Interventions for mucous membrane pemphigoid and epidermolysis bullosa acquisita (rare autoimmune blistering diseases of the skin, eyes and mouth)

Mucous membrane pemphigoid and epidermolysis bullosa acquisita are rare autoimmune blistering diseases of the skin and mucous membranes (eyes and mouth). They can result in scarring, which may lead to disabling and life threatening complications. Treatments include corticosteroids, mycophenolate mofetil and cyclophosphamide to suppress the immune system, and less toxic drugs such as antibiotics. These diseases often progress despite treatment. There is some evidence that mucous membrane pemphigoid involving the eyes may respond better to treatment with cyclophosphamide combined with corticosteroids, compared to treatment with corticosteroids alone. Cyclophosphamide is, however, associated with potentially severe adverse effects. Dapsone may help moderate disease. More research is needed to identify the most effective treatment options.There is not enough reliable evidence about treatments for the rare blistering diseases, mucous membrane pemphigoid and epidermolysis bullosa acquisita.

Authors' conclusions: 

There is limited evidence that mucous membrane pemphigoid involving the eyes responds best to treatment with cyclophosphamide combined with corticosteroids. However, mucous membrane pemphigoid with mild to modest inflammatory activity responds to dapsone in most participants and may therefore be best treated with dapsone due to its lower side effect profile compared to cyclophosphamide. Treatment with mycophenolate mofetil combined with topical steroids seems worth considering in a future randomised controlled trial for mucous membrane pemphigoid.

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Background: 

Mucous membrane pemphigoid and epidermolysis bullosa acquisita are rare acquired autoimmune blistering diseases of the skin. Both can result in scarring of mucous membranes which may lead to blindness and life threatening respiratory complications.

Objectives: 

To assess the effects of treatments for mucous membrane pemphigoid and epidermolysis bullosa acquisita.

Search strategy: 

We searched the Cochrane Skin Group Specialised Register (7th April 2005), the Cochrane Controlled Trials Register (The Cochrane Library Issue 1, 2005), MEDLINE / PubMed (from 1966 to April 2005), EMBASE (from 1980 to April 2005), www.controlled-trials.com (7th April 2005) and www.clinicaltrials.gov (7th April 2005) and reference lists of articles.

Selection criteria: 

Randomised controlled trials of any treatments for mucous membrane pemphigoid or epidermolysis bullosa acquisita involving participants of any age with a diagnosis of either disease confirmed by immunofluorescence.

Data collection and analysis: 

The data was independently extracted by three authors and subsequently checked for discrepancies. Two authors evaluated the studies in terms of the inclusion criteria.

Main results: 

Two small randomised controlled trials of mucous membrane pemphigoid, both conducted in participants with severe eye involvement were identified.

In the first trial, involving 24 participants, cyclophosphamide 2 mg/kg/day in combination with prednisone starting at 1 mg/kg/day and tapering was superior to prednisone alone (1 mg/kg/day) after 6 months of treatment. All 12 participants responded well to cyclophosphamide plus prednisone versus a good response in only 5 of 12 participants treated with prednisone (relative risk 2.40, 95% confidence interval 1.23 to 4.69).

In the second trial, involving 40 participants, all 20 participants treated with cyclophosphamide (2 mg/kg/day) responded well after three months of treatment, but only 14 of 20 participants responded to treatment with dapsone (2 mg/kg/day) (relative risk 1.43, 95% confidence interval 1.07 to 1.90). All non-responders had severe inflammatory activity. It was not explicitly stated whether these participants received prednisone in addition to dapsone or cyclophosphamide initially. Hair loss and suppression of the red and white blood cells were common adverse events in the cyclophosphamide groups.

No randomised controlled trials of treatments for epidermolysis bullosa acquisita were identified.

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