Yearly Archives: 2007

A Study to Evaluate the Intra-rater and Inter-rater reliability of the Pemphigus Disease Area Index (PDAI) and the Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) for Use by Dermatologists

To download and print the Informed Consent Form, click HERE
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Dr. Victoria Werth is looking for PV & PF patients with active oral or skin lesions to participate in an upcoming one-day study – August 4, 2007 at the University of PA, Philadelphia.  Dr. Werth and a team of doctors are working to finalize an outcome study of the IPPF Definitions Committee. When this study is completed, words to describe our disease may finally be defined medically – remission, control, etc., and used by dermatologists in describing our disease activity.

Anyone with more than three active lesions or one large active lesion (greater than 10 mm(3/8 inch)in size) can be part of the study. If you are with in driving distance or Amtrak distance from Philadelphia, this study welcomes you. There will be a $95 stipend and a parking validation for your involvement.

If you would like to be a part of this upcoming research project, read the rest of this page, download the Informed Consent Form, and contact Joyce Okawa, RN, or Matt Rose at (215) 898-0168 who will discuss the consent form and research study with you.


This study is meant to evaluate whether dermatologists, who are skin doctors, can use the PDAI easily and as well as another physician. In addition, this tool will be compared to the ABSIS to see which is superior. We hope that these research tools can be used by dermatologists for conducting research trials aimed at helping those with pemphigus. We need to show that some dermatologists who use the score will come to similar results when they examine and score the same patients.  It has been determined that at least 10 physicians are needed to complete these evaluations on a minimum of 10 subjects to validate these tools. Therefore you will have to be examined by about 10 physicians on one day. These physicians will use two or more of the above tools to examine and measure your skin disease activity, while you will complete the Patient Pain, Itch, and Skin Related Health questionnaire independently. They will examine your skin and discuss your disease with you. They will take pictures of your skin activity to use in the future for teaching or investigational purposes, as well as for publications related to this study.

The purpose of this trial is to (a) determine the ability of KC706 to prevent the appearance of new blisters and heal existing blisters, while maintaining stable doses of corticosteroids and/or immunosuppressants, in patients with pemphigus vulgaris (b) to determine the safety of KC706 and (c) to access the plasma levels of KC706.

No one asks to be “chosen” for chronic illness and/or pain.  There are, however, choices you can make which can either empower you or allow you to be a victim of circumstances.  Not feeling you are in total control does not mean you have no control but most of life is varying shades of grey. 

Rituximab (RTX) is anti-CD20 chimeric antibody that selectively targets B cells.  CD20 is a molecule that functions as an antigen for it.  CD20 is expressed on mature antibody producing B cells, but not on plasma cells.(1) The FDA has approved the use of Rituximab for the treatment of B cell lymphomas.(2) Since the pathogenic and clinical manifestations of PV appear to be antibody related, it could be hypothesized that eliminating the pathogenic antibody and the cells that produce it, might be more effective than general nonspecific immune suppression.  This is the rationale for using Rituximab in treating pemphigus vulgaris.  Indeed, success with the Rituximab has been observed in many autoimmune diseases which are mediated by antibodies such as systemic lupus erythematosus and immune thrombocytopenic purpura.(3)

Mucous Membrane Pemphigoid, also known as Cicatricial Pemphigoid, is a serious autoimmune blistering disorder that can result in blindness and other complications as the result of scarring of the mucous membranes.  To our knowledge, there have been 2 reported uses of Etanercept for MMP in the literature. 

The primary endpoint is to determine the number of days it takes to achieve a 50% reduction in blister count, and secondary endpoints include a reduction of baseline prednisone dose once the primary endpoint has been achieved. The trial will last for 16 weeks, encompasses 6 clinic visits (including screening), and patients will be compensated $200 for completion of the trial. Blood draws will take place 4 times during the course of the trial. 

To see if topical (applied to the skin) corticosteroid strengthens the skin of patients with Pemphigus vulgaris, thereby preventing blisters from forming.  The topical corticosteroid to be used in this study is called Clobetasol.  Clobetasol has been approved by the U.S. Food and Drug Administration (FDA) for use in people with skin diseases that are normally treated with oral corticosteroids, but not yet for Pemphigus vulgaris.