Kaiser Permanente Autoimmune Disease Registry

描述（由申请人提供）：本申请响应了广泛的挑战领域（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，重点关注五种疾病：类风湿性关节炎（RA）、幼年炎性关节炎（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，可获得DNA用于未来的基因分型和功能测定。该登记研究对于了解生物疗法在个体水平上的有效性和比较有效性将是非常宝贵的。我们提出了以下具体目标：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IBD登记研究开发和证明的工具和方法添加RA、JIA、PsO、PsA和AS，将现有的KP炎症性肠病（IBD）登记研究扩展并重新构建为自身免疫性疾病登记研究。目标二：对随机抽取的1000名患者进行病历审查，其中包括RA、JIA、PsO、PsA和AS各200名患者，以使用经IBD工作验证的工具和方法确认疾病范围和严重程度。目标3：对于600例开始依那西普、英夫利西单抗或阿达木单抗治疗的成人RA患者的分层随机样本，确定治疗偏好，并在基线和6个月后使用电话调查研究不同药物在患者报告的疾病活动、功能状态、总体健康和工作障碍方面的有效性差异。目标4：在1996-2010年的15年期间，再次使用针对IBD开发和证明的方法和计算机程序，在RA和银屑病患者以及RA易感人群中，测量生物制剂改善临床结局的有效性。自身免疫性疾病登记将包括1996-2010年15年期间KP北方加州的所有RA、JIA、PsO、PsA和AS流行的成员。目前，有超过26，000名患有这些疾病的患者的信息可用，其中4393人是生物制剂使用者（3063人患有RA，79人患有JIA，665人患有银屑病，214人患有AS，372人患有多种适应症）。拟议的书记官处将为以下工作奠定基础：（1）在广泛的终点和患者的整个生命周期中测量许多有效性领域，（2）个性化确定有效性，以考虑患者的遗传特征，不良事件的基线风险和偏好，（3）阐明和解决儿童和其他弱势群体的特殊问题，（4）确定可以在卫生系统一级减轻的影响有效性的障碍，以及（5）估计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处将包括1996-2010年期间KP北方加州流行类风湿性关节炎、幼年类风湿性关节炎、银屑病、银屑病关节炎和强直性脊柱炎的所有成员。目前，有超过2.6万名患有这五种疾病的患者的信息可用，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.