Clinical Trial: Autoimmune Blistering Diseases Study

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

Official Title: Prevalence and Clinical Severity of Autoimmune Blistering Diseases

Brief Summary: 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.

Detailed Summary:

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 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
Sponsor: University of Pennsylvania

Current Primary Outcome:

  • Pemphigus Disease Area Index (PDAI) [ Time Frame: one year ]
  • Bullous Pemphigoid Disease Area Index (BPDAI) [ Time Frame: one year ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Autoimmune Bullous Disease Quality of Life (ABQOL) [ Time Frame: one year ]
  • Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL) [ Time Frame: one year ]
  • Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) [ Time Frame: one year ]


Original Secondary Outcome: Same as current

Information By: University of Pennsylvania

Dates:
Date Received: April 20, 2016
Date Started: April 2016
Date Completion: April 2019
Last Updated: November 10, 2016
Last Verified: November 2016