Mucous membrane pemphigoid (MMP) describes a group of chronic auto-immune bullous diseases
(AIBD) of the basement membrane zone (BMZ), characterized by predominant or exclusive mucosal
involvement, including oral, naso-pharyngeal, laryngo-tracheal, genital, oesophageal, anal
and ocular mucous membranes. Circulating autoantibodies are directed against various antigens
of the BMZ: BP180, laminin 332 and type 7 collagen. MMP is a rare disease with an incidence
of 1.8 new cases/million inhabitants/year in France.
Scar formation which is secondary to initial inflammation, is a characteristic feature of
MMP, leading to major disability (e.g blindness and oesophageal, anal, vaginal stenosis) and
life-threatening situations (e.g. laryngeal stenosis leading to respiratory failure).
Dapsone is the first line treatment of mild/moderate forms of MMP. Dapsone is used both as
initial treatment, and as maintenance therapy. However, severe forms of MMP can rapidly
worsen leading to blindness, aphagia due to esophageal stenosis, respiratory failure due to
tracheal or laryngeal stenosis, and urinary and sexual dysfunctions due to genital
involvement. These patients are usually treated using immunosuppressive drugs. Indeed,
corticosteroids (CS) are not recommended in MMP.
Cyclophosphamide was considered as the most effective immunosuppressant in severe forms of
MMP, before the use of rituximab, an anti-CD20 monoclonal antibody (MAb).
Two series from our group have assessed the advantages and disadvantages of IV pulse and oral
administration of cyclophosphamide in MMP. Oral administration seems more rapidly effective
with 54% of complete remission (CR) after a median time of 24 weeks (16-52 weeks).
The results of 41 patients with severe types of MMP (including a French series of 20
patients) treated with rituximab have been published. Rate of CR after one and two cycles
were 66% and 90%, respectively. Clinical improvement was rapid, since a decrease in disease
activity was observed after 4 weeks of treatment in 64% of patients.
Our results and those of the literature suggest that rituximab might be more effective than
cyclophosphamide, which has been considered as the gold standard of treatment in severe forms
of disease, up to now.
Severe Forms of Mucous Membrane Pemphigoid
Placebo Oral Tablet,
Placebo of Rituximab,
Rituximab 1g IV,
Cyclophosphamide 50Mg Oral Tablet
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