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. Indications Intravenous immunoglobulin (IVIg) should be considered for severe cases refractory to conventional immunosuppressive therapy. Persistent severe EBA refractory to conventional immunosuppressive therapy. Worsening of EBA disease in responding patients with ongoing flare after ceasing Ig therapy Two indications have been developed, the first to provide three months of initial Ig therapy in eligible severe refractory patients. All patients will then have a trial off Ig therapy and if worsening of EBA disease should occur, patients may be eligible for maintenance therapy provided a response to initial therapy can be demonstrated. Description and Diagnostic Criteria Epidermolysis bullosa acquisita is a rare, potentially severe disease which has no cure. This sub-epidermal blistering disorder of the skin and mucous membranes primarily affects adults but has been described in a few children. Although the majority of patients may have mild disease with blistering over trauma prone areas, there have been cases of severe widespread disease that remains refractory to conventional therapies. The end stage results of severe disease include ocular disease with conjunctival scarring and blindness, mucosal disease with oesophageal strictures and scarring fibrosis of the skin with alopecia, nail loss and mitten like deformities of the hands. EBA is an autoimmune disease characterized by the production of antibodies against type VII collagen resulting in immune-mediated disruption of the anchoring fibrils that connect the basement membrane to dermal structures and the clinical development of This section has been developed following assessment of the literature and consultation with the Australasian College of Dermatologists.
blistering. Diagnosis of EBA is based on history, full skin examination, and skin biopsies. There is limited data on treatment options for EBA and optimal approach to treatment has not been established. Suggested initial treatment is with colchicine or dapsone (Grade 2C). If treatment is not effective, these agents may be used simultaneously. EBA that is refractory to above requires more aggressive therapy. Agents that may have efficacy for refractory EBA include immunosuppressants, intravenous immunoglobulin, and rituximab. Diagnosis is required Yes By which specialty Dermatologist Clinical Immunologist Treatment of will be limited to dermatologists and clinical immunologists as it is a rare condition, usually treated by these specialists. Diagnosis must be verified No By which specialty Exclusion Criteria Qualifying Criteria Persistent severe EBA refractory to conventional immunosuppressive therapy Persistent severe EBA disease confirmed by biopsy and Immunofluorescence including ophthalmological and/or mucosal sites AND Persistent disease despite standard treatment with colchicine and dapsone and at least two other immunosuppressant agents OR The definition of persistent and severe disease for eligibility for Ig therapy has been limited to those patients with ophthalmological and /or mucosal disease. The use of Ig therapy is as a fifth line agent after failure to respond to alternative therapies unless contraindicated or an unacceptable level of side effects or toxicity
Immunosuppressant medication has resulted in unacceptable side effects or significant toxicity OR Corticosteroids and/or immunosuppressant agents are contraindicated Three months treatment is authorised initially after which time a clinical response should have been demonstrated. If, in responding patients, disease worsens following cessation of Ig therapy, a request for low dose maintenance therapy can be made. Documentation of clinical effectiveness of initial Ig therapy will be required for further requests. Worsening of EBA disease in responding patients with ongoing flare after ceasing Ig therapy Ongoing flare of mucosal or ophthalmic EBA disease in previously responding patients AND Reduction in severity and/or the number of lesions was demonstrated in response to initial Ig therapy AND At least one Immunosuppressant medication is to be given concurrently has developed. Statement informing prescribers of the requirement to have demonstrated clinical response to previous Ig therapy and then relapsed prior to commencing maintenance treatment. The criteria confirm the worsening of mucosal and /or ophthalmological disease once Ig therapy is withdrawn in responding patients. A description of current symptoms is used for comparison to demonstrate response to Ig therapy at initial review. Maintenance Ig therapy must be given in combination with at least one immunosuppressant medication to all patients. Review Criteria Persistent severe EBA refractory to conventional immunosuppressive therapy Review is not mandated for this condition. All patients will receive an initial trial of three months Ig therapy. Outcome measures are defined to indicate to prescribers of the expected clinical response.