Rituximab treatment of severe pemphigus: long-term results including immunologic follow-up

J Am Acad Dermatol. 2012 Oct;67(4):623-9. doi: 10.1016/j.jaad.2011.12.019. Epub 2012 Jan 20.

Abstract

Background: Rituximab (RTX) has been shown to be effective and safe for short-term treatment of severe pemphigus. Its long-term results remain unknown.

Objective: We sought to evaluate long-term RTX efficacy and safety in comparison with classic immunosuppressants for the treatment of severe pemphigus.

Methods: This retrospective study included, from 1997 to 2010, 24 consecutive patients with severe pemphigus, treated with RTX (n = 13) or systemic corticosteroids alone or combined with immunosuppressants (n = 11 control subjects). Anti-desmoglein antibodies were titered by enzyme-linked immunosorbent assay, every 3 months the first year, then at least annually.

Results: Among the 13 patients treated with RTX, 9 achieved complete remission 3 months after a first RTX cycle. Thereafter, 7 patients (4 with maintenance therapy) relapsed within a mean of 18 months after the last RTX cycle and received 1 or 2 additional RTX cycles. With mean follow-up at 41 months after the first RTX cycle and 28 months after the last one, all 13 patients remained in complete remission (5 patients off therapy). No severe RTX side effects occurred. Anti-desmoglein-3 autoantibodies remained positive in 7 patients, despite long-term complete remission. Long-term remission rates and immunologic profiles did not differ between patients with pemphigus according to RTX status.

Limitations: This was a single-center, retrospective study.

Conclusions: RTX appeared to be an effective and well-tolerated treatment for severe pemphigus at long term. However, the long-term remission rate without maintenance therapy did not differ significantly from that of control subjects. Anti-desmoglein-1 autoantibody titers were more reliable than anti-desmoglein-3 titers for long-term follow-up.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antibodies, Monoclonal, Murine-Derived / administration & dosage*
  • Autoantibodies / blood
  • Child
  • Child, Preschool
  • Desmoglein 3 / immunology
  • Drug Therapy, Combination / methods
  • Female
  • Follow-Up Studies
  • Humans
  • Immunologic Factors / administration & dosage*
  • Immunosuppressive Agents / administration & dosage
  • Male
  • Middle Aged
  • Pemphigus / drug therapy*
  • Pemphigus / immunology*
  • Retrospective Studies
  • Rituximab
  • Severity of Illness Index
  • Time Factors
  • Treatment Outcome
  • Young Adult

Substances

  • Antibodies, Monoclonal, Murine-Derived
  • Autoantibodies
  • DSG3 protein, human
  • Desmoglein 3
  • Immunologic Factors
  • Immunosuppressive Agents
  • Rituximab