Category Archives: Clinical Trials

Background.  Pemphigus vulgaris (PV) and pemphigus foliaceus (PF) are autoimmune vesicobullous disorders with IgG autoantibodies directed against desmoglein (Dsg)1 and 3, which lead to intraepidermal acantholysis.

Aim.  To characterize the clinical and immunological profile of patients with PF or PV with umbilical involvement.

Methods.  In total, 10 patients (7 women, 3 men; age range 24–70 years, disease duration 3–16 years) diagnosed with either PV (n = 5) or mucocutaneous PF (n = 5) were assessed according to their clinical features, histopathology and immunological findings .

Results.  Erythema, erosions, crusts and vegetating skin lesions were the main clinical features of the umbilical region. DIF of the umbilical region gave positive results for intercellular epidermal IgG and C3 deposits in eight patients and for IgG alone in the other two. Indirect immunofluorescence with IgG conjugate showing the typical pemphigus pattern was positive in all 10 patients, with titres varying from 1 : 160 to 1 : 2560. ELISA with recombinant Dsg1 gave scores of 24–266 in PF and 0–270 in PV. Reactivity to recombinant Dsg3 was positive in all five patients with PV (ELISA 22–98) and was negative in all PF sera.

Conclusions.  All 10 patients with pemphigus with umbilical presentation had the clinical and immunopathological features of either PF or PV. This peculiar presentation, not yet completely elucidated, has rarely been reported in the literature. A possible explanation for this unique presentation may be the presence of either novel epitopes or an association with embryonic or scar tissue located in the umbilical-cord region.

Full article available at:

Neuromyelitis optica (NMO, also eponymously known as Devic’s disease) is an immune‐mediated demyelinating disease of the central nervous system that can lead to significant disability. Pediatric NMO is a rare disorder often reported after an infection. The authors report a 16year-old female patient with pemphigus foliaceus who developed subacute optic neuritis followed by cervical transverse myelitis. Restricted distribution of the lesions in the optic nerve and spinal cord was confirmed by ophthalmological evaluation and magnetic resonance imaging of the brain and spinal cord. She was started on intravenous methylprednisolone and then given a maintenance oral prednisone. Subsequently, she was treated with a nonsteroidal immunosuppressant, mycophenolate mofetil, with a target dose of 1000mg twice a day. Over the course of months, patient noted significant recovery of previous deficits and resolution of the cervical cord enhancement, expansion and cystic dilatation that was previously seen. This case is noteworthy for being the first patient reported with neuromyelitis optica associated with pemphigus foliaceus.



Randomized open comparative trial of dexamethasone-cyclophosphamide pulse and daily oral cyclophosphamide versus cyclophosphamide pulse and daily oral prednisolone

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in pemphigus vulgaris.

One of the newest treatments for pemphigus is intravenous immunoglobulin (IVIG). Immunoglobulins are one of the major classes of proteins in blood. IVIG is collected from normal blood donors, pooled, highly purified, and treated to destroy viruses and bacteria. IVIG is now used to treat a number of autoimmune diseases including pemphigus.

There are two particularly attractive features of IVIG as a treatment for pemphigus:

  1. It can rapidly control active pemphigus without resorting to increasing steroid doses, in many but not all patients.
  2. It is unique in being able to selectively decrease blood levels of pemphigus antibodies (the antibodies that cause the disease) without lowering the level of normal antibodies. This feature of IVIG is very desirable, and unique, as all other treatments for pemphigus interfere with the production of all antibodies — the good along with the bad — resulting in unwanted side effects.

IVIG appears to work by speeding the degradation or inactivation of all antibodies in blood  the good with the bad. Then, the normal antibodies are replaced by those present in the IVIG that is administered, whereas abnormal antibodies are absent in IVIG and so they are not replaced — only they remain reduced following IVIG.

Complicating this story is that a regulatory mechanism in the body maintains constant levels of each individual antibody in the blood. Decrease in blood level of any antibody (including the pemphigus ones) stimulates new production of that antibody, and a rebound in their levels in the blood. Thus, even though IVIG can decrease serum level of pemphigus antibodies, these will go right back up shortly after the procedure.

This rebound can be minimized, in animals, by administering a cytotoxic drug that blocks the cells that make new antibodies. This approach has been used in humans to improve the effectiveness of plasmapheresis, another procedure that lowers serum level of antibodies. Similarly, this approach should also improve the effectiveness of IVIG.
[box type=”orange”]Antibody: A protein produced by a B cell in response to a specific foreign substance. Antibodies are the ?soldiers? which protect us against bacteria and viruses and infections.

Cytotoxic drugs: Affect the growth and action of some cells that cause the joint pain, swelling, warmth, and damage of arthritis. Cytotoxic drugs work over a long period of time, however, patients may not notice much effect for the first several weeks or months of treatment.

Immunoglobulins: See Antibody

Plasmapheresis: Removal, treatment, and return of blood plasma from blood circulation.

* more definitions available here:[/box]

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.

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
(You will need Adobe Acrobat Reader to view this file)

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 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.