Flare assessment for hidradenitis suppurativa

化脓性汗腺炎的耀斑评估

• 批准号: 10611884
• 负责人: Joslyn S Kirby
• 金额: $ 21.41万
• 依托单位: PENNSYLVANIA STATE UNIV HERSHEY MED CTR
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-04-01 至 2024-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10611884
• 关键词: Abscess; Address; Affect; Area; Benchmarking; Biological Markers; Chronic; Cicatrix; Clinic; Clinical Research; Clinical Trials; Clinical Trials Design; Consensus; Data; Data Reporting; Databases; Development; Diabetes Mellitus; Dimensions; Disease; Disease remission; Emotions; Familial Mediterranean Fever; Flare; Frequencies; Future; General Population; Goals; Health; Hidradenitis; Hidradenitis Suppurativa; Immunologics; Inflammation; Inflammatory; Knowledge; Laboratories; Lesion; Managed Care; Measures; Metabolic syndrome; Natural History; Nodule; Observational Study; Outcome; Outcome Assessment; Outcome Measure; Pain; Participant; Patient Outcomes Assessments; Patients; Periodicals; Personal Satisfaction; Prevalence; Qualifying; Quality of life; Recommendation; Relapse; Reporting; Research; Research Design; Research Personnel; Research Priority; Rheumatoid Arthritis; Sample Size; Severities; Severity of illness; Skin; Swelling; Symptoms; Systemic Lupus Erythematosus; Testing; Translational Research; Unemployment; Validation; Work; chronic inflammatory disease; clinical examination; clinical outcome assessment; design; disease natural history; effective therapy; experience; imaging study; improved; innovation; instrument; novel; novel therapeutics; observational cohort study; patient population; patient registry; programs; prospective; psychiatric comorbidity; skin disorder; standard of care; tool; treatment response; trial design

PROJECT SUMMARY The overarching goal of this proposal is to enhance the quality of clinical trials in hidradenitis suppurativa (HS) and to improve the current outcome measures for assessing treatment response by incorporating valid and reliable outcome measures of periodic worsening, or flares, of HS. This work is significant because it addresses an outcome deemed crucial by HS researchers and patients; will be the first to develop valid and reliable outcome measures for HS flare; and generate data on flare frequency and severity from an observational study in order to inform future study design. This work is innovative because it seeks to refine and expand the current paradigm for outcome assessment for HS to include flare activity, long-term control, and minimal disease activity, which are assessed in other chronic inflammatory diseases that periodically worsen. The overarching goal of this study is to develop, validate, and longitudinally assess criteria for flare in HS. This project will test the central hypothesis that HS flare can be validly and reliably measured using patient- and clinician-reported outcome measures. Two aims are proposed: 1) Validate the patient-reported Hidradenitis Suppurativa Quality of Life (HiSQOL) insturment for longitudinal HS flare assessment; 2) Validate the clinician-reported Severity and Area Score for Hidradenitis (SASH) instrument for longitudinal HS flare assessment. We will address these aims in prospective observational cohort studies incorporated into a quaternary HS referral clinic with an integrated patient registry where patients with HS will receive standard of care management. This study will address key gaps in our knowledge and approach, ultimately leading to effective clinical trials that include outcomes for HS that change the disease course. We have an established team of investigators with experience in HS clinical trials and outcome measure development and validation. All of our data is stored in a robust prospective database, with experience performing the necessary data entry, data extraction and analysis. The knowledge gained from the proposal will be invaluable to future clinical trials in HS.

项目摘要 该提案的总体目标是提高汗腺炎临床试验的质量 化脓性（HS），并改善目前的结果评估治疗措施 通过纳入有效和可靠的定期恶化或突发事件的结局指标， 的HS。这项工作意义重大，因为它解决了HS认为至关重要的结果 研究人员和患者;将是第一个为HS开发有效和可靠的结局指标的人 发作;并从观察性研究中生成关于发作频率和严重程度的数据，以便 告知未来研究设计。这项工作是创新的，因为它试图完善和扩大 HS结局评估的当前范例包括耀斑活动、长期控制和 最小的疾病活动，在其他慢性炎症性疾病中评估， 定期恶化。本研究的总体目标是开发，验证和纵向 评估HS中耀斑的标准。该项目将测试中心假设，即HS耀斑可以 使用患者和临床医生报告的结局指标进行有效和可靠的测量。两 目的：1）评估患者报告的化脓性汗腺炎的生活质量 （HiSQOL）用于纵向HS耀斑评估的仪器; 2）检查临床医生报告的 纵向HS耀斑的汗腺炎严重程度和面积评分（SASH）仪器 考核我们将在前瞻性观察性队列研究中阐述这些目标 纳入四级HS转诊诊所，并提供综合患者登记处， HS患者将接受标准护理管理。本研究将解决以下方面的主要差距： 我们的知识和方法，最终导致有效的临床试验，包括结果 改变疾病进程。我们有一个成熟的调查团队， 在HS临床试验和结局指标开发和验证方面的经验。我们所有的 数据存储在一个强大的前瞻性数据库中，具有执行必要数据的经验 数据提取和分析。从提案中获得的知识将是无价的 用于未来的HS临床试验