CHOP Pediatric Center of Excellence in Nephrology

CHOP小儿肾脏病卓越中心 我们建议在纽约建立一个儿科肾脏病学卓越中心（PCEN）。 费城儿童医院（CHOP）我们的主题是解决临床试验的障碍 肾脏疾病患儿的治疗。由于儿童肾脏疾病并不常见， 确定足够数量的患有特定疾病的儿童并招募他们参加试验， 难此外，根据肾脏疾病的临床表型的方法， 生长、发育、营养问题、心血管疾病风险因素、骨骼和矿物质 疾病往往因研究而异，质量控制也是可变的。研究专业知识 需要设计和分析，以从观测数据中得出适当的推论， 然而，设计临床试验时，肾脏病学的儿科中心往往缺乏这方面的专业知识。我们 将通过区域和国家临床合作应对这些挑战， CHOP、约翰霍普金斯和儿童医院的翻译研究人员参与了 PEDSnet。PEDSnet是一个由八个学术儿科健康中心组成的联盟， 为超过450万名儿童提供护理。PEDSnet建立了一个共同的机构审查 董事会，并已协调其参与中心的不同EHR系统，以创建一个 标准化的多机构数据网络，以促进有效开展 观察性研究、质量改进和临床试验。 CHOP-PCEN将有三个生物医学研究核心;一个设计和分析核心，一个 临床表型核心关注心血管疾病风险因素和营养/骨骼健康，一项学习 卫生系统核心，以及一个试点和可行性方案和一个浓缩核心。 我们包括2个利用核心的研究项目提案：“骨质量和血管健康 青少年泌尿系结石病”，“影像学生物标志物的推导和验证 第三个是纳入LHS核心的“儿童Skills结局” 和患有肾小球疾病的年轻人”。该研究基地由39个 研究者，他们是150多个资助项目的主要研究者、共同研究者或合作者 每年的直接费用总计超过3500万美元，其中超过1200万美元用于研究 由核心服务的潜在用户领导的儿科肾病相关项目。PCEN将 建立在CHOP和霍普金斯临床研究的坚实基础上，以及 宾夕法尼亚大学成人肾脏病学分部和临床流行病学中心， 生物统计学促进肾病儿童临床试验的设计和实施 疾病

CHOP PEDIATRIC CENTER OF EXCELLENCE IN NEPHROLOGY ABSTRACT We propose to establish a Pediatric Center of Excellence in Nephrology (PCEN) at the Children’s Hospital of Philadelphia (CHOP). Our theme is to address barriers to clinical trials implementation in children with kidney disease. As kidney disease in children is uncommon, identification of adequate numbers of children with specific disorders and recruitment for trials is difficult. Additionally, methods to clinically phenotype children with kidney disease in terms of growth, development, nutritional issues, cardiovascular disease risk factors, bone and mineral disorders often vary from study to study, and quality control is variable. Expertise in study design and analysis is needed to achieve appropriate inferences from observational data, and to design clinical trials, however, frequently pediatric centers in nephrology lack this expertise. We will address these challenges through a regional and national collaboration of clinical and translational researchers at CHOP, Johns Hopkins, and children’s hospitals participating in PEDSnet. PEDSnet is a consortium of eight academic pediatric health centers that collectively provide care for >4.5 million children. PEDSnet has established a common institutional review board and has harmonized the diverse EHR systems of its participating centers to create a standardized multi-institutional data network in order to facilitate the efficient conduct of observational research, quality improvement and clinical trials. The CHOP-PCEN will have three biomedical research cores; a Design and Analysis core, a Clinical Phenotyping Core focused on CVD risk factors and Nutrition/Bone health, a Learning Health System (LHS) Core, as well as a pilot and feasibility program and an enrichment core. We include 2 research project proposals utilizing the cores: “Bone Quality and Vascular Health in Adolescents with Urinary Stone Disease”, “Derivation and Validation of Imaging Biomarkers for CKD Progression” and a third, integrated into the LHS core, “Skeletal Outcomes in Children and Young Adults with Glomerular Disease”. The research base is comprised of 39 investigators, who are PI’s, co-investigators or collaborators on over 150 funded projects totaling over $35 million in annual direct costs, over $12 million of this funding is in research projects relevant to pediatric nephrology led by likely users of the core services. The PCEN will build upon the strong foundation of clinical research at CHOP and Hopkins, as well as the University of Penn Adult Nephrology Division and Center for Clinical Epidemiology and Biostatistics to catalyze the design and implementation of clinical trials in children with kidney disease.