Assessment in Morphea for the Establishment of Non-Cutaneous Disease (AMEND)

硬斑病是否存在非皮肤疾病的评估 (AMEND)

• 批准号: 8120450
• 负责人: Heidi T Jacobe
• 金额: $ 11.55万
• 依托单位: UT SOUTHWESTERN MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-08-01 至 2013-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8120450
• 关键词: Accounting; Address; Adult; Affect; Age; Area; Attention; Autoantibodies; Autoimmune Diseases; Autoimmune Process; Award; Case-Control Studies; Characteristics; Child; Childhood; Clinical; Clinical Investigator; Clinical Research; Clinical Sciences; Clinical and Translational Science Awards; Cohort Studies; Collaborations; Collection; Commit; Complement; Confusion; Control Groups; Cutaneous; Data; Databases; Defect; Deformity; Dermatology; Development; Disease; Disease Management; Disease Outcome; Disease Progression; Enrollment; Environment; Epidemiology; Evaluation; Face; Family; Family history of; Frequencies; Funding; Future; Genetic; Genotype; Goals; Health Sciences; Immune; Immunogenetics; Institution; Joints; Knowledge; Lichen - organism; Limb structure; Measures; Medical center; Mentors; Monitor; Morphea; NCI Scholars Program; National Center for Research Resources; National Institute of Arthritis and Musculoskeletal and Skin Diseases; Natural History; Outcome; Pain; Patients; Phase; Phenotype; Predictive Factor; Prevalence; Principal Investigator; Race; Recording of previous events; Recruitment Activity; Research; Research Personnel; Research Project Grants; Resources; Risk; Sampling; Science; Scleroderma; Sclerosis; Serological; Serum; Skin; Skin Tissue; Source; Staging; Subcutaneous Tissue; Syndrome; Systemic Scleroderma; Systemic disease; Testing; Texas; Time; Training; Training Activity; Training Programs; Translational Research; United States National Institutes of Health; Universities; Variant; Wages; biobank; career; case control; clinical phenotype; cohort; demographics; design; disease characteristic; disease natural history; epidemiology study; experience; follow-up; functional disability; genetic epidemiology; genetic profiling; human leukocyte antigen gene; insight; interest; outcome forecast; patient oriented research; prognostic; prospective; response; sex; skills; systemic autoimmune disease; trait

DESCRIPTION (provided by applicant): The purpose of this application is to support my advanced training in patient-oriented research with the goal of becoming an independently funded investigator studying the epidemiology, immune profiles, and genetics of morphea in adults and children. I will achieve this objective with the help of a rich and supportive environment at the University of Texas Southwestern Medical Center (my institution) which includes: an experienced mentoring committee representing national experts in my fields of interest; a robust training nfrastructure through the Department of Clinical Sciences' Clinical Scholars Program; and an NIH NCRR linded Clinical and Translational Science Award (CTSA) supporting clinical research. These local resources are complemented by the University of Texas Health Science Center in Houston, also the recipient of a TSA award directed by one of my principle mentors, Dr. Frank Arnett. The training program detailed herein as well as the proposed research, represent the inaugural collaborative efforts between two geographically adjacent CTSA centers; which is a mandate and priority for CTSA awards. The Department of Dermatology has committed its full support to my training as a clinical investigator through its sponsorship of my participation in the Clinical Scholars Program (which requires salary support and 75% protected time to pursue training and a research project), access to the departmental clinical research center as well and its outstanding research coordinator, and the active dermatology practice for subject recruitment. The mentored research project that I will conduct in conjunction with my formal training in clinical research nvolves the epidemiology, autoantibody profiles, and immunogenetics of morphea. Morphea is characterized by thickening and hardening of the skin affecting both adults and children and has serious sequelae in terms of joint, limb, and facial deformities producing pain and functional impairment. At this time, there is controversy regarding the exact implications of morphea in terms of its risk for the development of other diseases, either autoimmune or systemic; if there are characteristic autoantibodies or HLA types associated with the disease that may predict disease activity or outcome; and the natural history of the disease. This confusion impairs the appropriate treatment of morphea patients, who suffer serious consequences from either over or under treatment. Having gained initial research experience in this area, I aim to conduct a case control study examining the frequency of systemic and autoimmune disease, presence of autoantibodies, and HLA associations in morphea patients vs. healthy and scleroderma (putatively the systemic form of morphea) controls. Morphea subjects enrolled in the first phase of the study will be asked to continue in a prospective cohort study for ascertainment of the natural history of the disease and if the characteristics measured at baseline predict these outcomes. Design and implementation of this study along with the training activities listed above will develop skills in epidemiological research as well as the translational research skills necessary to investigate the mechanistic aspects of my discoveries including immunogenetics, autoantibody characterization, and genetic epidemiology, thus launching a successful career as an independent clinical investigator.

描述（由申请人提供）：本申请的目的是支持我在以患者为导向的研究中进行的高级培训，目的是成为一名独立资助的研究人员，研究成人和儿童的流行病学，免疫学和遗传学。我将在德克萨斯大学西南医学中心（我的机构）的丰富和支持环境的帮助下实现这一目标，其中包括：代表我感兴趣的国家专家的经验丰富的指导委员会；通过临床科学系的临床学者计划进行了强大的培训NFRHAUSTURE；以及支持临床研究的NIH NIH NCRR临床和转化科学奖（CTSA）。这些本地资源得到了休斯敦德克萨斯大学健康科学中心的补充，这也是由我的一位主要导师弗兰克·阿内特（Frank Arnett）博士指导的TSA奖的获得者。本文详细介绍的培训计划以及拟议的研究代表了两个地理邻近的CTSA中心之间的开幕式合作。这是CTSA奖项的任务和优先事项。皮肤病学系通过赞助我参与临床学者计划（需要薪水支持和75％的受保护时间进行培训和研究项目），访问部门临床研究中心及其出色的研究协调员以及积极的皮肤病学招聘的培训，并为我的临床研究项目提供了75％的保护时间，并获得了75％的保护时间，并获得了75％的保护时间，并提供了75％的保护时间，并获得了积极的皮肤病学实践。我将在临床研究中进行的正式培训进行指导的研究项目，该研究的流行病学，自身抗体概况和Morphea免疫遗传学。形态的特征是影响成人和儿童的皮肤增厚和硬化，并且在关节，肢体和面部畸形方面具有严重的后遗症，从而产生疼痛和功能障碍。目前，关于morphea在其出现其他疾病（自身免疫性或全身性）的风险方面的确切含义存在争议。如果与该疾病相关的特征自身抗体或HLA类型可以预测疾病活动或预后；以及疾病的自然史。这种混乱会损害对形态患者的适当治疗，这些患者会因过度或治疗而遭受严重后果。在这一领域获得了最初的研究经验后，我旨在进行一项案例控制研究，研究系统性和自身免疫性疾病的频率，自身抗体的存在以及morphea患者中的HLA关联与健康和硬皮病（推定的形式形式）对照。将要求在研究第一阶段入学的形态受试者继续进行一项前瞻性队列研究，以确定疾病的自然史以及在基线时测量的特征是否预测了这些结果。这项研究的设计和实施以及上面列出的培训活动将发展流行病学研究的技能，以及研究我发现的机械方面所需的转化研究技能，包括免疫遗传学，自身抗体表征和遗传流行病学，从而成为独立临床研究者的成功职业。