Feasibility of a Novel Approach to Studying Early Knee Osteoarthritis: an Offspring Study.

研究早期膝骨关节炎的新方法的可行性：后代研究。

• 批准号: 9233041
• 负责人: Grace Lo
• 金额: $ 8.11万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-03-01 至 2018-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9233041
• 关键词: Absence of pain sensation; Adult Children; Age; Applications Grants; Arthritis; Body mass index; Clinic Visits; Consensus; Data; Degenerative polyarthritis; Development; Diagnosis; Disease; Etiology; Feasibility Studies; Funding; Genetic; Heberden' s Node; Internet; Intervention; Knee; Knee Osteoarthritis; Metabolic syndrome; Observational Study; Parents; Participant; Patients; Persons; Phenotype; Physical activity; Physical therapy; Pilot Projects; Prevalence; Privacy; Public Health; Radiology Specialty; Recruitment Activity; Replacement Arthroplasty; Research Design; Research Infrastructure; Risk Factors; Severities; Source; Structure; Surveys; Symptoms; Target Populations; Time; Universities; Venous blood sampling; base; clinical research site; cost; disability; effective intervention; effective therapy; follow-up; innovation; interest; modifiable risk; novel; novel strategies; offspring; prevent; public health relevance; willingness

 DESCRIPTION (provided by applicant): The public health burden of osteoarthritis (OA) is substantial. It is the most common form of arthritis, increasing in prevalence over time. The disability and cost related to the disease are high, and effective treatments for the condition are few, limited to physical therapy, analgesia, and then total joint replacement. Therefore, there is an urgent need to better understand the underlying etiology of this disease and to identify effective treatments for the condition. To date, no credible combined structure and symptom modifying intervention has been identified, perhaps because efforts have been focused on the wrong timeframe of disease. The window of opportunity to provide an effective intervention may only exist prior to the development of OA or early in the development of OA. Studying patients with early disease is challenging, however, because there is no consensus on case definition and symptom assessment in this group. An innovative approach to this problem, capitalizes on the substantial genetic component of knee OA. We will use the parental phenotype for case definition, where the disease is well-established, but our focus will be on risk factors in their offspring, where disease has either not yet occurred or is less well-established. Differences in modifiable risk factors of case versus control offspring could inform interventions that may prevent incident disease or progression of early disease. The Osteoarthritis Initiative (OAI) is an observational study where 4796 participants have been followed for 8 years and knee OA status will be available. Leveraging data already obtained about disease status and the existing OAI infrastructure to access parents of potential participants, provides a unique opportunity to deploy this proposed study. Although studying offspring of participants has the appeal of studying an earlier timeframe of disease, it also has its attendant challenges, including contacting and recruiting potential offspring participants within the framework of the OAI, while respecting their privacy and confidentiality. Therefore, in this grant application, we propose a pilot study to comprehensively assess feasibility of deploying a large scale offspring study and to obtain preliminary data to inform an R01 application to fund such a study. The two aims of this study are (1) to assess feasibility of deploying an offspring study within the OAI and (2) to assess whether it is possible to use known and exploratory offspring risk factors to predict the parental OA phenotype. Obtain preliminary data that would inform an R01 application to fund a larger scale study of the OAI Offspring. This study would break new ground by implementing a novel exploratory study design in a disease that is a major public health problem for which few effective treatments currently exist.

 描述（由申请人提供）：骨关节炎（OA）的公共卫生负担很大。它是关节炎最常见的形式，随着时间的推移，患病率增加。与疾病相关的残疾和费用很高，有效的治疗方法是 很少，仅限于物理治疗，镇痛，然后全关节置换术。因此，迫切需要更好地了解这种疾病的潜在病因，并确定有效的治疗方法。到目前为止，还没有确定可靠的结构和症状改善干预措施，可能是因为努力集中在疾病的错误时间框架上。提供有效干预的机会之窗可能仅存在于OA发展之前或OA发展早期。然而，研究早期疾病患者具有挑战性，因为该群体的病例定义和症状评估尚未达成共识。一种解决这个问题的创新方法是利用膝关节OA的大量遗传成分。我们将使用父母的表型的情况下定义，疾病是明确的，但我们的重点将是在他们的后代，疾病尚未发生或不太明确的风险因素。病例与对照后代可改变的风险因素的差异可以为可能预防早期疾病发生或进展的干预措施提供信息。骨关节炎倡议（OAI）是一个 一项观察性研究，其中4796名受试者已随访8年，将提供膝关节OA状态。利用已经获得的有关疾病状态的数据和现有的OAI基础设施来访问潜在参与者的父母， 这项拟议的研究。虽然研究参与者的后代具有研究早期疾病的吸引力，但它也有其随之而来的挑战，包括在OAI框架内联系和招募潜在的后代参与者，同时尊重他们的权利。 隐私和保密。因此，在这项拨款申请中，我们建议进行一项试点研究，以全面评估开展大规模后代研究的可行性，并获得初步数据，为R01申请提供信息，以资助此类研究。本研究的两个目的是（1）评估在OAI内开展后代研究的可行性和（2）评估是否可以使用已知和探索性后代风险因素来预测亲本OA表型。获得初步数据，为R01申请提供信息，以资助OAI后代的更大规模研究。这项研究将通过实施一种新的探索性研究设计来开辟新的领域，这种疾病是一个主要的公共卫生问题，目前几乎没有有效的治疗方法。