Autoimmune Blistering Diseases Study (AIBD)

Prevalence and Clinical Severity of Autoimmune Blistering Diseases

Pemphigus and bullous pemphigoid (BP) are severe autoimmune blistering diseases (AIBD) that pose a critical need for new therapeutic approaches. Clinical trials in pemphigus and BP will require the availability of validated disease severity measures that can be used to define primary outcomes.

Study Overview

• Status: Recruiting
• Conditions: Bullous Pemphigoid; Pemphigus Vulgaris; Pemphigus Foliaceus
• Intervention / Treatment: Other: Questionnaires

Detailed Description

The disease severity instruments for pemphigus and BP, the Pemphigus Disease Area Index (PDAI) and the Bullous Pemphigoid Disease Area Index (BPDAI), respectively, have the potential to capture changes in all grades of disease activity, including mild disease, and therefore represent a substantial improvement over simple lesion counts or measurements of affected body surface area. However, full validation of the PDAI and BPDAI for use in clinical trials will require additional and more extensive measurements that will enable us to classify patients correctly by disease severity and be able to define the minimal change in score that is clinically significant.

This database study will compare the Pemphigus Disease Area and Severity Index (PDAI) and the Bullous Pemphigoid Disease Area Index (BPDAI) against other disease severity measures, including the Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) and a physician's global assessment. Additionally, patients will be asked to rate their overall disease severity using a visual analogue scale, the patient's global assessment. Participants will also have the option to donate blood samples to our AIBD blood bank for immunologic and pathophysiologic studies.

The purpose of this database study will also be to evaluate quality of life (QoL) measures and correlate these QoL measures with disease severity in patients with pemphigus and BP. QoL is an important and independent component of disease impact on patients. This database study will use the following QoL measures: the SF-36, which is a nonspecific QoL measure; the Skindez-29 and the Dermatology Life Quality Index (DLQI), which are dermatology-specific instruments; the Autoimmune Bullous Disease Quality of Life (ABQOL) and Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) questionnaires, which are AIBD-specific tools. The results will be utilized in planning future clinical studies.

The AIBD study will be conducted only at Penn at this time. Data from the study will be entered into a password protected, web-based database that is used only at Penn.

• Study Type: Observational
• Enrollment (Estimated): 150

Contacts and Locations

• Study Contact: Name: Joyce Okawa, RN; Phone Number: 215-615-2960; Email: Joyce.Okawa@pennmedicine.upenn.edu
• Study Contact Backup: Name: Victoria P Werth, MD; Phone Number: 215-615-2940; Email: werth@pennmedicine.upenn.edu

Study Locations

• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Hospital of the University of Pennsylvania
      • Contact: Victoria P Werth, MD; Phone Number: 215-615-2940; Email: werth@pennmedicine.upenn.edu
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Hospital of the University of Pennsylvania, Department of Dermatology
      • Principal Investigator: Victoria P Werth, MD
      • Contact: Victoria P Werth, MD; Phone Number: 215-615-2940; Email: werth@pennmedicine.upenn.edu
      • Contact: Joyce Okawa, RN; Phone Number: 215-615-2940; Email: joyce.okawa@pennmedicine.upenn.edu
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • University of Pennsylvania, Department of Dermatology
      • Principal Investigator: Victoria P. Werth, MD
      • Contact: Victoria P Werth, MD; Phone Number: 215-615-2940; Email: werth@pennmedicine.upenn.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients over the age of 18 who have been diagnosed with pemphigus vulgaris, pemphigus foliaceus or bullous pemphigoid.

Description

Inclusion Criteria:

• Gender/Age: Males or females above 18 years old
• Diagnosis: pemphigus vulgaris, pemphigus foliaceus, bullous pemphigoid
• Subjects able to give informed consent

Exclusion Criteria:

• Patients who are under age 18 years.
• Patients without pemphigus or pemphigoid diseases
• Penn employees
• Penn students
• Cognitively impaired persons

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Pemphigus Disease Area Index (PDAI)	one year
Bullous Pemphigoid Disease Area Index (BPDAI)	one year

Secondary Outcome Measures

Outcome Measure	Time Frame
Autoimmune Bullous Disease Quality of Life (ABQOL)	one year
Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL)	one year
Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)	one year

Collaborators and Investigators

• Sponsor: University of Pennsylvania

Study record dates

Study Major Dates

• Study Start: April 1, 2016
• Primary Completion (Estimated): January 2, 2025
• Study Completion (Estimated): January 2, 2025

Study Registration Dates

• First Submitted: April 20, 2016
• First Submitted That Met QC Criteria: April 25, 2016
• First Posted (Estimated): April 28, 2016

Study Record Updates

• Last Update Posted (Actual): April 4, 2024
• Last Update Submitted That Met QC Criteria: April 2, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Autoimmune Diseases; Skin Diseases, Vesiculobullous; Pemphigoid, Bullous; Pemphigus
• Other Study ID Numbers: 824790

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO