Clinical trial NCT02753777: Autoimmune Blistering Diseases Study
Official title: Prevalence and Clinical Severity of Autoimmune Blistering Diseases
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Pemphigus Vulgaris
Pemphigus Foliaceus
Bullous Pemphigoid
Lead sponsor: University of Pennsylvania
Sponsor class: OTHER
First posted: 2016-04-28
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

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 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.

Eligibility criteria: 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

Primary outcomes:
measure: Pemphigus Disease Area Index (PDAI)
timeFrame: one year
measure: Bullous Pemphigoid Disease Area Index (BPDAI)
timeFrame: one year
Secondary outcomes:
measure: Autoimmune Bullous Disease Quality of Life (ABQOL)
timeFrame: one year
measure: Treatment of Autoimmune Bullous Disease Quality of Life (TABQOL)
timeFrame: one year
measure: Autoimmune Bullous Skin Disorder Intensity Score (ABSIS)
timeFrame: one year
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania, Department of Dermatology
state: Pennsylvania
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania, Department of Dermatology
state: Pennsylvania
Study officials:
References: