Rituximab for the Management of an Australian Cohort of Treatment Refractory Mucous Membrane Pemphigoid.

Abstract

Background: Mucous membrane pemphigoid (MMP) has a broad range of clinical manifestations, from relatively benign self-limiting oral lesions to significant scarring (cicatrizing) of the oral, nasal and ocular tissues with severe functional impairment and morbidity. European Guidelines recommend rituximab as only second- or third-line therapy, based on the extent/severity of the disease; however, there are no established clinical or serological markers that are predictive of severe disease warranting the use of agents such as rituximab.

Methods: Retrospective cross-sectional cohort study of patients who met the following criteria: (1) biopsy confirmed MMP; (2) required a steroid-sparing immunosuppressant therapy, that is, mycophenolate and/or rituximab and (3) at least 6 months of clinical monitoring. The primary end point was complete or partial remission.

Results: Of the 45 patients who met the criteria, 12 (27%) had sustained remission with mycophenolate. Thirty-three (73%) patients had either relapsed or were refractory to mycophenolate and, therefore, were treated with rituximab. Of those who received rituximab, 97% achieved a complete remission after a single course (1 g given intravenously on Days 1 and 14), but 24% needed repeat treatment. The detection rates of key circulating antibodies, namely skin basement membrane antibodies (SBMA), BP180/230, collagen VII and laminin 332, were low and did not identify those patients refractory to mycophenolate. Adverse reactions, including infectious complications, were minimal in both patient groups.

Conclusion: In our study of mostly localised mucosal MMP patients, there was an excellent response to a single course of treatment with rituximab, with durable remission and no major adverse complications.