Omalizumab in the treatment of bullous pemphigoid resistant to first-line therapy: a French national multicentre retrospective study of 100 patients

Abstract Background Interest in the use of omalizumab to treat bullous pemphigoid (BP) in the event of resistance or contraindication to conventional therapies is currently based on limited evidence. Objectives To assess the effectiveness and safety of omalizumab in BP and to identify predictive factors in response to treatment. Methods We conducted a French national multicentre retrospective study including patients with a confirmed diagnosis of BP treated with omalizumab after failure of one or several treatment lines. We excluded patients with clinically atypical BP, as per Vaillant’s criteria. The criteria for clinical response to omalizumab were defined according to the 2012 international consensus conference. Anti-BP180-NC16A IgE enzyme-linked immunosorbent assay was performed on sera collected before initiating omalizumab, when available. Results Between 2014 and 2021, 100 patients treated in 18 expert departments were included. Median age at diagnosis was 77 years (range 20–98). Complete remission (CR) was achieved in 77% of patients, and partial remission in an additional 9%. CR was maintained ‘off therapy’ in 11.7%, ‘on minimal therapy’ in 57.1%, and ‘on non-minimal therapy’ in 31.2%. Median time to CR was 3 months (range 2.2–24.5). Relapse rate was 14%, with a median follow-up time of 12 months (range 6–73). Adverse events occurred in four patients. CR was more frequently observed in patients with an increased serum baseline level of anti-BP180-NC16A IgE (75% vs. 41%; P = 0.011). Conversely, urticarial lesions, blood total IgE concentration or eosinophil count were not predictive of CR. Patients with an omalizumab dosage > 300 mg every 4 weeks showed a similar final outcome to those with a dosage ≤ 300 mg every 4 weeks, but control of disease activity [median 10 days (range 5–30) vs. 15 days (range 10–60); P < 0.001] and CR [median 2.4 months (range 2.2–8.2) vs. 3.9 months (range 2.3–24.5); P < 0.001] were achieved significantly faster. Conclusions We report the largest series to date of BP treated by omalizumab and confirm its effectiveness and safety in this indication. Serum baseline level of anti-BP180-NC16A IgE may predict response to treatment.