Long-term Outcomes of Knee OA in the OAI Cohort

OAI 队列中膝关节 OA 的长期结果

• 批准号: 9125680
• 负责人: REBECCA D JACKSON
• 金额: $ 99.23万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-15 至 2022-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9125680
• 关键词: Address; Aging; Arthralgia; Clinic Visits; Clinical; Clinical Course of Disease; Cohort Studies; Comorbidity; Consequentialism; Data; Degenerative polyarthritis; Development; Disease; Disease Progression; Event; Gait speed; Goals; Health; Heterogeneity; Intervention; Investigation; Knee; Knee Injuries; Knee Osteoarthritis; Knowledge; Leg; Longitudinal Studies; Measures; Mental Depression; Musculoskeletal; Natural History; Obesity; Observational Study; Outcome; Outcome Assessment; Pain; Participant; Pathology; Patients; Performance; Persons; Phenotype; Phonation; Physical Performance; Physical activity; Population; Prevention; Prevention strategy; Probability; Psychosocial Factor; Quality of life; Research Personnel; Risk; Risk Factors; Scientific Inquiry; Severities; Severity of illness; Source; Stratification; Symptoms; Telephone; Time; clinical imaging; clinically significant; cohort; comorbid depression; coping; cost; design; disability; effective therapy; follow-up; illness length; improved outcome; knee pain; knee replacement arthroplasty; minimal risk; outcome forecast; outcome prediction; psychosocial; public health relevance; standardize measure

 DESCRIPTION (provided by applicant): Knee osteoarthritis (OA) is a major cause of pain, functional limitation and disability and among the most costly musculoskeletal conditions. A burgeoning population with knee OA and poor clinical outcomes in the absence of effective treatments are key drivers of the soaring rates and costs of knee replacement. Knee OA pathology and clinical outcomes typically unfold over decades and have a highly varied time course. It is a priority to understand the range of factors that contribute to poor and good outcomes in knee OA, but there are critical gaps in knowledge about the long-term course of the disease and its determinants. It is sensible to target prevention and treatment on those most at risk for poor outcomes. However, there is a paucity of longitudinal studies long enough (≥10 years) with frequent assessment using standardized measures to capture the full trajectory, range and variability of outcomes and investigate their determinants. In addition, investigation of these questions is hampered by baseline heterogeneity in knee OA severity and impact, which can be a source of imprecision and bias in observational studies of disease prognosis. Started in 2002, the OAI is a unique cohort study of 4796 persons with or at risk for knee OA that has uniform, rich clinical and imaging data from annual assessments and has been followed comprehensively for up to 8 years with good retention. We now propose to continue assessment of outcomes of knee OA for up to 15 years after baseline, primarily by phone and mail, an efficient and less burdensome approach that will yield more complete follow-up than clinic visits alone in this aging cohort. A brief clinic visit in a subset of participants will enale us to study performance measures of function as a long-term outcome. Our goal is to take advantage of this unprecedented opportunity for long-term follow-up to describe the full trajectory and probability of outcomes at different stages of disease, to identify vulnerable and protected phenotypes in its long-term course and investigate potentially modifiable predictors of these outcomes. Outcomes will span a range of health domains, including: knee-OA related (pain, functional limitation and performance, OA global impact, knee replacement), disability/participation and general health. The extended follow-up provides the opportunity for an analysis design that reduces the risk of common sources of bias and imprecision in studies of disease prognosis by allowing the use of changes from baseline to year 4 to define "inception events," such as the transition to more advanced stages of knee pain or structural OA or the development of favorable and unfavorable trajectories of risk factors (knee OA-related, physical performance, general health and psychosocial variables). These changes will then be evaluated for their association with subsequent outcomes occurring from year 5 to year 15 in analyses conditioned on a range of baseline knee OA-related and other covariates with a minimal risk of collider stratification bias. Using this approach we will investigate questions that can inform emerging strategies for prevention and treatment that are focused on improving outcomes in persons with knee OA. These include describing the probabilities of both poor and good long-term outcomes at different stages of knee pain and structural OA, comparing outcomes in those with recent transitions to more advanced stages with those whose course has been stable, and identifying the modifiable determinants of long-term outcomes at different stages of disease and whether these differ by stage, thus suggesting the need for tailored interventions to improve outcomes.

 描述（由申请人提供）：膝骨关节炎 (OA) 是疼痛、功能限制和残疾的主要原因，也是最昂贵的肌肉骨骼疾病之一。膝骨关节炎患者数量不断增加，且在缺乏有效治疗的情况下临床结果不佳，是膝关节置换率和成本飙升的关键驱动因素。膝关节 OA 的病理学和临床结果通常会持续数十年，并且时间进程变化很大。当务之急是了解导致膝骨关节炎预后不良和良好的一系列因素，但对该疾病的长期病程及其决定因素的了解还存在重大差距。针对那些最有可能出现不良结果的人进行预防和治疗是明智的。然而，缺乏足够长（≥10年）的纵向研究，并使用标准化措施进行频繁评估，以捕获结果的完整轨迹、范围和变异性并调查其决定因素。此外，调查 这些问题受到膝骨关节炎严重程度和影响的基线异质性的阻碍，这可能是疾病预后观察研究中不精确和偏差的根源。 OAI 始于 2002 年，是一项独特的队列研究，纳入了 4796 名膝关节骨关节炎患者或有膝关节骨关节炎风险的患者，该研究拥有来自年度评估的统一、丰富的临床和影像数据，并已进行长达 8 年的全面随访，且保留良好。我们现在建议在基线后长达 15 年的时间里继续评估膝关节 OA 的结果，主要通过电话和邮件，这是一种有效且负担较轻的方法，在这个老龄群体中，将比单独就诊产生更完整的随访。对一部分参与者进行简短的临床访问将使我们能够研究功能的表现测量作为长期结果。我们的目标是利用这一前所未有的长期随访机会来描述疾病不同阶段结果的完整轨迹和概率，识别长期病程中的脆弱和受保护表型，并研究这些结果的潜在可修改预测因子。结果将涵盖一系列健康领域，包括：膝关节骨关节炎相关（疼痛、功能限制和表现、骨关节炎全球影响、膝关节置换）、残疾/参与和一般健康。延长的随访为分析设计提供了机会，通过允许使用从基线到第4年的变化来定义“起始事件”，例如过渡到膝关节疼痛或结构性骨关节炎的更高级阶段，或风险因素有利和不利轨迹（膝关节骨关节炎相关、身体表现、一般健康和心理社会变量）的发展，从而降低疾病预后研究中常见偏差和不精确来源的风险。然后，将评估这些变化与第 5 年至第 15 年发生的后​​续结果的关联，分析以一系列基线膝关节 OA 相关和其他协变量为条件，碰撞分层偏差风险最小。使用这种方法，我们将调查一些问题，这些问题可以为新兴的预防和治疗策略提供信息，这些策略的重点是改善膝关节骨关节炎患者的预后。其中包括描述膝关节疼痛和结构性骨关节炎不同阶段长期结果不佳和良好的概率，将最近过渡到更晚期阶段的患者的结果与病程稳定的患者的结果进行比较，并确定疾病不同阶段长期结果的可修改决定因素以及这些决定因素是否因阶段而异，从而表明需要采取量身定制的干预措施来改善结果。