Comprehensive Sickle Cell Center

描述（由申请人提供）：辛辛那提综合镰状细胞中心提供了一个计划，将最先进的临床护理与前沿研究-基础，转化，临床，心理和健康结果-相结合，以改善镰状细胞病（SCD）患者的生活。一项合作的I/II期临床试验将炎症作为SCD的重要组成部分，以测试抗炎药物齐留通的长期使用。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。转化研究项目将建立在辛辛那提儿童医院医学中心基因治疗试验的独特基础设施和专业知识的基础上，以优化新型慢病毒载体，安全有效地纠正人SCD表型，并进行对开发I/II期基因治疗试验至关重要的临床研究。这些努力得到了临床中心的支持，该中心在辛辛那提为265名儿科患者和195名成人提供护理，并得到了附属研究中心的支持，这些研究中心拥有经验丰富的临床研究者，他们在俄亥俄州州立大学和南亚拉巴马大学为另外387名成人提供护理。设在CCHMC的病人服务中心通过协调和协助附属医院支持的大量专业人员，为病人和家属的心理社会需求提供服务。将与辛辛那提大学内科/儿科项目合作，开展一项创新项目，以促进基于“医疗之家”初级保健概念的从儿科向成人护理的过渡。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训新一代学者的坚定承诺。敷设总结：辛辛那提综合镰状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，以测试抗炎药物是否可以减轻SCD的炎症。两个项目使用基因转移到血液干细胞中，研究改善镰状细胞功能的方法。建议进行研究以提高SCD患者对治疗的依从性。这些项目得到了临床设施的支持，这些设施照顾近850名患者，他们可以选择参加临床研究。

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.