Improving Clinical Trial Recruitment and Outcome Measures in Bullous Pemphigoid

项目总结/摘要 该提案符合NIAMS临床观察性研究R 01 FOA的使命，以增强 大疱性类天疱疮（BP）患者临床试验的质量。本报告的总体目标 建议以前瞻性方式更好地表征疾病活动性、瘙痒的变化 并捕获患者报告的结果，这些结果可用于设计更好的治疗试验。 博士埃默里大学的费尔德曼积累了多年的治疗和测量经验， 来自美国东南部各地的一大群BP患者的疾病活动。 BP中的许多临床测量工具最近才得到验证， 已经以前瞻性的方式严格应用，如果没有，设计未来的试验 是相当不切实际的。研究策略是在4个月内随访患者， 将评估客观和主观测量结果的访视。由于BP是一个罕见的 疾病，更有效的招聘和保留战略是必要的，以提高入学率。 目标1将前瞻性监测大疱性类天疱疮疾病面积指数（BPDAI）的变化 在4个月的规定时间内，我们建议量化BPDAI总活动的变化， ≥50%作为治疗疗效的基准临床终点。此外，具体变化 将评估4个月期间患者报告的结局，重点是 瘙痒，通过瘙痒生活质量测量ItchyQoL测量。主要结局 将改善瘙痒QoL，并将该变化频率与BPDAI进行比较 活动和瘙痒评分。最近证实的自身免疫性水疱病的具体质量 生命测量也将被用作次要结果。Aim 2将探索创新 改善未来试验患者招募/保留的方法，我们计划利用 格鲁吉亚州现有的远程皮肤病学网络， 在诊所访问之间进行远程访问。主要目标是评估患者对 远程皮肤科访问，从而提高了遵守，不必长途跋涉， 实地考察。次要目标包括根据照片评估BPDAI评分的可行性 与面对面相比。重要的是，这种远程皮肤病学基础设施的发展 将促进未来的临床试验向前发展。本提案中的数据对于以下方面至关重要： 设计未来的BP临床试验，采用适当的重复测量，基准 客观和主观质量的结果，提高患者参与度。

Project Summary/Abstract This proposal fits the mission of the NIAMS Clinical Observational Studies R01 FOA to enhance the quality of clinical trials for patients with bullous pemphigoid (BP). The overall objective of this proposal is to better characterize changes in disease activity, pruritus in a prospective manner and capture patient reported outcomes that could be utilized to design better therapeutic trials. Dr. Feldman at Emory University has accumulated years of experience in treating and measuring disease activity from a large group of BP patients referred from all over the Southeastern US. Many of the clinical measurement tools in BP have only recently been validated and have not been rigorously applied in a prospective fashion, in the absence of which, designing a future trial is quite impractical. The research strategy is to follow patients over a 4 month period with monthly visits in which both objective and subjective measurements will be assessed. Since BP is a rare disease, more effective recruitment and retention strategies are necessary to improve enrollment. Aim 1 will prospectively monitor changes in the Bullous Pemphigoid Disease Area Index (BPDAI) over a defined time of 4 months, and we propose to quantify the change in BPDAI total activity of ≥50% as a benchmark clinical endpoint for treatment efficacy. In addition, specific changes in patient reported outcomes over the 4 month period will be assessed with emphasis on change in pruritus as measured by the Itching Quality of Life measurement, ItchyQoL. The primary outcome will be improvement in the ItchyQoL and this changing frequency will be compared to BPDAI activity and pruritus scores. The recently validated autoimmune blistering disease specific quality of life measurements will also be utilized as secondary outcomes. Aim 2 will explore innovative methods to improve patient recruitment/retention for future trials in which we plan to leverage the existing teledermatology network across the State of Georgia to allow patients to be followed remotely between clinic visits. The primary goal is to assess patient satisfaction with teledermatology visits, thus improving compliance by not having to travel long distances for on- site visits. Secondary goals include assessing the feasibility of BPDAI scoring based on photo images compared to face-to-face. Importantly, development of this teledermatology infrastructure will facilitate future clinical trials moving forward. Data from this proposal will be essential for designing future clinical trials in BP with appropriate repeated measurements, benchmark outcomes for both objective and subjective qualities with improved patient engagement.