title: Rituximab for patients with refractory mucous membrane pemphigoid.
abstract: BACKGROUND
  Mucous membrane pemphigoid (MMP) still represents a potentially life- and sight-threatening disease. In a subset of patients with severe MMP, conventional immunosuppressants are ineffective or contraindicated.
  
  
  METHODS
  Twenty-five patients with severe refractory MMP, including 5 with mucous membrane-dominant epidermolysis bullosa acquisita, received 1 or 2 cycles of rituximab (375 mg/m(2) weekly for 4 weeks). Twenty-one of the patients were receiving concomitant therapy with dapsone and/or sulfasalazine therapy, which was maintained during rituximab cycles. Complete responses in all affected sites (ocular and/or extraocular) were obtained in 17 patients (68%) by a median time of 12 weeks after the first cycle, and 5 additional patients responded completely after a second cycle, yielding an 88% complete response rate. In all but 1 of the 10 patients with ocular lesions, their eyes became noninflammatory within a mean of 10 weeks. Among the 3 patients (12%) who developed severe infectious complications, 2 (8%) died; they had been receiving concomitant conventional immunosuppressants and high-dose corticosteroids and were hypogammaglobulinemic. Treatment with immunosuppressants was discontinued for all other patients, and no other infection was observed. Ten patients experienced relapse after a mean of 4 (range, 1-16) months after achieving complete responses.
  
  
  CONCLUSIONS
  Rituximab appears to have rapid and dramatic efficacy in patients with severe, refractory MMP. The occurrence of severe infections in patients receiving concomitant conventional immunosuppressants supports using rituximab without other immunosuppressants. Controlled prospective studies are warranted to define an optimal treatment protocol.
authors.affiliation: Department of Dermatology, Hôpital Avicenne, Assistance Publique–Hôpitaux de Paris, Université Paris 13, Bobigny, France.christelle.le-roux@avc.aphp.fr
authors.forename: Christelle
authors.initials: C
authors.lastname: Le Roux-Villet
authors.forename: Catherine
authors.initials: C
authors.lastname: Prost-Squarcioni
authors.forename: Marina
authors.initials: M
authors.lastname: Alexandre
authors.forename: Frédéric
authors.initials: F
authors.lastname: Caux
authors.forename: Francis
authors.initials: F
authors.lastname: Pascal
authors.forename: Serge
authors.initials: S
authors.lastname: Doan
authors.forename: Marie-Dominique
authors.initials: MD
authors.lastname: Brette
authors.forename: Isaac
authors.initials: I
authors.lastname: Soued
authors.forename: Éric
authors.initials: É
authors.lastname: Gabison
authors.forename: Françoise
authors.initials: F
authors.lastname: Aucouturier
authors.forename: Rémi
authors.initials: R
authors.lastname: Letestu
authors.forename: Liliane
authors.initials: L
authors.lastname: Laroche
authors.forename: Hervé
authors.initials: H
authors.lastname: Bachelez
mesh_terms: D000293:Adolescent; D000328:Adult; D000361:Agammaglobulinemia / Q000139:chemically induced / Q000276:immunology; D000368:Aged; D000369:Aged, 80 and over; D058846:Antibodies, Monoclonal, Murine-Derived / Q000276:immunology / Q000627:therapeutic use*; D001323:Autoantibodies / Q000276:immunology; D015331:Cohort Studies; D003141:Communicable Diseases / Q000139:chemically induced / Q000276:immunology; D003622:Dapsone / Q000276:immunology / Q000627:therapeutic use; D003879:Dermatologic Agents / Q000276:immunology / Q000627:therapeutic use; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000276:immunology / Q000627:therapeutic use*; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D010390:Pemphigoid, Benign Mucous Membrane / Q000188:drug therapy* / Q000276:immunology; D000069283:Rituximab; D012720:Severity of Illness Index; D012460:Sulfasalazine / Q000276:immunology / Q000627:therapeutic use; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D001323:Autoantibodies; D003879:Dermatologic Agents; D007155:Immunologic Factors; D007166:Immunosuppressive Agents; D012460:Sulfasalazine; D000069283:Rituximab; D003622:Dapsone