AN ALTERNATIVE APPROACH TO HYDROXYUREA THERAPY IN SICKLE CELL DISEASE

羟基脲治疗镰状细胞病的替代方法

基本信息

  • 批准号:
    5213965
  • 负责人:
  • 金额:
    --
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
  • 资助国家:
    美国
  • 起止时间:
  • 项目状态:
    未结题

项目摘要

Hydroxyurea appears to be a promising agent for the prevention of vascular occlusion in sickle cell disease (SCD). Current thinking holds that the clinical benefits of hydroxyurea are mediated by increases in Hb F concentration. Therefore, the current treatment strategy is to use maximally tolerated doses of hydroxyurea in order to achieve the maximimum Hb F response. However, several lines of evidence suggest that factors other than increased Hb F per se are also important. We hypothesize that a key effect of hydroxyurea is a marked reduction in the dense subpopulation of sickle RBC. We therefore propose to investigate whether titration of hydroxyurea dosage to this endpoint can achieve significant clinical benefits without the hematologic toxicity often associated with maximal doses, thus resulting in an improved risk:benefit ratio, better patient compliance and broader applicability to the patient population. We further propose to characterize the effects of this treatment strategy on a broad range of RBC, WBC and hemorheologic properties in SCD, and to determine its efficacy in treating several important vasoocclusive complications of SCD: painful crisis, pulmonary hypertension, renal insufficiency and leg ulcers. These investigations should provide an improved understanding of the physiologic effects of hydroxyurea in SCD, as well as give new insights into its clinical utility.
羟基脲似乎是一种很有前途的预防剂

项目成果

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CAGE S JOHNSON其他文献

CAGE S JOHNSON的其他文献

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{{ truncateString('CAGE S JOHNSON', 18)}}的其他基金

LABORATORY CORE
实验室核心
  • 批准号:
    4695093
  • 财政年份:
  • 资助金额:
    --
  • 项目类别:
ALPHA THALASSEMIA AND CLINICAL SEVERITY IN SIBLINGS WITH SICKLE CELL ANEMIA
阿尔法地中海贫血及其兄弟姐妹患有镰状细胞贫血症的临床严重程度
  • 批准号:
    4695088
  • 财政年份:
  • 资助金额:
    --
  • 项目类别:
SICKLE CELL CENTER--ADULT PROGRAM
镰状细胞中心--成人项目
  • 批准号:
    4695084
  • 财政年份:
  • 资助金额:
    --
  • 项目类别:
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