The IPPF does not endorse any drugs, treatments, or products. Information is provided for informational purposes only. Because the symptoms and severity of pemphigus and pemphigoid vary among individuals, we recommend all drugs and treatments be discussed with the reader’s physician(s) for proper evaluation, treatment, and care.
Note: Information is a key factor in treating and living with any condition. However, every patient’s situation is unique. The IPPF reminds you that any information found on the internet or during presentations should be discussed with your own doctor or healthcare team to determine if it applies to your specific situation.
Three Phases of Blistering Disease Treatment
- Control: A period of intense therapy given to suppress disease activity until no new lesions appear.
- Consolidation: Drugs and doses are maintained until complete clearance of lesions.
- Maintenance: Medications can be gradually tapered aiming for the lowest dose that prevent new lesions from appearing.
Initial therapy: control and consolidation
Initial therapy is determined by the extent and rate of the progression of lesions. The priority is to control lesions. Usually in a slow progressive form of the disease, initial treatment includes intralesional injections of corticosteroids or topical application of corticosteroids.
Maintenance therapy
Once most lesions have healed, the dose and type of medication are gradually reduced to limit the risk of side effects. Understanding the rate of dose reduction is determined by clinical response and overall disease activity. It is important to monitor this balance and limit use of un-necessary medication as many fatalities are related to complications with therapy.
Relapse may occur at any time, resulting in renewed disease control effort.
Types of Therapies
Corticosteroids
Corticosteroids mimic the effect of the adrenal hormones your body naturally produces. Systemic corticosteroids are the most established therapy for the management of PV. In most cases, when used in high doses, they can rapidly control disease. The most common corticosteroids include Prednisone and Prednisolone.
Prednisone suppresses the immune system and limits inflammation in the body. Prednisolone is an oral corticosteroid that is usually used in combination with an immunosuppressant.
Once controlled, steroid use is slowly reduced to minimize side effects. Some patients then go into remission; however, many need a small maintenance dose to keep the disease under control.
Topical steroids can be used for the treatment of pemphigus. To address oral erosion, steroid mouthwash, paste, ointment or aerosol can be used. Topical cyclosporine can also be used for the treatment of oral pemphigus lesions.
If only the mouth and nose are impacted, treatment should be limited to topical steroids, intralesional steroid injections, or occasional short burst of oral corticosteroids. If the gums are involved, topical therapy should be applied with flexible dental trays.
Immunosuppressants
The following immunosuppressants are used to suppress the immune system. For more information on any of the following products, visit the manufacturer website.
Azathioprine (Imuran®, Azasan®) is used after initial treatment to manage pemphigus.
Mycophenolate (CellCept®, Myfortic®) is composed of several penicillium species that is used after initial treatment for pemphigus.
Cyclophosphamide (Cytoxan®) is an oral cyclophosphamide that is considered an alternative to azathioprine. Due to the potential toxicities, this drug should be reserved for patients who do not respond to other immunosuppressives.
Cyclosporine Gengraf®, Neoral®, Sandimmune® Capsules, Sandimmune® are oral solutions.
Rituximab (Rituxan®)
Rituximab is a B-cell antibody treatment option for patients with pemphigus that is being used as first line therapy by many clinicians. In June 2018, the FDA approved Rituxan for the treatment of adults with moderate to severe PV. Earlier in the year, the FDA had granted Priority Review, Breakthrough Therapy Designation, and Orphan Drug Designation to Rituxan for the treatment of PV.
How does it work? B-cells are responsible for producing antibodies for the body, rituximab works as an immunosuppressant that destroys B-cells of the immune system. A course of rituximab is administered with the hope that it will destroy all the B-cells that make antibodies in pemphigus or pemphigoid are removed. Retreatment with rituxan may be required, usually at six months or longer after the initial treatment.
Learn more on our Rituxan information page.
IVIG
Intravenous Immunoglobulin (IVIG) therapy is prepared from extracting the plasma in human blood. IVIG is given intravenously; under the skin via a syringe or catheter. The dosage required is patient-specific. To treat pemphigus, the doses are as high as 2000mg/kg. Due to the fact that doses are higher, infusions are administered over the course of up to five days. This treatment can become a lifetime commitment or the condition may be resolved and IVIG can be discontinued.
Anti-inflammatory agents
Anti-inflammatory agents such as dapsone and tetracyclines are used as they also may have a steroid sparing effect in mild to moderate disease (e), often in patients who are in maintenance phase but corticosteroid-dependent. Dapsone is a first-line treatment in dermatitis herpetiformis, linear IgA disease, and milder cases of pemphigus foliaceus:
Dapsone
Dapsone must be started after glucose-6-P-dehydrogenase screening and is administered as 7.5mg/kg/day, up to 200mg/day.
- Topical corticosteroids (high potency) are commonly used for management of lesions.
- Coping with erosions and the pain of the erosions.
- In an open prospective study of 18 cases, low-dose methotrexate was shown to be effective for maintenance of clinical remission induced by initial short-term use of potent topical steroids;
- Considering that the prognosis of untreated BP is better than that of pemphigus, side effects of treatment are of greater concern.
- Two small studies of severe ocular mucous membrane pemphigoid suggest that this condition responds more favorably to treatment with cyclophosphamide combined with prednisone, whereas dapsone suppresses some cases of mild to moderate disease.
Tetracycline antibiotics
Tetracycline, doxycycline and minocycline have been used by some in glucocorticoid-dependent patients in the maintenance phase of therapy, often with niacinamide (nicotinamide). It is administered as tetracycline 2g/day and niacinamide 1.5g/day (in divided doses, or minocycline 100mg twice daily) and niacinamide 1.5g/day (in divided doses).
Oral
For multiple oral erosions, corticosteroid mouthwashes are practical, for example, soluble betamethasone sodium phosphate 0.5 mg tablet dissolved in 10 mL water may be used up to four times daily, holding the solution in the mouth for about 5 min. Isolated oral erosions could be treated with application of triamcinolone acetonide 0.1% in adhesive paste or clobetasol 0.05% gel. Topical cyclosporine (100 mg/ m1) in oral pemphigus has been described and may be of some benefit but is expensive (qq).
Side Effects & Precautions
It is important that all physicians, doctors, and specialists involved with a treatment are in contact with one another to avoid conflicting medications and to be sure that each doctor’s treatments are working in harmony. Lab results should also be shared with all physicians. Each individual may experience side effects when they begin a new treatment, it is important to monitor and contact your physician if you experience any adverse reactions.
Potential side effects may include:
- Headaches
- Nausea
- Stomach aches
- High blood pressure
- Stroke
- Emotional difficulties or mood swings
- Weight gain
A common side effect of prednisone is Type 2 Diabetes (steroid-induced diabetes), this creates a need for a modified diet. Generally, this type of diabetes will diminish as the dosage of prednisone is reduced and will no longer be present when prednisone is discontinued.
Weight gain is another commonly reported side effect of prednisone. A high protein, low carbohydrate, low fat diet, and a regular exercise program is recommended for those taking prednisone. Osteoporosis glaucoma, and cataracts are also known side effects of prednisone.
TIP: Preventing osteoporosis
Osteoporosis, thinning of bones, is a side effect of high-dose steroids. Periodic bone density tests are important to determine bone health and start any early treatments or begin taking supplements if necessary.
Side effects may include:
- Headache
- Fever
- Fatigue
- Chills
- Flushing
- Dizziness
- Urticaria
- Chest Tightness
- Nausea and Vomiting
- Muscle cramping
- Blood pressure changes
IVIG is considered to be safe, and the majority of people tolerate it without problems. The adverse reactions occur only in less than 1% of patients.
Patients with pemphigus and pemphigoid who suffer from side effects of steroid therapy have a higher risk of adverse reactions. Most of the side effects occur because it’s administered too quickly. In order to avoid this, it is gradually infused, starting at a very low rate and increased at intervals until the maximum rate is reached.
Side effects may include:
- Dizziness
- Weakness
- Nausea
- Light-headedness
- Itch
Additional symptoms for an individual with a fever may include:
- Chills
- Muscle pain
- Sneezing
- Sore throat
- Trouble breathing
- Pain in chest or shoulders
Infusion reactions often occur within the first 24 hours after first rituximab infusion.
Azathioprine
- Chest pain
- Cough or hoarseness
- Fever or chills
- Lower back or side pain
- Painful or difficult urination
- Pinpoint red spots on the skin
- Shortness of breath
- Sore throat
- Bleeding gums
- Blood in the urine or stools
Mycophenolate
- Blood in the urine
- Chest pain or discomfort
- Cough or hoarseness
- Fever or chills
- Increased cough
- Lower back or side pain
- Painful or difficult urination
- Shortness of breath
- Swelling of the feet or lower legs
Cyclophosphamide
More common:
- Cough or hoarseness
- Fever or chills
- Lower back or side pain
- Missing menstrual periods
- Painful or difficult urination
With high doses and/or long-term treatment:
- Blood in the urine
- Dizziness, confusion, or agitation
- Fast heartbeat
- Joint pain
- Shortness of breath
- Swelling of the feet or lower legs
- Unusual tiredness or weakness
Less common:
- Black, tarry stools
- Pinpoint red spots on the skin
- Unusual bleeding or bruising
Rare:
- Frequent urination
- Redness, swelling, or pain at the injection site
- Sores in the mouth and on the lips
- Sudden shortness of breath
- Unusual thirst
- Yellow eyes or skin
Other Complications
You may experience difficulty:
- Getting necessary information.
- Coping with the high drug doses that you will need in the initial stages of treatment.
- Coping with frequent outpatient visits if your drugs have to be administered in hospital.
- Coping with erosions and the pain of the erosions.
Once the disease is controlled, you might encounter difficulty:
- Coping with relapses and flare-ups.
- Living with pain and minor lesion activity.
- Itching and burning of skin erosions.
- Coping with the side effects of drug treatments especially prednisolone and other immunosuppressive drugs.
- Coping with other reported effects like muscle pain, insomnia, exhaustion, or nausea.
Some patients find that once P/P are controlled, their lives are not changed too much. Others find the disease impacts their lives in many different ways, including:
- Financial problems related to prescriptions, special dressings and creams, special liquidized food etc. This can be impacted by a change in employment status or difficulties obtaining incapacity benefits or disability living allowances.
- A change in energy.
- Managing the social effects of P/P, specifically the unpredictability of flare-ups.
- Additional effects on the body including weight gain (from steroids) and visible erosions on the skin which may leave discolored marks.