Health Values and Minimally Important Differences in Systemic Sclerosis

描述（由申请人提供）：系统性硬化症（Scleroderma, SSc）是一种慢性风湿性疾病，没有有效的治疗或治愈方法，患者面临疼痛、毁容、残疾和无助感，这些都会影响其健康相关的生活质量（HRQoL）。该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员，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SSc患者的健康价值和生活满意度，并设计方法学上合理的SSc临床试验。这个

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.