Epidermolysis Bullosa Acquisita Medication

Updated: Apr 29, 2016
  • Author: Daniel L Croom, MD; Chief Editor: Dirk M Elston, MD  more...
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Medication

Medication Summary

In epidermolysis bullosa acquisita (EBA), as in other autoimmune blistering diseases, treatment is directed at decreasing the development of new blisters, promoting healing, and preventing scarring and the sequelae of scarring.

The main goal of treatment is to modify or reduce the autoimmune response and decrease the production of autoantibodies. To date, only non–target-specific immunosuppressive and anti-inflammatory agents are available. There have been no randomized controlled trials to assess epidermolysis bullosa acquisita treatments. Most treatments are based on case reports and retrospective case series. Corticosteroids remain a mainstay of treatment, although many patients do not respond well and require various other immunosuppressants.

Prednisone doses commonly range from 0.5-1.5 mg/kg/day. [33] A retrospective analysis of 30 epidermolysis bullosa acquisita patients demonstrated that high-dose methylprednisolone (>8 mg/day) for at least 1 month achieved remission in 3 months as compared with remission in 12 months in those treated with a low dose (34</ref>

Dapsone is an antibiotic as well as an anti-inflammatory agent used to treat non‒pathogen-related inflammatory diseases. A review in 2011 summarized the treatment of 18 patients who received dapsone at doses of 25-100 mg/day or 1-2 mg/kg/day in conjunction with prednisone. Clinical improvement was noted in all 18 patients, of which half were able to discontinue steroid use. [35] A retrospective review in 2014 documented 7 patients treated with dapsone (50-150 mg/day) in conjunction with corticosteroids. Two patients achieved complete remission and 2 patients achieved partial remission. Of note, adverse effects occurred in 2 patients, both at doses greater than 100 mg. [13]

Colchicine administration at a dose of 0.5-2.0 mg/day has been reported in 15 patients, both as a sole agent and in combination with other treatments. Some form of remission occurred in 13 of 15 patients, with a majority requiring maintenance therapy. [13, 35] Diarrhea is a common adverse effect.

Immunosuppressives are used in patients with severe disease. Azathioprine, cyclophosphamide, mycophenolate mofetil, and cyclosporine have all been described as treatments in small case reports and case series. Immunosuppressive agents are often used in combination with steroids or other anti-inflammatory treatments. [33, 35]

Extracorporeal photochemotherapy (photophoresis) has been shown to be effective in 3 of 4 patients after 3-6 cycles of treatment. [35]

Rituximab use has been reported in 7 patients with refractory disease. Five of 7 patients had clinical improvement. One patient died of septicemia. [13]

Intravenous immunoglobulin (IVIg) may be an option in severe disease. A 2011 review documented 15 cases of IVIg use in patients mostly with severe widespread refractory disease. Clinical improvement was noted in 14 of 15 patients.  Thirteen of 15 patients remained on IVIg for maintenance treatment. [35] A more recent retrospective case series demonstrated similar clinical response rates, but suggested IVIg may induce a more sustained remission. Ten patients, all nonresponsive to conventional treatments, were started on 2 g/kg/cycle of IVIg for a mean of 23 cycles over 39 months. All 10 demonstrated clinical response and were able to completely withdraw their previous therapy; no recurrence was observed during a mean follow-up period of 54 months after cessation of treatment. [36]

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Anti-inflammatory agents

Class Summary

In the inflammatory form of epidermolysis bullosa acquisita (EBA), there may be an inflammatory cell infiltration near the basement membrane zone. Theoretically, anti-inflammatory agents would block the inflammatory process and improve the disease. Prednisone, dapsone, and colchicine all have been reported to induce clinical improvement.

Prednisone (Deltasone)

Prednisone is used as a sole agent or in conjunction with other medications (eg, immunosuppressives) to treat epidermolysis bullosa acquisita (EBA). It may decrease inflammation by reversing increased capillary permeability and suppressing PMN activity. Prednisone stabilizes lysosomal membranes and suppresses lymphocytes and antibody production.

Dapsone (Avlosulfon)

Dapsone is bactericidal and bacteriostatic against mycobacteria; its mechanism of action is similar to that of sulfonamides where competitive antagonists of PABA prevent the formation of folic acid, inhibiting bacterial growth. It is used in conjunction with other anti-inflammatory medications and immunosuppressives. In children, physicians should consult the patient's pediatrician before prescribing this medication.

Colchicine (Colcrys, Mitigare)

Colchicine is an anti-inflammatory agent that disrupts cytoskeletal functions by inhibiting B-tubulin polymerization, preventing neutrophil activation, degranulation, and migration.

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Photophoresis agents

Class Summary

Extracorporeal photochemotherapy (photophoresis) a therapeutic method that uses ultraviolet-sensitizing medication (psoralen) and extracorporeal ultraviolet A irradiation of the sensitized WBCs. The photoinactivated cells are reinfused into the patient.

Methoxsalen (8-MOP, Oxsoralen)

Methoxsalen inhibits mitosis by binding covalently to pyrimidine bases in DNA when photoactivated by ultraviolet A.

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Biological response modulators

Rituximab (Rituxan)

Rituximab is a monoclonal antibody that specifically targets the immune cell surface marker CD20, which is primarily expressed by B lymphocytes.

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Immune Globulins

Immune globulin IV (IGIV) (Bivigam, Carimune, Carimune NF)

IVIg is a purified preparation of gamma globulin. It is derived from large pools of human plasma comprising 4 subclasses of antibodies, approximating the distribution of human serum. They may decrease autoantibody production and increase solubilization and removal of immune complexes.

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