Tag Archives: treatment

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On March 15, 2019,  Roche announced that the European Commission has approved MabThera® (rituximab) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), a rare condition characterised by progressive painful blistering of the skin and/or mucous membranes. Extensive blistering can lead to serious, life-threatening fluid loss, infection and/or death.

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On Thursday, June 7th, the FDA approved Rituxan for the treatment of adults with moderate to severe pemphigus vulgaris (PV). Rituxan is the first biologic therapy approved by the FDA for PV and the first major advancement in the treatment of PV in more than 60 years.

Syntimmune recently announced positive preliminary results from its phase 1b proof-of-concept trial of SYNT001 in pemphigus vulgaris and foliaceus patients. It’s exciting for the IPPF to share good news related to research and treatments. The full press release from Syntimmune can be found here. The following is an excerpt:

 

Syntimmune, Inc., a clinical-stage biotechnology company developing antibody therapeutics targeting FcRn, today announced positive preliminary results from its Phase 1b proof-of-concept trial of SYNT001 in patients with pemphigus vulgaris and pemphigus foliaceus. The data showed clinically meaningful benefit of SYNT001, with a favorable safety and tolerability profile similar to that observed in the Phase 1a study.

“There remains a clear unmet need for a safe and fast-acting treatment for patients with pemphigus, who face serious symptoms and complications associated with their disease,” said Donna Culton, M.D., Ph.D., an assistant professor at the University of North Carolina School of Medicine. Culton presented preliminary results of the Phase 1b study at the International Investigative Dermatology conference being held on May 16-19, 2018 in Orlando, FL. “These preliminary data demonstrate safety as well as a rapid reduction in PDAI scores and lowering of IgG levels with treatment of SYNT001, which support further studies of this drug as a potential new therapeutic option,” Culton said.

Read Syntimmune’s press release, including additional information, here. 

image of an anitgenGenentech recently announced an important FDA decision that could potentially impact future treatment options for pemphigus. Here at the IPPF, it’s especially exciting when we get to share good news related to research and treatments. The full press release from Genentech can be found here. The following is an excerpt:

The U.S. Food and Drug Administration (FDA) has accepted Genentech’s Supplemental Biologics License Application (sBLA) and granted Priority Review for the use of Rituxan® (rituximab) for the treatment of pemphigus vulgaris (PV). Last year, the FDA granted Breakthrough Therapy Designation and Orphan Drug Designation to Rituxan for the treatment of PV.

“We are committed to developing medicines for rare diseases with limited treatment options, such as pemphigus vulgaris,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We look forward to continued work with the FDA to hopefully provide patients with a new treatment for this serious and potentially life-threatening disease.”

The sBLA submission is based on data from a Roche-supported randomized trial conducted in France which evaluated Rituxan plus a tapering regimen of low dose oral corticosteroid (CS) treatment compared to a standard dose of CS alone as a first-line treatment in patients with newly diagnosed moderate to severe pemphigus. Results of the study show that Rituxan provides substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of CS therapy. These results were published in The Lancet in March 2017. Genentech is currently conducting another Phase III study in PV which is evaluating Rituxan plus a tapering regimen of CS compared to Cellcept (PEMPHIX, NCT02383589).

Read Genentech’s press release, including additional information and references, here. 

Now that you know what you have been diagnosed with, it is important that all of the doctors who treat you are aware of pemphigus and pemphigoid (P/P), too.

This includes pemphigus vulgaris, bullous pemphigoid, mucous membrane pemphigoid (aka ocular cicatricial pemphigoid), pemphigus foliaceus, pemphigus vegetans, IgA pemphigus, paraneoplastic pemphigus, or gestational pemphigoid.  

All P/P rare autoimmune diseases are skin-blistering conditions. Steroid medication is often recommended in conjunction with immunosuppressant therapy in order to lower steroid dosages and bring the patient to remission (or at the very least, on a low-dosage maintenance program).

Since this is a condition that affects the skin, any invasive surgery will require an adjustment to medications prior to and after the procedure. This includes dental work (i.e. tooth extraction). Definitely for knee replacements, hip replacements, and any other invasive procedure.

Therefore, it is very important to advise your primary care physician, dentist, internist, rheumatologist, ob-gyn, and any other medical professional you are seeing on a regular basis. Each doctor needs to know about your new diagnosis and the medications you are taking, including any adjustments throughout your treatments, so they can keep track as well.

Your entire medical team is working together to keep you healthy, and keeping all of them in the loop is to your advantage.

Working for the IPPF has been something I have been interested in doing for about five years, since my pemphigus vulgaris finally got under control. I knew from my first contact with the Foundation that this is an amazing group of people. I am proud of the way our community pulls together and rallies for each other; it’s amazing how we sincerely care about one another.

In the beginning of treating a bullous skin disease like pemphigus, prednisone is usually prescribed.  A high dosage over time, can be tapered down. While we are thrilled that the dosage is being lowered, the side effects of tapering can be debilitating.

It is safer to decrease the dosage by no more than 5mg per week. Tapering too quickly can either cause a flare-up or have you feeling like your muscles are rebelling.

If you abruptly stop taking prednisone or taper off too quickly, you might experience prednisone withdrawal symptoms: A gradual reduction in prednisone dosage gives your adrenal glands time to resume their normal function.

(1) http://www.mayoclinic.org/prednisone-withdrawal/expert-answers/faq-20057923

Essentially this is a drug that mimics your body’s natural hormones produced from the adrenal glands. When prescribed in significant doses, Prednisone works to help suppress inflammation. In the event that a person’s immune system is attacking its own tissues (as is the case with autoimmune diseases), this drug can help reduce activity by suppressing immune system functioning. It affects the “HPA” or hypothalamus-pituitary-adrenal axis when taken longer than 7 days.

Side effects can include the following:  Abdominal pain, anxiety, body aches, decreased appetite, depression, dizziness, fatigue, fever, joint pain, mood swings, muscle soreness, nausea, weakness.  Not everyone experiences the same side effects, but these are the most common.

Taking an OTC (Over The Counter) pain relief can be helpful as well as taking in more salt and sugar that helps with low blood pressure and blood sugar.

(2) http://mentalhealthdaily.com/2014/06/10/prednisone-withdrawal-symptoms-how-long-do-they-last/

The body needs to keep moving although body aches can make you feel as if you don’t want to move!  What you can do is take walks at a leisurely pace until you are comfortable enough to step up the pace to a brisk walk.  Stretching every day is essential to keep your muscles flexible. Hurts,

But do what you can.  The price of being motionless is worse

if you don’t.  Your muscles will tighten and you’ll lose mobility.  A beginners yoga DVD can help you move in the beginning. Exercises in a pool help because the weight of the water pushes against you gently and cushions movement.

Meditation can help to sooth the nerves…keeping calm is very important anyway but especially when tapering because you more anxious than ever.  Listening to meditation music (Youtube if don’t have any) helps.  Talking to family members and closest friends helps too as they will understand if you seem moody or nervous.  The more you communicate, the more they can empathize and realize you need patience and humor from them!  Yes, laughter helps!

If you find that you are still having difficulty with body aches and pains and muscle weakness, consider asking your dermatologist for a referral for physical therapy.  Six visits are all you need to help you with exercises to keep you in motion and help you to get to feel better.

Remember, when you need us we will be in your corner!