Health Values and Minimally Important Differences in Systemic Sclerosis

描述（由申请人提供）：系统性硬化症（硬皮病，SSc）是一种慢性风湿性疾病，无法有效治疗或治愈，患者科普疼痛、毁容、残疾和无助感，每种情况都会影响其健康相关生活质量（HRQoL）。候选人将成为加州大学洛杉矶分校（UCLA）大型硬皮病项目的一部分，并致力于改善SSc患者的HRQoL。候选人的长期目标是进行干预，以改善健康价值观和生活满意度与SSc的人，并设计方法学上健全的SSc临床试验。的 候选人已经招募了SSc，HRQoL评估和临床试验设计领域的顶尖专家来指导他，教他完成拟议研究所需的技能，并帮助他发展作为独立研究人员的职业生涯。该奖项的具体目标是：a）接受额外培训，并获得HRQoL，临床试验设计和数据分析方面的先进方法技能; B）定义一个概念模型，将生物医学观点与影响SSc患者健康结果的心理，行为和社会机制相结合;以及c）确定SSc患者认为具有临床意义的HRQoL工具评分的差异，也称为最小重要差异（MID）。两个具体的研究目标是：a）估计用于评估SSc的健康状况和健康价值指标的MID，和B）评估人口统计学、健康状况、心理特征和社会环境与SSc患者的健康价值观和生活满意度的关系。将从UCLA硬皮病诊所招募250例SSc受试者。目标1将为SSc研究中使用的HRQoL指标提供MID估计值，目标2将包括评估功能、社会支持、 乐观，无助，身体形象，对生活的满意度和健康价值观在两个数据收集会议相隔约12个月。纵向研究设计将告知自变量之间的假设关联（例如，健康状况）和健康价值观以及生活满意度。这些初步研究的结果将导致设计方法上合理的干预措施，以针对影响SSc患者HRQoL的非健康相关因素。与公共卫生的相关性：该研究将提供估计，以帮助未来硬皮病临床试验的样本量计算，确定哪些因素与生活满意度和生活价值或愿望相关，并将针对这些因素设计干预措施，以改善硬皮病患者的生活质量。

DESCRIPTION (provided by applicant): Systemic Sclerosis (Scleroderma, SSc) is a chronic rheumatic disease with no effective treatment or cure, in which patients cope with pain, disfigurement, disability, and feelings of helplessness, each of which can affect his or her health-related quality of life (HRQoL). The candidate will be part of a large Scleroderma Program at University of California, Los Angeles (UCLA) and is committed to improving the HRQoL of people with SSc. The long-term goals of the candidate are to conduct interventions to improve the health values and the satisfaction with life of people with SSc, and to design methodologically sound SSc clinical trials. The candidate has enlisted leading experts in the fields of SSc, HRQoL assessment, and clinical trial design to mentor him and teach him the skills he needs to complete the proposed research, and to help him develop his career as an independent researcher. The specific objectives for this award are: a) to take additional training and acquire advanced methodological skills in HRQoL, clinical trial design, and data analysis; b) to define a conceptual model that integrates the biomedical perspective with the psychological, behavioral, and social mechanisms that influence health outcomes of people with SSc; and c) to determine the difference in the score of HRQoL instruments that people with SSc consider clinically meaningful, also known as the minimally important difference (MID). The two specific research aims are: a) to estimate the MID for health status and health value measures used in evaluating SSc, and b) to assess the relationships of demographics, health status, psychological characteristics, and social environment to SSc patients' health values and satisfaction with life. Two hundred and fifty subjects with SSc will be recruited from the UCLA Scleroderma Clinic. Aim 1 will provide estimates of the MID for the HRQoL measures that are used in SSc research, and Aim 2 will consist of administering instruments assessing functioning, social support, optimism, helplessness, body-image, satisfaction with life, and health values in two data collection sessions separated by approximately 12 months. The longitudinal study design will inform the hypothesized associations between the independent variables (e.g., health status) and health values and life satisfaction. Findings from these preliminary studies will lead to the design of methodologically sound interventions to target non-health-related factors that impact the HRQoL of people with SSc. Relevance to public health: The study will provide estimates that will aid in the sample size calculation for future clinical trials in scleroderma, determine which factors are associated with life satisfaction and valuation or desirability of life, and will design interventions targeted toward these factors in order to improve the quality of life for people with scleroderma.