Kaiser Permanente Autoimmune Disease Registry

描述（由申请人提供）：本申请响应广泛的挑战领域（05）：比较有效性研究（CER）和特定的挑战主题05- ar -101：生物制剂在自身免疫性风湿病和皮肤病中的比较有效性，重点是五种疾病：类风湿关节炎（RA），幼年炎症性关节炎（JIA），银屑病关节炎（PsA），银屑病（PsO）和强直性脊柱炎（AS）。我们建议建立Kaiser Permanente （KP）自身免疫性疾病登记处，其中包含大量不同人群的全面临床信息，并可获得DNA用于未来的基因分型和功能分析。该注册表对于了解个体水平上生物治疗的有效性和比较有效性将是非常宝贵的。我们提出以下具体目标：目标#1：链接涵盖1996-2010年15年期间的临床数据库，通过使用为我们的IBD登记处开发和验证的工具和方法，通过添加RA， JIA, PsO， PsA和AS，将现有的KP炎症性肠病（IBD）登记处扩展和重新构建为自身免疫性疾病登记处。目标2：对1000名随机样本患者进行图表审查，其中包括200名RA， JIA, PsO， PsA和AS患者，以确认疾病的程度和严重程度，使用我们在IBD工作中证明的工具和方法。目的#3：对600名开始依那西普、英夫利昔单抗或阿达木单抗治疗的成年RA患者进行分层随机抽样，确定治疗偏好，并通过基线和6个月后的电话调查，调查不同药物在患者报告的疾病活动性、功能状态、整体健康和工作残疾方面的有效性差异。目标4：在1996-2010年的15年间，测量生物制剂在人群水平上改善RA和牛皮癣患者以及RA易感人群的临床结果的有效性，再次使用针对IBD开发并经过验证的方法和计算机程序。自身免疫性疾病登记处将包括1996-2010年15年间患有常见RA、JIA、PsO、PsA和AS的KP北加州所有成员。目前，有超过26,000名这些疾病患者的信息可用，其中4393名是生物制剂使用者（3063名RA患者，79名JIA患者，665名牛皮癣患者，214名AS患者，372名多指征患者）。拟议的书记官处将为以下工作奠定基础：(1)在广泛的终点范围内和患者的整个生命周期内测量有效性的许多领域；(2)根据患者的遗传概况、不良事件的基线风险和偏好对有效性的确定进行个性化；(3)阐明和解决儿童和其他弱势群体的特殊问题；(4)确定可在卫生系统层面减轻的有效性障碍。(5)估算和比较个人和社会的经济成本。Kaiser Permanente （KP）自身免疫性疾病登记将包括在1996-2010这15年间患有流行类风湿性关节炎、青少年类风湿性关节炎、牛皮癣、银屑病关节炎和强直性脊柱炎的KP北加州的所有成员。目前，可获得这五种疾病的26 000多名患者的信息，其中包括4393名生物制剂使用者。

DESCRIPTION (provided by applicant): This application responds to broad Challenge Area (05): Comparative Effectiveness Research (CER), and specific Challenge Topic 05-AR-101: Comparative Effectiveness of Biologics in Autoimmune Rheumatic and Skin Diseases, with emphasis on five diseases: rheumatoid arthritis (RA), juvenile inflammatory arthritis (JIA), psoriatic arthritis (PsA), psoriasis (PsO), and ankylosing spondylitis (AS). We propose to establish the Kaiser Permanente (KP) Autoimmune Disease Registry, containing comprehensive clinical information for a large, diverse population with access to DNA for future genotyping and functional assays. This registry will be invaluable for understanding the effectiveness and comparative effectiveness of biologic therapies at the level of the individual. We propose the following Specific Aims: Aim #1: Link clinical databases covering the 15-year period 1996-2010 to expand and reframe the existing KP Inflammatory Bowel Disease (IBD) Registry into an Autoimmune Disease Registry by adding RA, JIA, PsO, PsA, and AS using the tools and methods developed and proven for our IBD Registry. Aim #2: Perform chart review on a random sample of 1000 patients, including 200 each with RA, JIA, PsO, PsA, and AS to confirm disease extent and severity using the tools and methods proven for our IBD work. Aim #3: For a stratified random sample of 600 adult RA patients initiating etanercept, infliximab, or adalimumab treatment, determine preference for therapy and investigate differences in effectiveness across drugs in patient-reported disease activity, functional status, global health, and work disability using telephone surveys at baseline and 6 months later. Aim #4: Measure the effectiveness of biologics for improving clinical outcomes during the 15-year period 1996-2010 at the population-level among persons with RA and psoriasis and in vulnerable populations with RA, again using methods and computer programs developed and proven for IBD. The Autoimmune Disease Registry will include all members of KP Northern California with prevalent RA, JIA, PsO, PsA, and AS during the 15-year period 1996-2010. Currently, information is available for more than 26,000 patients with these diseases, of which 4393 are biologics users (3063 with RA, 79 with JIA, 665 with psoriasis, 214 with AS, and 372 with multiple indications). The proposed Registry will provide the foundation for: (1) Measuring the many domains of effectiveness across a wide range of endpoints and throughout the patient's lifespan, (2) individualizing the determination of effectiveness to account for the patient's genetic profile, baseline risk for adverse events, and preferences, (3) elucidating and addressing special problems in children and other vulnerable populations, (4) identifying barriers to effectiveness that can be mitigated at the level of the health system, and (5) estimating and comparing economic costs to the individual and society. The Kaiser Permanente (KP) Autoimmune Disease Registry will include all members of KP Northern California with prevalent rheumatoid arthritis, juvenile rheumatoid arthritis, psoriasis, psoriatic arthritis, and ankylosing spondylitis during the 15-year period 1996-2010. Currently, information is available for more than 26 thousand patients with these five diseases, including 4393 biologics users.