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Oral cyclophosphamide without corticosteroids to treat mucous membrane pemphigoid

E M Munyangango1, C Le Roux-Villet, S Doan

  • 1Departments of Dermatology OtorhinolaryngologyHistology, Avicenne Hospital, AP-HP, UFR Paris 13, 93009 Bobigny, France.

The British Journal of Dermatology
|September 12, 2012
PubMed
Abstract

Insights

Daily oral cyclophosphamide (CYC) effectively treated severe mucous membrane pemphigoid (MMP) without corticosteroids. This immunosuppressant showed rapid efficacy and safety in refractory cases, with a 69% overall response rate.

Area of Science:

  • Immunodermatology
  • Autoimmune Blistering Diseases
  • Pharmacotherapy

Background:

  • Mucous membrane pemphigoid (MMP) is a severe, potentially sight- and life-threatening autoimmune condition.
  • Refractory or severe cases often necessitate potent immunosuppressive therapy.

Purpose of the Study:

  • To assess the efficacy and safety of using daily oral cyclophosphamide (CYC) as a sole immunosuppressant for severe MMP.
  • To determine if CYC can be used effectively without concurrent corticosteroid administration.

Main Methods:

  • A cohort of 13 patients with severe, refractory MMP received daily oral CYC (2 mg/kg) without corticosteroids.
  • Concomitant therapies like dapsone, sulfasalazine, or topical agents were continued.
  • Treatment response was evaluated using clinical scoring, with CYC cessation upon complete remission, progression, or significant lymphopenia.

Main Results:

  • An overall response rate of 69% was observed after 52 weeks of CYC treatment.
  • 54% of patients achieved complete remission (CR), with sustained CR at week 52.
  • The most frequent adverse event was lymphopenia (77%), leading to treatment discontinuation in some cases; no sepsis occurred.

Conclusions:

  • Daily oral cyclophosphamide demonstrates rapid efficacy in managing severe, refractory mucous membrane pemphigoid.
  • The use of cyclophosphamide without corticosteroids appears to be a safe and effective therapeutic option for this condition.

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