Azathioprine

There are no RCTs investigating the use of azathioprine in pemphigus, although it is probably the most commonly used adjuvant agent. The largest case series was reported by Aberer et al.12 Twenty-nine patients with pemphigus vulgaris were treated with steroid plus azathioprine. Of eight additional patients excluded from the study because of insufficient follow up, two had been switched from azathioprine to cyclophosphamide as a result of inadequate disease control and later dropped out of the study. All patients were treated initially with corticosteroids (80-200 mg daily) plus azathioprine (2-3 mg/kg). Patients were followed for a minimum of 4 years, during which time there was only one (3%) disease- or therapy-related death. Partial remission was induced in all patients. Complete remission of at least 2 months (average 4 years) was achieved in 45% of patients. The duration of treatment needed to achieve either partial or complete remission was not reported. Sixty-six per cent of patients had a relapse, which the authors ascribed to too rapid a reduction in drug dose.

In another case series, Lever and Schaumburg-Lever13 treated 21 patients with pemphigus vulgaris initially with one of two steroid regimens plus azathioprine. Thirteen patients with milder disease received prednisone 40 mg every other day plus azathioprine 100 mg daily. Eight patients with more severe disease initially received high-dose corticosteroid (200-400 mg/ day) for 5-10 weeks, followed by tapering of the steroid and initiation of the combined regimen described above for patients with mild disease. Overall, no deaths occurred between 1976 and 1981, and complete remission was induced in 12 patients (57%). The duration of treatment required to achieve remission was not reported. Five patients had relapses, all of which were reportedly mild and were sensitive to either combined therapy or steroid alone.

Five earlier case series also reported no deaths using azathioprine, but reported lower rates of remission than the series summarised above.12,13 The range in results may reflect the small number of patients in individual studies, and highlights the lack of reliability of the available data and the need for controlled studies. The higher rates of remission in more recent studies may reflect improved management of issues unrelated to azathioprine, such as improved management of steroid dosing regimens and complications resulting from therapy.

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