BACKGROUND: Pemphigus vulgaris (PV) and pemphigus foliaceus (PF) are potentially life-threatening autoimmune blistering diseases. Treatment is based on long term immunosuppression with high doses of glucocorticosteroids in combination with potentially corticosteroid-sparing agents and/or rituximab. Immunoadsorption (IA) has emerged as fast acting adjuvant treatment option.

OBJECTIVES: To assess the clinical efficacy of IA in addition to best medical treatment.

METHODS: Multicentre (26 centres from Germany and Austria) randomised controlled trial in 72 patients with newly diagnosed, relapsed or chronic active PV or PF (34 females, 38 males, aged 42-72 years) comparing best medical treatment (BMT) (prednisolone 1.0 mg/kg/d plus azathioprine or mycophenolate) to adjuvant IA (BMT+IA). Central 1:1 randomization was done at the coordinating centre for clinical trials (KKS) Marburg. The primary endpoint was analyzed using Kaplan-Meier and Cox regression methods.

RESULTS: The study was ended prematurely due to safety concerns after random allocation of 72 patients to BMT+IA (n=34) or BMT (n=38). The primary endpoint, time to complete remission on therapy, was not significantly different between the groups (p=0.39; HR 1.35 (95%CI: 0.68-2.69). The cumulative dose of prednisolone was significantly lower in the BMT+IA compared to BMT alone (difference -1,214; 95%CI, -2,225 - -70; p=0.03). In a post hoc analysis, patients with more extensive PV/PF showed a tendency towards a shorter time to remission in the BMT+IA group compared to the BMT group (HR=1.87, p=0.17 in patients with baseline PDAI ≥ 15). While more adverse events were observed in patients of the BMT group (29 vs. 25), severe adverse events were more frequent in patients of the BMT+IA group (17 events in 10 patients vs. 11 events in 8 patients).

CONCLUSIONS: In this study, adjuvant IA did not show a shorter time to clinical remission but a corticosteroid-sparing effect. In patients with extensive PV/PF, post hoc analysis suggests that adjuvant IA may possibly lead to earlier remission, but potential adverse events must be carefully weighed against the expected benefits.