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

backgroundMucous 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.

methodsRetrospective 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.

resultsOf 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.

conclusionIn 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.