Improving Clinical Trial Recruitment and Outcome Measures in Bullous Pemphigoid

项目摘要/摘要 这项建议符合NIAMS临床观察研究R01 FOA的使命，以增强 大疱性类天疱疮患者的临床试验质量。这样做的总体目标是 建议以前瞻性的方式更好地描述疾病活动、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。 埃默里大学的费尔德曼博士在治疗和测量方面积累了多年的经验 来自美国东南部各地的一大群BP患者的疾病活动。 BP的许多临床测量工具最近才得到验证，还没有 以预期的方式严格应用，在没有这种情况下，设计一项未来的试验 是非常不切实际的。研究策略是每月对患者进行为期4个月的跟踪 将对客观和主观衡量标准进行评估的访问。由于英国石油公司是一家罕见的 为了提高入学率，有必要制定更有效的招募和保留战略。 AIM 1将前瞻性监测大疱性类天疱疮病区指数(BPDAI)的变化 在定义的4个月时间内，我们建议量化BPDAI总活动的变化 ≥50%作为临床疗效的基准终点。此外，还将对 将评估患者在4个月期间报告的结果，重点是 通过瘙痒生活质量测量ItchyQOL来衡量瘙痒。主要结果是 将改善ItchyQOL，并将此变化频率与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特异性 寿命测量的结果也将被用作次要结果。AIM 2将探索创新 为将来的试验改进患者招募/保留的方法，在这些试验中，我们计划利用 佐治亚州现有的远程皮肤病网络，允许对患者进行跟踪 在诊所就诊之间远程进行。主要目标是评估患者对以下方面的满意度 远程皮肤科就诊，因此无需长途跋涉 实地考察。次要目标包括评估基于照片的BPDAI评分的可行性 图像与面对面的比较。重要的是，这种远程皮肤病基础设施的发展 将促进未来的临床试验向前发展。来自该提案的数据将对以下项目至关重要 设计BP未来的临床试验，适当的重复测量，基准 改善患者参与度的客观和主观质量的结果。

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.