研究。 SCC-CHOP 有着长期参与多项活动的历史 中心合作临床研究以及当地单一机构研究。拟议的临床中心 将能够利用 CHOP 的广泛资源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 名镰状细胞病临床项目和成人中招募患者 来自费城慈善医院的 110 名患者镰状细胞项目。 输血疗法是对已经出现严重症状的 SCD 患者最常用的干预措施。 临床并发症。然而,这种疗法仅在中风治疗中进行了测试。 组织良好的对照研究。此外,SCD 中越来越多地使用输血疗法预示着 未来铁过载的问题。我们提出了两项​​临床试验,两者都利用了我们的优势 在血红蛋白病的输血治疗和输血铁超负荷方面拥有丰富的经验。研究1 旨在比较部分换血 (PET) 与单纯输血加的效果 螯合疗法对 SCD 患者铁超负荷程度的影响此外,该研究旨在 确定长期铁负荷与肝脏和心脏铁负荷之间的关系 输血的SCD患者以及器官特异性铁超负荷与器官的关系 SCD 功能障碍。研究 2 将比较羟基脲 (HU) 疗法与慢性红细胞疗法的效果 输血(CRCT)疗法可降低患者复发性急性胸部综合征(ACS)的频率 患有镰状细胞病 SS (SCD-SS) 且无哮喘的患者,并对患有 SCD-SS 和哮喘的患者进行了类似的研究。 在随附的申请中,该中心还为 SCO-CRN 申请患者结果核心。 镰状细胞病是美国最常见的遗传性疾病之一。这些研究有 改善最严重患者的健康并延长其预期寿命的潜力 该疾病的并发症。

项目成果

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Kwaku Ohene-Frempong其他文献

Kwaku Ohene-Frempong的其他文献

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{{ truncateString('Kwaku Ohene-Frempong', 18)}}的其他基金

Patient Services Core
患者服务核心
  • 批准号:
    7538875
  • 财政年份:
    2007
  • 资助金额:
    $ 19.19万
  • 项目类别:
Incidence of Infection in Young Children with Sickle Cell Disease in Africa
非洲镰状细胞病幼儿感染率
  • 批准号:
    7538868
  • 财政年份:
    2007
  • 资助金额:
    $ 19.19万
  • 项目类别:
Scholar Program
学者计划
  • 批准号:
    7538876
  • 财政年份:
    2007
  • 资助金额:
    $ 19.19万
  • 项目类别:
Clinical Core
临床核心
  • 批准号:
    7538874
  • 财政年份:
    2007
  • 资助金额:
    $ 19.19万
  • 项目类别:
Sickle Cell Disease Clinical Research Network
镰状细胞病临床研究网络
  • 批准号:
    7224235
  • 财政年份:
    2006
  • 资助金额:
    $ 19.19万
  • 项目类别:
Sickle Cell Disease Clinical Research Network
镰状细胞病临床研究网络
  • 批准号:
    7060588
  • 财政年份:
    2006
  • 资助金额:
    $ 19.19万
  • 项目类别:
Oxyhemoglobin Desaturation and Vasculopathy in SCD
SCD 中的氧合血红蛋白饱和度降低和血管病变
  • 批准号:
    7288957
  • 财政年份:
    2005
  • 资助金额:
    $ 19.19万
  • 项目类别:
Oxyhemoglobin Desaturation and Vasculopathy in SCD
SCD 中的氧合血红蛋白饱和度降低和血管病变
  • 批准号:
    7276029
  • 财政年份:
    2005
  • 资助金额:
    $ 19.19万
  • 项目类别:
Oxyhemoglobin Desaturation and Vasculopathy in SCD
SCD 中的氧合血红蛋白饱和度降低和血管病变
  • 批准号:
    7110957
  • 财政年份:
    2005
  • 资助金额:
    $ 19.19万
  • 项目类别:
Oxyhemoglobin Desaturation and Vasculopathy in SCD
SCD 中的氧合血红蛋白饱和度降低和血管病变
  • 批准号:
    7484083
  • 财政年份:
    2005
  • 资助金额:
    $ 19.19万
  • 项目类别:

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