Development of Outcome Measures for Hidradenitis Suppurativa

化脓性汗腺炎结局指标的制定

• 批准号: 9767755
• 负责人: Joslyn S Kirby
• 金额: $ 15.56万
• 依托单位: PENNSYLVANIA STATE UNIV HERSHEY MED CTR
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-09-01 至 2020-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9767755
• 关键词: Development; Outcome; Measures; Hidradenitis; Suppurativa

ABSTRACT Hidradenitis suppurativa (HS) is an important inflammatory disease, which affects approximately 1% of adults, and causes painful nodules, draining abscesses, and disfiguring scars that make walking, sitting, and working difficult or impossible. Progress to investigate HS is impeded by the lack of comprehensive outcome measures for use in trials and clinical care. Existing HS-specific disease severity tools are limited because signs of disease damage, such as discoloration and scarring, are either not captured or incorporated into a single score. In addition, some tools are impractical or not validated, reliable, or responsive. A disease-specific patient-reported outcome measure does not exist and given the impact of HS on patient well-being, it is vital to engage patients in outcome measurement. The goal of this proposal is to develop HS-specific measures to support accurate disease assessment. The specific aims of the proposed research are: (1) to test the hypothesis that a novel HS-specific patient-reported outcome measure will have substantial validity, reliability and responsiveness; (2) to test the hypothesis that a new provider-reported disease severity measure will have substantial validity, reliability and responsiveness. Patients with HS, age 18 or older, will be recruited from the PaTH network, an integrated clinical data research network across four Mid-Atlantic health systems, Penn State Hershey Medical Center, University of Pittsburgh Medical Center, Johns Hopkins Medical Center, and Temple Health System. The patients in the PaTH network are diverse; over 25% are non-white; 20% have public insurance or no insurance. The development and validation process will be based on the NIH PROMIS Instrument Development and Validation Scientific Standards. Briefly, we will include stakeholders, patients and providers, in the development and refinement of the tools. We will rigorously test the psychometric and measurement properties, including the reliability and validity, with the input of patients and providers. Finally, we will focus on evaluating the responsiveness of these novel patient- and clinician-reported outcome measures for HS.

摘要 化脓性汗腺炎（HS）是一种重要的炎性疾病，其影响约1%的成人， 并引起疼痛的结节、排水性脓肿和毁容性疤痕，使行走、坐着和工作 困难或不可能。由于缺乏全面的结果指标，调查HS的进展受到阻碍 用于试验和临床护理。现有的HS特异性疾病严重程度工具有限，因为 疾病损害，如变色和疤痕，要么不被捕获或纳入一个单一的， 得分此外，有些工具不切实际，或者未经验证、不可靠或响应速度不够快。疾病特异性 不存在患者报告的结局指标，考虑到HS对患者健康的影响， 让患者参与结果测量。本提案的目标是制定针对HS的措施， 支持准确的疾病评估。本研究的具体目的是：（1）检验 假设一种新的HS特异性患者报告结局指标将具有实质有效性、可靠性 和反应性;（2）测试假设，即新的提供者报告的疾病严重程度措施将有 有效性、可靠性和反应性。18岁或以上的HS患者将从 PaTH网络，一个跨四个中大西洋卫生系统的综合临床数据研究网络，宾夕法尼亚州 州好时医疗中心，匹兹堡大学医疗中心，约翰霍普金斯医疗中心，和 寺庙卫生系统。PaTH网络中的患者是多样化的;超过25%是非白人; 20%有 公共保险或没有保险。开发和验证过程将基于NIH PROMIS 仪器开发和验证科学标准。简而言之，我们将包括利益相关者、患者和 供应商，在开发和完善的工具。我们将严格测试心理测量， 测量属性，包括可靠性和有效性，与患者和提供者的输入。最后， 我们将重点评估这些新的患者和临床医生报告的结果的反应性， HS的措施。