A: The Aspreva CellCept study was a good example of how things work in the market of rare diseases. Company will not try to find new drugs unless there is at least 1 billion dollars a year in sales. For our diseases, it is very tough to do that. Other parts of the world may help because trials are more likely to take place where there is universal healthcare.
Q: Is there a registry?
A: The Definitions study sponsored by the IPPF and the Centric/IPPF HMP are the seedlings of a methodical record of disease symptoms, treatment protocols, cross-medicine experiments and legitimate clinical trials. It all starts here.
Q: Are there organizations that have been successful in setting up a registry on their own?
A: Yes, XP Foundation has its own registry.
A: Estimates have been made but there is no good grasp on the actually number.
Q: If an individual already has an autoimmune disease, is that person more susceptible of getting another autoimmune disease
A: There is an increased risk for people with one autoimmune disease to get another.
Q: How long will it take to get off steroids?
A: Very individual. Some people take months, some years. Goal is to reach 5 mg eod. Immunsuppressives can help.
Q: Are there people with high titers who don’ t have disease?
A: Not all antibodies are created equal. Antibodies need to target the right part of the cell. There are antibodies to parts of a cell that are non-pathogenic.
Q: Is there a maximum amount of prednisone one should take with CellCept?
A: No fixed amount.
Q: Are my chances greater to develop cancer from the immunosuppressive drugs?
A: Theoretically, there can be an increased risk of viral lymphoma. Data with Imuran comes from risks with patients with rheumatoid arthritis (RA), but patients with RA already have an increased risk. There probably is a smaller riskwith Cellcept, but it depends on how long on the drug and what the dose is. There is a small risk with Cytoxan as well, but higher than on Imuran.
Q: What about Rituxan?
A: Experts are excited about Rituxan because of the way it works. There is a lower chance of cancer. It doesn’t kill stem cells so the effects are not permanent.
Q: As steroids decrease will type 2 diabetes symptoms disappear?
A: There is prednisone-induced diabetes. Exercise will help the body use insulin properly. Anything higher than 126 (glucose) fasting, and 200 randomly is considered diabetes. Sometimes as you reduce steroids, sugar might go down. Insulin can be temporary. Take it if you need it. Januvia is a good drug if there is steroid-induced diabetes.
Q: How long does it take for prednisone to affect the organs?
A: It will affect the body almost immediately. The largest bone loss numbers will occur in the first 6 months.
Q: How often should a person have blood work?
A: If someone is on CellCept or Imuran a person should have monthly blood work. If you are on Cytoxan it should be weekly. Adequate monitoring is extremely important.
Q: I have been taking Imuran and prednisone for 8 years. Should I switch or change drugs
A: There is no guarantee if you switch drugs you will get off of prednisone. Low doses of steroids on alternative days is the goal. Not everyone can get off of drugs completely. Imuran and CellCept seem to be equal in effectiveness. One way to try and get off steroids would be to raise the dose of immunosuppressive.
Q: MMP diagnosis Does surgery cause vaginal scarring to get worse
A: Scarring cannot be reversed in MMP, only prevented. Surgery can make it worse in the area of MMP.
Q: In OCP, is frequent urination connected with drugs
A: Cytoxan if used most often for OCP. But Cytoxan can cause bladder problems. There are drugs that can lessen the toxicity in the blood if someone is on IV Cytoxan.
Q: Can a cold cause a flare
A: It can, but it doesn’t necessary mean you need to increase steroids use. You should wait a few days to see if it is only a temporary flare due to the cold.
Q: Are there any studies showing IVIG usefulness
A: IVIg causes a rapid reduction of antibodies. It is well described in the literature.
Q: Is Medic Alert helpful
A: Yes, if anyone is on steroids they should wear some form of medical identification.
Q: What would be your recommendation for general nutrition foods and/or supplements
A: One should watch high calorie intact. Lean protein is important. Basic multi-vitamins recommended. If someone isn’t eating, then something like Ensure would be good. Ask doctor to check Vitamin D levels.
Q: What about alternatives acupuncture, milk thistle, for drug side effects Can they help a patient
A: There is a basis for the exploration of alternative therapies. But, there is not enough data to give a definite opinion. Acupuncture is worth trying because it can help with stress. Red yeast extract may affect liver. Make sure you tell the doctor exactly what you might be taking so the physician can assess side effects.
Q: Should I stay out of the sun on immunsuppressives
A: Yes. Stay out of the sun if you are immunosuppressed.
Q: Should people with pemphigus not drink caffeine
A: There is an increased risk for bone loss for people on systemic steroids, caffeine may affect this too.
Q: Is it safe to take an over the counter sleep aid with prednisone
A: You should always talk to your doctor first. Occasional over the counter sleep aids are fine. Doctors can prescribe something to help you sleep.
Q: What are good topical treatments for PV in the gums
A: It is difficult for medicine to be retained in this area. Best things to try are the trays made from vinyl. Gel is probably the best medicine to use on the trays. It has shown to be helpful in MMP, but disappointing in PV. The disease really needs to be treated systemically.
Q: What about Swish & Spit
A: You should not swallow the drug because it will add a lot more steroids to your system, but it can be used on occasion.
Q: Environmental triggers vaccines correlated to disease
A: No hard evidence linking vaccines. Theoretically it could be possible, but there is no proof.
Q: Does the flu or shingles vaccine trigger the disease
A: It worthwhile getting vaccines because the benefits outweigh the risks. Do not take live vaccines.
Q: Do the diseases cause sleep apnea
A: The diseases themselves would not necessary interfere with sleep, but gaining weight from medications could have an effect and facilitate sleep apnea.
Q: Is there any connection between Menieres disease and pemphigoid
A: There does not seem to be any connection.
Q: Can pemphigus or pemphigoid cause plaque
A: No, but sore gums can cause plaque built up if the teeth are not taken care of. The key to long term success is keeping teeth clean.
Q: When gums recede can you replace missing gums
A: Yes, there are drugs that help, but you cannot get back all of it.
Q: Is there a relationship between tooth and gum disease and conditions such as cardiovascular disease
A: There is research going on to looking at a possible relationship.
Q: What do you do when missing a tooth
A: Not all teeth need to be replaced, absent esthetic problems.
Q: What can we, the patients, give back to the physicians or to the Foundation
A: Provide Information by taking part in the Health Management Program. Form a support group if there isn’t one. Spread the word to health care professions so they will be able to recognize the symptoms. Be willing to participate when doctors bring students into the room. Send us your ideas, do a local fundraiser, donate, and share your story.
Q: Have there been any clinical trials for MMP or OCP
A: At present, there are no trials but the need is there. It is harder to do trials because of the small number of people with the disease.
Q: In a double blind study, how do you make sure that a person would not get sick if they were getting the placebo
A: Most of the patients in pemphigus studies are usually on steroids, so most will not be in jeopardy because they are already on some medication.
Q: What are the benefits of being part of a trial
A: You would give back to community. Establish relationships with other patients. Contributors would get the drugs, laboratory testing, and follow-ups at no cost.
Q: How long does a clinical trial take
A: Some trials are now 3-5 months, but they really need to be longer, the cost can be many of hundreds of thousands of dollars.
The Journal of the European Academy of Dermatology and Venereology (JEADV) printed, in its Letters to the Editor section, a case study from the Department of Dermatology, Valencia University General Hospital, in Spain. Pemphigus Vulgaris Associated with Cocaine Snorting. It was reported that a patient was admitted to the hospital with severe erosions on his body and diagnosed with pemphigus vulgaris. The patient was given 90 mg of prednisone and sent home. His disease did not subside so they added 1 gram of CellCept. The patient’s disease did not get better.
After a toxicology report was positive for cocaine, the patient admitted to being addicted to the drug. After management of his addiction began, the patient’s pemphigus rapidly improved. The patient had 2 severe relapses while on 60 mg of prednisone and 1 g daily of CellCept. The PV relapses coincided with his continued use of cocaine.
Because of the sequence of events, the authors concluded that cocaine consumption might have been a factor in the development of disease process. They suggested that physicians should be aware of the possibility of the use of recreational drugs among certain patients, and that because of a lack of coordinated efforts between the disease state and the addiction, these patients are more likely to relapse back into their drug habits and cause the worsening of their disease.
When it comes to action, this group definitely puts the pedal to the metal… I am so grateful to all of you that offered me input about Dr. Grant Anhalt, getting around the Baltimore area, lodging suggestions, and most of all encouragement. When I was flying the friendly skies, all of you were with me. I could feel your love, support and cheering me on as I approached the city of Baltimore. It’s difficult enough finding a local doctor, giving him your trust and praying that his treatment will be successful. Traveling 1500 miles away is a totally different story. Hopefully, this story will have a happy ending. I was very impressed with Dr. Anhalt, his staff and the Johns Hopkin’s facility in Baltimore, Maryland. His knowledge, enthusiasm, and willingness to move on to Plan B made the trip all that I had hoped it would be. I am now on Cellcept and awaiting my first infusion of Rituxan on June 6th in my home town of Lake Charles, Louisiana. Dr. Anhalt is always willing to share his expertise with other physicians and responds to email in a timely manner. My local rheumatologist will be consulting with Dr. Anhalt, so I feel confident in the new treatment plan. I want to once again thank all of you for being in my corner. Our group definitely pays it forward. From welcoming new members, to answering the craziest of questions, to responding to someone in need, you are the very best.
The PEM Friends 2008 away weekend here in sunny Ashton-under-Lyne, Gtr. Manchester, will take place over three days and two nights, June 27th-29th, 2008. Happily, Janet Segall, founder of the International Pemphigus & Pemphigoid Foundation in America, is expecting to come over from California to join us.
Dave from America is also going to be with us this year. On Saturday evening, there will be a gala dinner in our house – fancy gowns ladies and smart casual for the men. There will be a whole dressed salmon delivered, vegetarians will be catered for, wine and non-alcholic drinks provided. For more information contact Carolyn at firstname.lastname@example.org.
Our shining star, Carolyn Blain came to Providence, Rhode Island for Thanksgiving this past year to take part in a family event. This happy circumstance became the occasion to get together with some of our community in Rhode Island and Massachusetts to sit down for a lively lunch. So on November 24, 2007, the Saturday following the big feast day, a few of us gathered at the ZBar on Wickenden Street in Providence for yet more wonderful food, and we had the place almost all to ourselves. We took full advantage of that to make ourselves right at home. Carolyn brought many requested items from England, and even a marvelous gift or two.
Once again, I am reminded that the International Pemphigus & Pemphigoid Foundation has had many terrific outcomes. One in particular is the almost immediate links of friendship.
Around our table were, Linda and Merv McKee, Gloria and Alan Papert, Carolyn Blain, Dieter and Yvette Nachmias-Baeu, and Ellen Levine.
Nearly all members of the IPPF Medical Advisory Board attended the autoimmune blistering disease meeting in Japan and many gave presentations on new research in pemphigus and pemphigoid. Clinical speakers from the MAB included Dr. Jean-Claude Bystryn, who spoke about rituximab experience in the USA, and Dr. Pascal Joly who spoke on his rituximab experiences in France. General discussion centered around the design of a new clinical trial of rituximab for pemphigus to be conducted in Europe that will be coordinated by Dr. Joly using different dosing schedules.
The other main discussion was about the development of accurate and clinically useful scoring systems for pemphigus and other AIBDs. Dr Michael Hertl, Germany, spoke on a scoring system proposed by the German group, termed the autoimmune bullous disease intensity score (ABSIS), one of the two present scoring systems. Dr. Victoria Werth, USA, presented on the comparison study performed with patients last August at the University of Pennsylvania, in terms of reliability and validity of two outcome instruments for pemphigus, as well as the international Pemphigus Disease Activity Index (PDAI) and the ABSIS.
Presentations on the first day covered the pathogenesis of pemphigus and included IPPF MAB members Dr. Luis Diaz and Dr. Sergei Grando. The chairmen of the meeting included Dr. John Stanley and Dr. Masa Amagai. Other world leaders on the causes of pemphigus presented their new work, including scientists Kathy Green (Chicago), Peter Koch (Denver), Andrew Kowalcyk (Atlanta), and Eliane Mueller (Switzerland), along with dermatologists David Rubenstein (Chapel Hill), Carlo Pincelli (Italy), Aimee Payne (Philadelphia), Yumi Aoyoma (Japan), John Zone (Salt Lake City) and Detlef Zillikens (Germany). Topics included signalling pathways, apoptosis, new animal models of blistering diseases and the role of the immune system.
The International Pemphigus Definitions group held a meeting organized by Dr. Dedee Murrell (Australia), with the help of Dr. Takashi Hashimoto (Japan), at which Dr. Werth presented the international photovalidation study that is underway to compare the two different scoring systems using photographs, so that more experts can participate.
Additional participants in these meetings included MAB members Dr. Russell Hall (Durham, NC), and Dr. Neil Korman (Cincinatti) and blistering disease experts Drs. Kim Yancey (Dallas), Robin Eady (London), Leena Bruckner-Tuderman (Germany), David Woodley (USC) and Giovanna Zambruno (Italy).
Leadership starts at the top and nowhere is that more evident than with the President of the IPPF’s Board of Directors. Dr. Dave Sirois has been instrumental in leading the Foundation to new levels of success. That being said, having the right people in the right places allows the IPPF to continue forward on our journey to new heights.
Joining the IPPF Board of Directors are Dr. Badri Rengarajan from California and Mrs. Rebecca Albrecht-Oling from New York.
Badri Rengarajan is a member of the Lehman Brothers biotechnology equity research team supporting financial markets evaluation of development- and commercial-stage biotechnology companies.
Badri has over eight years of interdisciplinary experience in business strategy, product development, regulatory/clinical analysis, and investment evaluation. He is responsible for assessing clinical studies and pipeline compounds.
Previously, he was the regulatory lead for several projects in Genentech’s portfolio, including Rituxan (lupus and indolent non-Hodgkins lymphoma), Tarceva (lung and pancreatic cancer), and anti-NGF (pain), for which Badri crafted regulatory strategy, successfully executed several regulatory filings, and negotiated with the FDA. As a member of Genentech’s regulatory intelligence group, Badri synthesized product approval histories for over 100 oncology, cardiovascular, autoimmune, and specialty therapeutics. Badri began his career in management consulting with McKinsey and Company where he served clients in biotechnology, pharmaceuticals, and devices.
Badri earned his bachelor’s degree in Biology and Public Policy from Harvard University and his M.D. from Yale University, where he received a nomination for best thesis project.
Rebecca Albrecht-Oling coordinates the Library Instruction Program at Purchase College, State University of New York, which focuses on training students, faculty, and staff how to frame information needs, create effective research strategies, and use the library’s resources to execute those strategies. Rebecca is also the liaison for Assistive Technology needs in the Library (coordinating this with Counseling services and computing services on campus). Her collection development responsibilities include literature and Jewish studies. A graduate of Kent State University (M.L.S.), Kansas State University, (M.A.) and Purdue University, B.A., she writes extensively on literacy and electronic resources.
Rebecca admits she has the Gift of Gab and has been a huge resource in attracting gifts and grants to the IPPF as well as to campus library collections. Rebecca and her husband Scott were a bright addition to our Annual Meeting in Dallas, despite missing their two children.
We would like to invite you to be part of our new pemphigus/pemphigoid Health Management Program. This program was specifically designed at Centric Health Resources with the help of the IPPF Medical Advisory Board members and staff. With this information, we will be creating a registry of information about the impact of pemphigus/pemphigoid on patients. This will open the door to improved treatments for current patients and provide information to further the chances of finding treatments with less negative impacts — for generations to come.
Many of you already know me as Director of Patient Services for the IPPF and a pemphigus patient myself. I will be your Peer Health Coach and I will be the person you will be talking with to complete the data collection. After you complete the Enrollment and Consent Form and return it to the IPPF, I will call you to set up an appointment to complete our initial survey. During our call, which will take approximately 30-45 minutes, I will ask you specific questions about your disease process: what drugs you are on, if any; what side effects you might be having; additional health problems that might have occurred due to treatment, etc. Depending on where you are with your disease process that will determine any follow-up contacts. We expect that through this relationship and with the additional support materials for you, your caregiver and your doctor, you, personally, will achieve better health and improved quality of life.
This is a voluntary program, but we need your participation to be successful. Your participation is critical to achieving these goals. Whether you have active disease or not, we need you to help us gather information to help future patients with diagnosis, treatment and cure. The Enrollment and Consent Form and a brochure outlining information about the program are on our website at www.pemphigus.org/hmp or we can mail them to you. If you decide to enroll, additional documents will be sent to you. This Health Management Program will help to increase knowledge about pemphigus and pemphigoid, which will ultimately lead to more funding toward research to find a cure.