Monthly Archives: March 2009

Much has happened since our daughter, Amanda Staso, was featured in the Winter 2006 issue of the Quarterly. At that time, she was in eighth grade, and had just started treatment with Dr. Grant Anhalt after receiving a diagnosis of pemphigus vulgaris (PV) on her 14th birthday in February, 2006.

By the Summer of 2006, Amanda had almost tapered off the prednisone, but was still taking 2000 mg of CellCept a day. She was feeling pretty good, and was nervous, but excited to start high school. Then, in early August, she got sick. It took several doctors to figure out what was wrong, and it turned out that she had contracted the cocksackie virus. Things were confusing because this virus causes mouth sores, just like the PV. The virus resolved, but the mouth sores did not. The virus triggered a full blown flare.

Amanda was very sick. She missed her first two weeks of high school, and had to go back on high doses of prednisone and begin the long taper process all over again. What should have been a fun, exciting time in her life was consumed with physical and emotional turmoil. Physically, her face was swollen, she gained weight, suffered from migraine headaches. Emotionally, the side effects were just as bad. A very conscientious student, Amanda started high school academically behind. She felt isolated, scared, and angry. Yet, as she said in the paper she wrote in eighth grade, she lived life to the fullest, did not dwell on the negative, and soldiered on.

Amanda’s high school years have been very different than what I dreamed they would be before PV. I would never wish this disease on anyone, but I have to say that in some ways, her life is fuller, richer, and more balanced because of it. She is incredibly compassionate, perseverant, and is wise beyond her years.

Amanda is now a junior in high school, and has begun the process of choosing the path for the next phase of her life. She wants to go to college, and study either medicine or education. She takes 3 mg of prednisone every other day, and 2000 mg of CellCept every day. Side effects are minimal.

PV is something Amanda has, but it is not who she is. For her senior project, Amanda wrote, a paper entitled “The Case for Continued Public and Private Commitment to Rare Disease Research and Development” [Editor: See her paper on our website at]. Her research made us all aware that her disease is one of thousands of rare diseases, and how very lucky we are that hers is manageable and that we have the support of the IPPF to help us.

As Amanda’s mom, I never let my guard down. I mourn that her life is not care-free like a teenager’s should be. I live in fear of another flare. And I do worry about what will happen if and when she decides to have children and has to stop taking CellCept. But I take my lead from my strong, courageous daughter, try to live one day at a time, and be thankful for our blessings.

School Support
Open communication is the most important tool in managing the school situation. Each school year, I meet with all of Amanda’s teachers, counselors, and the school nurse to explain her situation and put in place mechanisms should she have to be absent for extended periods of time. Normally, a student has to miss two weeks of school before home schooling can begin. For Amanda, if she flares, the school has arranged for home schooling to begin immediately. Likewise, if she is having a minor flare, but still goes to school, I email her teachers so they know that all is not as it should be.

Family Support
Not only has Amanda’s disease affected all of us my husband, son, myself, and most of all, her but it has changed the course of all of our lives. It is thanks to our extended family and friends that we have coped as well as we have. I never hesitate to ask for help. Whether it is medical research, prayers, or just a hot cooked meal, we are surrounded by a network of people who love us and generously offer their special gifts to help us through the difficult times and celebrate the victories.

Medical Support
Since PV is so rare among the general population, and even rarer among young people, finding a dermatologist with experience treating it was not easy. We found Dr. Anhalt through the IPPF, and were quite willing to make the 3-hour trip from Pittsburgh to Baltimore to see him. My concern was what to do if Amanda had an emergency illness. I couldn’ go to Baltimore every time she got a cold. So I worked closely with Amanda’s pediatrician, Dr. Paul Trainer, and Dr. Anhalt, putting them in contact with each other, and with me, via email, so that we are a three-person team managing Amanda’s health.

This issue of the Quarterly focuses on our theme of Support. Support is one of the founding principles of the IPPF, stated in our Mission as: The IPPF provides direct access to innovative and effective support that:

  • promotes the very best health care,
  • improves quality of life,
  • stimulates community resources,
  • advocates for favorable government policies,
  • accelerates the pace of scientific discovery, and
  • is the world’s best source of information on P/P

Interestingly, this issue of our Newsletter became one of our most jam packed when we announced the theme. What I loved, as all the stories came in, was the many different ways people have found to provide support to each other through the IPPF.

Marc answers specific questions and cheers people on in the Forum; Mindy offers to use her expertise to promote donor development (Bio page 4); Amanda submits an English paper for a Senior project, on rare disease funding challenges, and takes the message to her high school (; our insurance coverage advocate, Brian, secured IVIg and/or Rituxan for two extra-challenging insurance coverage puzzles for IPPF members (see page 4), Alan and Terry made presentations at dental professional Seminars/Conferences to educate about P/P (pp. 5 and 6); Madeline and Danielle created new Wound Care information sheets (; Cathy has gathered more than $5,000 worth of items for auction at the Annual Meeting (; Rebecca finished the video check it out at (there’s both an educational one and the tear jerker); Badri and Miki edited d endless dry medical documents and fun new brochures; Will developed a News and Info page on the website (; and Janet has undertaken a rigorous Professional Health Coach Certification Program which will result in her accreditation by the American College of Sports Medicine, and will allow her to expand her coaching services to even more patients in need ( Many support us with direct donations.

When I was a Trauma Chaplain we had a few standard prayers that could be of service no matter the spiritual (or not) background of the person being ministered to. My favorite ended with, When the cold dark wind blows through the hollows of your soul, gather together and share what you know to be true.

It’s a scary, rainy night here in Sacramento as I write this; the economy is troubling and someone today was told they have pemphigus. All we have to comfort us on this crazy journey is the compassion shared by another. The magic thread, the uncommon bond, that draws each of you into the service of other P/P patients is the deep understanding that comes because you also have walked that path, and can share what you know to be true.

I look forward to the gathering together part, at the IPPF Annual Meeting, April 24-26, 2009.

One of our volunteers soliciting gifts for our Annual Meeting auction mentioned to me that several of the places she has contacted asked the question, ?Does this disease affect kids?? When they asked her that question, she thought, ?Yes, of course it affects kids.? She remembered how difficult it was on her daughter and her whole family when she was diagnosed. I immediately understood what she was talking about. Just because a child doesn?t have a disease, doesn?t mean that the ?kids? are not affected.

As we all know, people and companies are more inclined to give to organizations that involve diseases that affect kids. Also, getting any kind of media acknowledgement without cute kids is very difficult as well. And, although we do, unfortunately, know children and young adults who are diagnosed each year with one of our diseases, most of our patient base is people over 40.

We often overlook the effect that a disease like pemphigus or pemphigoid can have on a family. It is so important that not only should the person with our diseases get the support and care that they need, but that kids, spouses, significant others, grandparents, and close extended families have the opportunity to find sympathetic ears and hearts for their needs as well. Sometimes, diseases can be more devastating to the families than the patient themselves.

When I was diagnosed some 25 years ago, I was raising my 6 year old daughter alone. Although I had the support of family, my daughter was emotionally devastated. She had lost her father when she was too young to remember him, but nevertheless, he had made his imprint on her life. She started exhibiting out of character behavior which was very concerning, so I brought her to a child therapist. After 3 months of therapy, she revealed to the therapist that she was afraid that she would be an orphan, and who would love and take care of her? I told my dermatologist at the time, Dr. Clarke, what had transpired and she said, ?Bring her in. I?ll talk to her.? So I brought my daughter to see Dr. Clarke. Dr. Clarke went to her desk and took out her business card. She said to my daughter, ?You?re mother is not going to die. I want you to take this card. It has my name and my phone number on it. If you have any questions about anything, you just call me.? When we got home, she put the card in her clothes draw where it sat for many years. Her uncharacteristic behavior stopped. She never used the card, but just knowing that the card was there made all the difference in the world.

Our diseases affect everyone in our families and if anyone ever asks whether your disease affects children, the answer is always ?Yes!? Emotional issues can sometimes be more difficult than physical ones. At our Annual Meetings, we always have a Caregivers meeting during our Sunday event. We have a section on the website for Caregivers. From my observations, women seem to be more open to therapy than men, but there is nothing shameful in seeking help. Communication is so important in keeping families together.

The IPPF is pleased to welcome the newest member to our Board of Directors. Mindy Unger is a mother of three children, ages 19, 16, and 9 and is actively engaged in a number of philanthropic activities.

Mindy graduated from Binghamton University in 1982 with a B.S. in Accounting and received her CPA. She was an Auditor with Deloitte Haskins and Sells (now Deloitte and Touche), a Vice President at First Chicago and the Director of Accounting at Clarendon Ltd.

As a Board Member of Bet Torah, where she is currently Chair of the Ways and Means Committee, Mindy is responsible for more than doubling the fundraising efforts of previous years. She is a founding and active member of the Neshamot Fund, a Westchester Women’s Impact Philanthropy Group. She is also actively involved in various volunteer positions, from monthly visits to the Pleasantville Cottage School, a residential treatment center caring for 200 emotionally troubled youngsters, to being treasurer for multiple school plays.

Mindy is excited about using her financial, organizational, and fundraising skills to benefit the IPPF. Having the disease herself, she is most anxious for the IPPF to continue developing educational programs and research efforts.

In a number of P/P patients, standard treatment plans just don’t seem to work so other evidence-based treatments may be prescribed. IVIg or Rituximab  two expensive treatments  require approval by a patient’s medical plan. Many insurers choose to deny these treatments.

The IPPF has always been instrumental in supporting clinical and scientific meetings. In addition to supporting the American Academy of Dermatology’s (AAD) Symposium on Autoimmune Blistering Diseases, the IPPF is co-sponsoring the International Pemphigus Meeting in Bern, Switzerland in June 2009. This meeting will gather the finest minds in the world studying and researching pemphigus.

Subjects include: models in pemphigus, cell signaling, immune responses, unconventional therapies, and how stem cells might play a part. Janet Segall, IPPF Director of Patient Services & Education, will add a human face to the ramifications of these diseases among the scheduled scientific discussions.

The hope for better results comes from the work of people speaking and attending this meeting. The IPPF is proud to partner and co-sponsor such an important discussion and thank all those involved. The registration deadline is March 31, 2009. Anyone interested in attending this meeting should register at

Painful and scary symptoms no one seems to be able to explain or understand; a diagnosis you have never heard of before; understanding the actual illness; treatment options  most of which have not been FDA approved or may be considered experimental; side effects to consider; possible remission but no cure. . . Just trying to take in all of the above is totally overwhelming. Now, how do you explain it to others

Before you’ve even adjusted you will be asked by others how you are or what you have. How do you answer these questions You will try to find a relatively easy to understand explanation (e.g. My body’s immune system is not working correctly; it is attacking my body.)

I find that some people are even confused on the difference between autoimmune disease (AID) and acquired immune deficiency (as in AIDS). [See Terrys full article about AID vs. AIDS on our website at or call 916.922.1298] It is disturbing when others are scared that they will contract pemphigus or pemphigoid from us.

While there has been education about MS, Lupus, Arthritis and other autoimmune disorders, the more rare ones remain a mystery  and a scary one at that. From a psychological point of view all of this puts people with these rare autoimmune diseases at a distinct disadvantage. Now you need to cope with the fears of others as well as your own. Like most fears, they can often be addressed with a good dose of information.

Personally, I find it easiest to just say pemphigus is an autoimmune disease like Arthritis or Lupus. Since I have arthritic hands I can just put out my hand and say it is not a disease I can give anyone any more than I can actually give someone Arthritis. If people want more information, I usually just refer them to the website. Those who go learn a lot very quickly. Education is part of the mission statement of the IPPF. From the website you can get and give out key points to raise awareness and minimize fear in others.

If, as a patient, you feel compelled to further explain your particular autoimmune disease, a few copied/printed pages may suffice, or you can write a succinct explanation on index cards which can easily be carried around in a wallet (or use the sample at the left).

By the time this article is in print, I will have presented at the University of Pittsburgh Dental School for dentists receiving continuing education credits. I am not an expert on dentistry, but I understand the challenges of this disease and I have help and support from Janet and my fellow Board Members including Dr. David Sirois. I have made this opportunity happen through sheer persistence. I feel very strongly about patients getting their diagnoses in a more timely fashion.

I truly believe we are all capable of making a difference for others by giving our time and energy to help people — both doctors and our community — become more aware of diagnoses and challenges. Whether it is time, money, or other resources, every little bit helps! My hope and belief is that we can all touch others in positive ways and that whatever we are able to contribute of ourselves will make a difference.

A member of our Board of Directors and I attended the National Health Council’s (NHC) annual meeting February 11-13, 2009.The National Health Council sets operating Standards of Excellence for its members  such as the IPPF and over 50 of the nations leading patient advocacy groups  regarding budgets, policies for working with pharma companies and expenditures. The NHC advocates in Washington, D.C. and is the only organization of its kind that brings together all segments of the health care community to provide a united voice for more than 100 million people with chronic diseases and disabilities and their family caregivers.

A major problem physicians and patients face when deciding on the treatment of rare diseases such as pemphigus and bullous pemphigoid is deciding on the best course to take. The decision is difficult as it is best made on the basis of objective evidence that the selected treatment works, and that evidence is sparse. The best objective evidence that a treatment is effective is to conduct a randomized trial, in which two or more treatments are compared head-to-head. Unfortunately, as bullous diseases are rare (which is a good thing), very few randomized trials have been conducted.

But there is another problem, which is rarely appreciated. It is that even when a randomized trial is conducted, the conclusions may not be clear. The problem is illustrated by a recent randomized trial in which the effectiveness of mycophenolate mofetil (CellCept) was compared to that of azathioprine (Imuran) for the treatment of bullous pemphigoid. All patients were also treated with systemic steroids. The physicians need to be commended on conducting the trial – it is one of the very few randomized trial in this disease. Their conclusion was that mycophenolate mofetil was preferable to azathioprine because it was similarly effective but less toxic.

However, the opposite conclusion can be drawn from the same data that azathioprine is more effective and overall just as safe as mycophenolate mofetil. The time to induce complete remissions, the cumulative dose of steroids used, and the duration of remission were all better in patients treated with azathioprine than in those treated with mycophenolate mofetil. The average time to induce complete remission was 50% shorter, the time to induce remission in 100% of patients was 3 times less, the cumulative dose of steroids used was 15% less, and the duration of remissions was 30% longer in patients treated with azathioprine (n=38) that in those treated with mycophenolate (n=35). While these differences were not statistically significant, because the number of patients was small, the trend points to azathioprine as the more effective drug.

The authors also concluded mycophenolate mofetil was a safer drug because it was associated with fewer liver toxic effects. However, infections were more common in patients treated with mycophenolate and the overall number of serious side effects (grades 3 and 4) was similar in both groups; i.e. 11 serious adverse events in patients treated with azathioprine compared to 13 in those treated with mycophenolate mofetil.

What to make of these different interpretations of the same data That even the results of randomized trials need to be interpreted carefully. In this trial, where the same data can be interpreted differently, there probably was little difference in the efficacy or safety of azathioprine and mycophenolate mofetil as adjuvant therapies for the treatment of bullous pemphigoid. A more fundamental question is whether either of these drugs provides a benefit over the use of only systemic steroids in the treatment of this disease. A randomized trial is needed to answer this question.