Comprehensive Sickle Cell Center

综合镰状细胞中心

• 批准号: 7343525
• 负责人: Clinton H Joiner
• 金额: $ 151.3万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-07-11 至 2012-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7343525
• 关键词: Comprehensive; Sickle; Cell; Center

DESCRIPTION (provided by applicant): The Cincinnati Comprehensive Sickle Cell Center offers a program that integrates state-of-the-art clinical care with cutting-edge research - basic, translational, clinical, psychological, and health outcomes - to improve the lives of those affected by sickle cell disease (SCD). A Collaborative Phase l/ll Clinical Trial addresses inflammation as an important component of SCD to test the chronic use of an anti-inflammatory drug, Zileuton. A Patient Services Research Project will assess barriers to adherence with treatment in pediatric patients and design individualized approaches to improve compliance. A Basic Science Research Project will use innovative methods of gene transfer and RNA interference to address the mechanisms of pathological dehydration of sickle cells and will explore the therapeutic potential of this approach. A Translational Research Project will build on unique infrastructure and expertise in gene therapy trials at Cincinnati Children's Hospital Medical Center to optimize novel lentivirus vectors for safe and efficacious correction of human SCD phenotype and to perform clinical studies critical for developing a phase l/ll gene therapy trial. These efforts are supported by a Clinical Core caring for 265 pediatric patients and 195 adults in Cincinnati, and by affiliated sites with experienced clinical investigators who care for an additional 387 adults at Ohio State University and the University of South Alabama. A Patient Services Core, based at CCHMC, serves the psychosocial needs of patients and families by coordinating and assisting a large staff of professionals supported by the affiliated hospitals. An innovative program to facilitate transition from pediatric to adult care based on the primary care concept of a "medical home" will be conducted in collaboration with the University of Cincinnati Internal Medicine/Pediatrics Program. Our dedication to the future of sickle cell patients includes a firm commitment to training a new generation of academicians through the Sickle Cell Scholars Program. Lay Summary: The Cincinnati Comprehensive Sickle Cell Center provides clinical care for sickle cell disease (SCD) and basic and clinical research to develop new treatments. A multi-center trial is proposed to test if an anti-inflammatory drug can reduce inflammation in SCD. Two projects use gene transfer into blood stem cells to study ways to improve the function of sickle cells. Research is proposed to improve the adherence of SCD patients with treatment. These projects are supported by clinical facilities caring for almost 850 patients, who have the option of participating in clinical studies.

描述（由申请人提供）：辛辛那提综合镰状细胞中心提供了一项计划，将最新的临床护理与尖端研究（基本，转化，临床，心理和健康成果）相结合，以改善受镰状细胞病（SCD）影响的人的生活。协作期L/LL临床试验将炎症作为SCD的重要组成部分，以测试抗炎药的长期使用Zileuton。患者服务研究项目将评估儿科患者遵守治疗的障碍，并设计个性化方法以提高依从性。一个基础科学研究项目将使用创新的基因转移和RNA干扰方法来解决镰状细胞病理脱水的机制，并将探索这种方法的治疗潜力。一个转化研究项目将基于辛辛那提儿童医院医疗中心基因治疗试验中的独特基础设施和专业知识，以优化新型的慢病毒载体，以实现对人类SCD表型的安全有效纠正，并进行临床研究，并为开发L/LL基因治疗期开发的临床研究至关重要。这些努力得到了针对辛辛那提265名儿科患者和195名成年人的临床核心照顾，以及有经验丰富的临床研究人员的附属地点，他们照顾俄亥俄州立大学和南阿拉巴马大学的387名成年人。位于CCHMC的患者服务核心通过协调和协助附属医院支持的大量专业人员来满足患者和家庭的心理需求。将与辛辛那提大学内科/儿科计划合作进行一项创新计划，以促进基于“医疗之家”的初级保健概念的过渡到成人护理的过渡。我们对镰状细胞患者未来的奉献精神包括通过镰状细胞学者计划培训新一代院士的坚定承诺。 LIE摘要：辛辛那提综合镰状细胞中心为镰状细胞疾病（SCD）以及基本和临床研究提供了开发新疗法的临床护理。提出了一项多中心试验，以测试抗炎药是否可以减少SCD中的炎症。两个项目使用基因转移到血液干细胞中来研究改善镰状细胞功能的方法。提出研究以改善SCD患者对治疗的依从性。这些项目得到了近850名患者的临床设施的支持，他们可以选择参加临床研究。