Improving Clinical Trial Recruitment and Outcome Measures in Bullous Pemphigoid

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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.