NCMHD Southeastern Exploratory Sickle Cell Center of Excellence

NCMHD 东南镰状细胞卓越研究中心

• 批准号: 8011095
• 负责人: Robert William Gibson
• 金额: $ 137.19万
• 依托单位: AUGUSTA UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-05-28 至 2013-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8011095
• 关键词: NCMHD; Southeastern; Exploratory; Sickle; Cell

DESCRIPTION: Sickle cell disease (SCD) is a genetic disorder of hemoglobin and afflicts ~110,000 African-Americans in the US. Because of its complex pathophysiology through chronic hemolytic anemia, microvascularocclusion, and a chronic inflammatory state, it affects multiple organ systems and leads to significant morbidity and organ damage as well as leads to frequent hospitalizations and health care encounters. During the past 35 years, primarily through research and patient care conducted by the NIH funded Comprehensive Sickle Cell Centers and some pivotal clinical trials, the life expectancy of patients with SCD has increased from the teens to mid- to late forties. This is still considerably shorter than that of African-Americans who do not have sickle cell disease and can be viewed as a major disparity even in this underserved minority population. While significant advances have been made in the understanding of the disease pathophysiology and in novel therapies through basic and translational research, these advances have been slow to be taken to clinical practice. The Southeastern Exploratory Sickle Cell Center of Excellence seeks to improve the care and quality of life of the SCD patient population by i) investigating the basic mechanism of action of a highly successful and effective hemoglobin F inducing drug, hydroxyurea, ii) identifying genetic variations underlying the frequency of pain, response to narcotics, and thus addressing the important issue of biologic/genetic bases of pain and its under treatment leading to the stigmatization of many SCD patients and its resulting disparity, iii) investigating the medical, social, and economic reasons for underutilization of hydroxyurea in SCD, iv) training primary care physicians with evidence based medicine in the care of patients with SCD, given the sobering reality that there will not be enough specialists in non-malignant hematology to meet the needs of the growing adult SCD population, and v) implementing innovative methods and concepts for the care of SCD patients in the ED and for transitioning from pediatric to adult care. Relieving the health disparity of SCD patients is the primary goal of this application.

描述：镰状细胞病 (SCD) 是一种血红蛋白遗传性疾病，困扰着美国约 110,000 名非裔美国人。由于其通过慢性溶血性贫血、微血管闭塞和慢性炎症状态的复杂病理生理学，它影响多个器官系统，导致显着的发病率和器官损伤，并导致频繁住院和医疗保健。在过去的 35 年里，主要通过 NIH 资助的综合镰状细胞中心进行的研究和患者护理以及一些关键的临床试验，SCD 患者的预期寿命从十几岁延长到四十岁中后期。这仍然比没有镰状细胞病的非裔美国人要短得多，即使在这个服务不足的少数群体中，也可以被视为一个重大差距。虽然通过基础和转化研究在对该疾病病理生理学的理解和新疗法方面取得了重大进展，但这些进展在临床实践中的应用进展缓慢。东南镰状细胞卓越中心致力于通过以下方式改善 SCD 患者群体的护理和生活质量：i) 研究高度成功且有效的血红蛋白 F 诱导药物羟基脲的基本作用机制，ii) 识别疼痛频率和麻醉药反应背后的遗传变异，从而解决疼痛的生物/遗传基础及其治疗导致的重要问题。 许多 SCD 患者的耻辱及其由此产生的差异，iii) 调查羟基脲在 SCD 治疗中未充分利用的医学、社会和经济原因，iv) 考虑到没有足够的非恶性血液学专家来满足不断增长的成人 SCD 人口的需求，对初级保健医生进行循证医学培训，以护理 SCD 患者，以及 v) 实施创新方法和概念 用于急诊科 SCD 患者的护理以及从儿科护理过渡到成人护理。缓解SCD患者的健康差距是该应用的首要目标。