Comprehensive Sickle Cell Center

描述(由申请者提供)：辛辛那提综合镰状细胞中心提供一项计划，将最先进的临床护理与尖端研究-基础、翻译、临床、心理和健康结果-相结合，以改善受镰状细胞疾病(SCD)影响的患者的生活。L/111协作期临床试验将炎症作为SCD的重要组成部分，以测试抗炎药齐留通的长期使用情况。患者服务研究项目将评估儿科患者坚持治疗的障碍，并设计个性化的方法来提高依从性。一个基础科学研究项目将使用基因转移和RNA干扰的创新方法来解决镰状细胞病理性脱水的机制，并将探索这种方法的治疗潜力。翻译研究项目将建立在辛辛那提儿童医院医学中心独特的基因治疗试验基础设施和专业知识的基础上，以优化用于安全有效纠正人类SCD表型的新型慢病毒载体，并进行对开发L/11期基因治疗试验至关重要的临床研究。这些努力得到了辛辛那提265名儿科患者和195名成年人的临床核心的支持，以及俄亥俄州立大学和南阿拉巴马大学护理另外387名成年人的经验丰富的临床研究人员的附属网站的支持。患者服务中心设在CCHMC，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社会需求。将与辛辛那提大学内科/儿科项目合作，实施一项创新方案，以促进从儿科向成人护理的过渡，该方案以“医疗院”的初级保健概念为基础。我们致力于镰状细胞患者的未来，包括通过镰状细胞学者计划培养新一代院士的坚定承诺。报道摘要：辛辛那提综合镰刀细胞中心为镰状细胞病(SCD)提供临床护理，并进行基础和临床研究，以开发新的治疗方法。建议进行一项多中心试验，以测试一种抗炎药是否可以减轻SCD的炎症。两个项目使用基因转移到血液干细胞来研究改善镰状细胞功能的方法。建议开展研究以提高SCD患者对治疗的依从性。这些项目得到了临床机构的支持，这些机构照顾着近850名患者，他们可以选择参与临床研究。

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.