From the Guidelines
Pemphigus should be treated with high-dose corticosteroids, such as oral prednisolone 1 mg/kg/day, as the first-line therapy, with the addition of steroid-sparing agents like azathioprine or mycophenolate mofetil, and rituximab considered for patients intolerant of or refractory to conventional corticosteroids and adjuvant immunosuppression. The treatment of pemphigus vulgaris typically begins with high-dose corticosteroids to rapidly control the disease, with clinical improvement seen within days of starting treatment and cessation of blistering taking 2-3 weeks on average [ 1 ].
First-Line Therapy
- Corticosteroids, such as oral prednisolone, are the cornerstone of therapy, with an optimal dose suggested to be 1 mg/kg/day, although this may vary depending on disease severity [ 1 ].
- The dose of prednisolone can be increased in 50-100% increments every 5-7 days if blistering continues, and pulsed intravenous corticosteroids may be considered if high doses of oral prednisolone are required or as initial treatment in severe disease [ 1 ].
Steroid-Sparing Agents
- Azathioprine and mycophenolate mofetil are commonly used steroid-sparing agents, with azathioprine dosed at 2-3 mg/kg/day and mycophenolate mofetil at 2-3 g/day [ 1 ].
- Rituximab, dosed at 1000 mg IV given twice, two weeks apart, is considered for patients intolerant of or refractory to conventional corticosteroids and adjuvant immunosuppression [ 1 ].
Supportive Care
- Proper wound care with non-adherent dressings, pain management, and prevention of secondary infections are essential components of supportive care [ 1 ].
- Patients should be monitored regularly for disease activity and medication side effects, with adjustments to treatment made as necessary to minimize morbidity and mortality, and improve quality of life [ 1 ].
The goal of treatment is to achieve remission, with treatment withdrawal a realistic aim, although this should be done cautiously to avoid relapse [ 1 ].
From the FDA Drug Label
- 8 Pemphigus Vulgaris (PV) PV Study 1 (NCT00784589) Non-U.S. -licensed rituximab in combination with short-term prednisone was compared to prednisone monotherapy as first-line treatment in 90 newly diagnosed adult patients with moderate to severe pemphigus (74 Pemphigus Vulgaris [PV] and 16 Pemphigus Foliaceus [PF]) in this randomized, open-label, controlled, multicenter study (PV Study 1) The primary endpoint for the study was complete remission (complete epithelialization and absence of new and/or established lesions) at Month 24 without the use of prednisone therapy for 2 months or more (CRoff for greater than or equal to 2 months). Table 24 Percentage of Pemphigus Patients in Complete Remission Off Corticosteroid Therapy for Two Months or More (CRoff greater than or equal to 2 months) at Month 24, PV Study 1 (Intent-to-Treat Population)
Rituximab is used to treat Pemphigus Vulgaris (PV). The drug label provides information on a study where rituximab was used in combination with short-term prednisone as first-line treatment for moderate to severe pemphigus. The study showed that rituximab can be effective in achieving complete remission in patients with PV. 2
From the Research
Definition and Treatment of Pemphigus
- Pemphigus is a rare autoimmune bullous disorder that affects the skin and/or mucosa, and it may be life-threatening without proper treatment 3, 4.
- The main objective in the treatment of pemphigus vulgaris is to control the disease, prevent relapses, and avoid adverse events associated with the prolonged use of steroids and immunosuppressive agents 5.
Treatment Regimens
- Systemic corticosteroids remain the gold standard treatment for pemphigus vulgaris, but there are many differences in initial dose, tapering schedule, and management of relapse between different guidelines 4, 5.
- Azathioprine and mycophenolate mofetil are the first line of steroid-sparing treatment, and they have been shown to have a corticosteroid sparing effect 3, 5, 6.
- Rituximab is a monoclonal antibody targeting CD20-positive B lymphocytes that is approved as a first-line therapy in moderate-to-severe pemphigus, and it has been shown to be extremely effective in recalcitrant pemphigus 4, 5, 7.
- Other immunosuppressive agents, such as cyclophosphamide and cyclosporine, have also been used in the treatment of pemphigus, but their effectiveness and safety are not well established 3.
Comparison of Treatment Regimens
- A retrospective cohort study compared the efficacy and safety of azathioprine and mycophenolate mofetil as adjuvant drugs in patients with pemphigus, and found that mycophenolate mofetil demonstrates a shorter time to achieve complete remission on therapy and has a significantly higher steroid-sparing effect 6.
- A pilot study of 23 patients with severe pemphigus found that a combination of immunoadsorption, rituximab, pulsed dexamethasone, and azathioprine/mycophenolate mofetil induces a fast and durable remission in severe pemphigus, with a low rate of severe adverse events 7.