Health Values and Minimally Important Differences in Systemic Sclerosis

系统性硬化症的健康价值观和最不重要的差异

• 批准号: 8321754
• 负责人: DINESH KHANNA
• 金额: $ 12.34万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-09-15 至 2012-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8321754
• 关键词: Affect; Award; Behavioral; Benchmarking; Body Image; California; Characteristics; Chronic; Client satisfaction; Clinic; Clinical; Clinical Trials; Clinical Trials Design; Commit; Data Analyses; Data Collection; Decision Making; Educational process of instructing; Feeling; Future; Goals; Health; Health Sciences; Health Status; Healthcare; Immunology; Individual; Intervention; Interview; Joints; Lead; Life; Longitudinal Studies; Los Angeles; Measures; Mediating; Mentors; Modeling; Outcome; Pain; Pathogenesis; Pathway interactions; Patients; Perception; Population; Positioning Attribute; Public Health; Quality of life; Quality-of-Life Assessment; Recruitment Activity; Research; Research Design; Research Personnel; Rheumatism; Sample Size; Scleroderma; Social Environment; Social Sciences; Social support; Systemic Scleroderma; Time; Training; Universities; Value of Life; behavioral/social science; career; coping; cytokine; demographics; design; disability; effective therapy; health care delivery; health related quality of life; improved; insight; instrument; novel; optimism; patient oriented; programs; psychologic; satisfaction; skills; social; sound; therapy design

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.

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、 在相隔约12个月的两次数据收集过程中，研究人员对乐观、无助、身体形象、生活满意度和健康价值观进行了调查。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。

项目成果

Rapamycin versus methotrexate in early diffuse systemic sclerosis: results from a randomized, single-blind pilot study.

• DOI: 10.1002/art.24986
• 发表时间: 2009-12
• 期刊: ARTHRITIS AND RHEUMATISM
• 作者: Su, Tien-I Karleen;Khanna, Dinesh;Furst, Daniel E.;Danovitch, Gabriel;Burger, Christina;Maranian, Paul;Clements, Philip J.
• 通讯作者: Clements, Philip J.

Screening and therapy for malnutrition and related gastro-intestinal disorders in systemic sclerosis: recommendations of a North American expert panel.

系统性硬化症营养不良和相关胃肠道疾病的筛查和治疗：北美专家小组的建议。

• 发表时间: 2010
• 期刊: Clinical and experimental rheumatology
• 影响因子: 3.7
• 作者: Baron,Murray;Bernier,Paule;Côté,Louis-François;Delegge,MarkH;Falovitch,Glenda;Friedman,Gad;Gornitsky,Mervyn;Hoffer,John;Hudson,Marie;Khanna,Dinesh;Paterson,WilliamG;Schafer,Donna;Toskes,PhillipP;Wykes,L
• 通讯作者: Wykes,L

Validating and assessing the sensitivity of the Health Assessment Questionnaire-Disability Index-derived Short Form-6D in patients with early aggressive rheumatoid arthritis.

• DOI: 10.3899/jrheum.080959
• 发表时间: 2009-06
• 期刊: The Journal of rheumatology
• 作者: Amjadi SS;Maranian PM;Paulus HE;Kaplan RM;Ranganath VK;Furst DE;Khanna PP;Khanna D;Western Consortium of Practicing Rheumatologists
• 通讯作者: Western Consortium of Practicing Rheumatologists

Evidence-based management of rapidly progressing systemic sclerosis.

• DOI: 10.1016/j.berh.2009.12.002
• 发表时间: 2010-06
• 期刊: BEST PRACTICE & RESEARCH IN CLINICAL RHEUMATOLOGY
• 影响因子: 5.2
• 作者: Khanna, Dinesh;Denton, Christopher P.
• 通讯作者: Denton, Christopher P.

The minimally important difference and patient acceptable symptom state for the Raynaud's condition score in patients with Raynaud's phenomenon in a large randomised controlled clinical trial.

• DOI: 10.1136/ard.2009.107706
• 发表时间: 2010-03
• 期刊: Annals of the rheumatic diseases
• 影响因子: 27.4
• 作者: Khanna PP;Maranian P;Gregory J;Khanna D
• 通讯作者: Khanna D