Washington Area Comprehensive Sickle Cell Center

描述(由申请人提供)：拟建的华盛顿地区综合镰刀细胞中心包括霍华德大学，这是美国首都的一所历史悠久的黑人教学机构；儿童国家医学中心，该地区为患有镰状细胞疾病(SCD)的儿童提供医疗保健的首屈一指的提供者；以及NHLBI血管医学分部，一个国际公认的领导SCD血管并发症研究的单位。拟建的中心还包括与尼日利亚扎里亚的艾哈迈杜·贝洛大学合作，该机构治疗许多尚未接受输血或羟基尿素的SCD患者。拟建中心的主要重点是提高对SCD血管并发症的认识、预防和治疗。该项目的组成部分包括： 1.一项中心间第二阶段研究：在系统收缩压为120-139毫米汞柱的SCD患者中，使用一氧化氮联合疗法与血管紧张素转换酶抑制剂或安慰剂进行比较，以预防肾功能障碍、肺动脉高压、中风和其他并发症。 2.一项中心内临床研究，通过超声心动图测量三尖瓣返流速度、艾滋病毒/艾滋病和疟疾的作用，以及基因多态在调节内皮功能和黏附的候选基因中的作用，来确定尼日利亚SCD患者中肺动脉高压的患病率。 3.一项患者服务研究项目，以确定患有SCD的婴幼儿与年龄和人口统计上匹配的对照组相比，发育问题的患病率。 4.一项翻译研究项目，旨在通过给NAD前体烟酸来提高高密度脂蛋白水平，从而提高载脂蛋白A-1水平，以改善内皮依赖的血管松弛。 5.行政核心。 6.临床核心，包括霍华德大学的成人和儿童镰状细胞诊所、儿童国家医学中心的儿科镰状细胞诊所、NHLBI的成人镰状细胞诊所及其相关人员。 7.以教育、社区推广、遗传咨询和诊断检测为重点的病人服务核心。 8.镰刀单元格学者组成部分。

DESCRIPTION (provided by applicant): The proposed Washington Area Comprehensive Sickle Cell Center includes Howard University, a historically-black teaching institution in the nation's capital, Children's National Medical Center, the premier provider of healthcare to children with sickle cell disease (SCD) in the area, and the Vascular Medicine Branch of NHLBI, an internationally recognized unit that leads in the investigation of the vascular complications of SCD. The proposed Center also includes collaboration with Ahmadu Bello University in Zaria, Nigeria, an institution that treats many SCD patients who have not yet been exposed to blood transfusions or hydroxyurea. The primary focus of the proposed center is to improve understanding, prevention and treatment of the vascular complications of SCD. The components of the project are: 1. An inter-Center Phase II Study of therapy with a nitric oxide-donating combination compared to an angiotensin converting enzyme inhibitor or placebo in preventing renal dysfunction, pulmonary hypertension, stroke and other complications in SCD patients with systemic systolic blood pressure of 120-139 mm Hg. 2. An intra-Center clinical study to determine the prevalence of pulmonary hypertension in Nigerian SCD patients using echocardiographic measurements of the tricuspid regurgitant jet velocity, the contribution of HIV/AIDS and malaria, and the role of genetic polymorphisms in candidate genes that regulate endothelial function and adhesion. 3. A patient services research project to identify the prevalence of developmental problems among infants and toddlers with SCD compared to age and demographically matched controls. 4. A translational research project that seeks to raise levels of HDL and therefore apoA-1 to improve endothelium-dependent vasorelaxation by administration of the NAD precursor, niacin. 5. An Administrative Core. 6. A Clinical Core that includes the adult and pediatric sickle cell clinics at Howard University, the pediatric sickle cell clinic at Children's National Medical Center, the adult sickle cell clinic at NHLBI, and their associated personnel. 7. A Patient Services Core focusing on education, community outreach, genetic counseling and diagnostic testing. 8. A Sickle Cell Scholar Component.