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:至
- 项目状态:未结题
- 来源:
- 关键词:
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
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
数据更新时间:{{ journalArticles.updateTime }}
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
数据更新时间:{{ journalArticles.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ monograph.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ sciAawards.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ conferencePapers.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ patent.updateTime }}
CAGE S JOHNSON其他文献
CAGE S JOHNSON的其他文献
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
{{ truncateString('CAGE S JOHNSON', 18)}}的其他基金
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: