Rituximab is an effective treatment in patients with pemphigus vulgaris and demonstrates a steroid‐sparing effect Academic Article uri icon

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  • Background

    Corticosteroids (CS) with or without adjuvant immunosuppressant agents are standard treatment for pemphigus vulgaris (PV). The efficacy of adjuvant therapies in minimizing steroid-related adverse events (AEs) is unproven.


    To utilize data collected in a French investigator-initiated, phase III, open-label, randomized controlled trial to demonstrate the efficacy and safety of rituximab and seek approval for its use in PV.


    This was an independently conducted post hoc analysis of the moderate-to-severe PV subset enrolled in the Ritux 3 study. Patients were randomized to rituximab plus 0·5 or 1·0 mg kg-1 per day prednisone tapered over 3 or 6 months, or 1·0 or 1·5 mg kg-1 per day prednisone alone tapered over 12 or 18 months, respectively (according to disease severity). The primary end point was complete remission at month 24 without CS (CRoff) for ≥ 2 months, and 24-month efficacy and safety results were also reported.


    At month 24, 34 of 38 patients (90%) on rituximab plus prednisone achieved CRoff ≥ 2 months vs. 10 of 36 patients (28%) on prednisone alone. Median total cumulative prednisone dose was 5800 mg in the rituximab plus prednisone arm vs. 20 520 mg for prednisone alone. Eight of 36 patients (22%) who received prednisone alone withdrew from treatment owing to AEs; one rituximab-plus-prednisone patient withdrew due to pregnancy. Overall, 24 of 36 patients (67%) on prednisone alone experienced a grade 3/4 CS-related AE vs. 13 of 38 patients (34%) on rituximab plus prednisone.


    In patients with moderate-to-severe PV, rituximab plus short-term prednisone was more effective than prednisone alone. Patients treated with rituximab had less CS exposure and were less likely to experience severe or life-threatening CS-related AEs. What's already known about this topic? Pemphigus vulgaris (PV) is the most common type of pemphigus. Corticosteroids, a standard first-line treatment for PV, have significant side-effects. Although their effects are unproven, adjuvant corticosteroid-sparing agents are routinely used to minimize steroid exposure and corticosteroid-related side-effects. There is evidence that the anti-CD20 antibody rituximab is effective in the treatment of patients with severe recalcitrant pemphigus and in patients with newly diagnosed pemphigus. What does this study add? This study provides a more detailed analysis of patients with PV enrolled in an investigator-initiated trial. Rituximab plus prednisone had a steroid-sparing effect and more patients achieved complete remission off prednisone. Fewer patients experienced grade 3 or grade 4 steroid-related adverse events than those on prednisone alone. This collaboration between academia and industry, utilizing independent post hoc analyses, led to regulatory authority approvals of rituximab in moderate-to-severe PV.


  • Chen, DM
  • Odueyungbo, Adefowope
  • Csinady, E
  • Gearhart, L
  • Lehane, P
  • Cheu, M
  • Maho‐Vaillant, M
  • Prost‐Squarcioni, C
  • Hebert, V
  • Houivet, E
  • Calbo, S
  • Caillot, F
  • Golinski, ML
  • Labeille, B
  • Picard‐Dahan, C
  • Paul, C
  • Richard, MA
  • Bouaziz, JD
  • Duvert‐Lehembre, S
  • Bernard, P
  • Caux, F
  • Alexandre, M
  • Ingen‐Housz‐Oro, S
  • Vabres, P
  • Delaporte, E
  • Quereux, G
  • Dupuy, A
  • Debarbieux, S
  • Avenel‐Audran, M
  • D'Incan, M
  • Bedane, C
  • Bénéton, N
  • Jullien, D
  • Dupin, N
  • Misery, L
  • Machet, L
  • Beylot‐Barry, M
  • Dereure, O
  • Sassolas, B
  • Benichou, J
  • Musette, P
  • Joly, P

publication date

  • May 2020