Jeffrey Bronte, Schifter Mark, Arena Elizabeth, Sullivan Emily, Rose Stephanie, Joo David, Campbell David, Culican Suzanne, McDonald David, Lin Ming Wei
Department of Clinical Immunology, Westmead Hospital, Sydney, Australia.
St Vincent's Clinical School, University of New South Wales, Sydney, Australia.
Australas J Dermatol. 2025 Aug;66(5):e271-e278. doi: 10.1111/ajd.14523. Epub 2025 May 16.
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.
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.
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.
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.
黏膜类天疱疮（MMP）有广泛的临床表现，从相对良性的自限性口腔损害到口腔、鼻腔和眼部组织出现严重瘢痕形成（瘢痕化）并伴有严重功能障碍和发病情况。欧洲指南根据疾病的程度/严重程度，推荐利妥昔单抗仅作为二线或三线治疗药物；然而，尚无既定的临床或血清学标志物可预测严重疾病，从而确定是否有必要使用利妥昔单抗等药物。
对符合以下标准的患者进行回顾性横断面队列研究：（1）活检确诊为MMP；（2）需要使用能减少类固醇用量的免疫抑制剂治疗，即霉酚酸酯和/或利妥昔单抗；（3）至少有6个月的临床监测。主要终点是完全或部分缓解。
在符合标准的45例患者中，12例（27%）使用霉酚酸酯后获得持续缓解。33例（73%）患者病情复发或对霉酚酸酯耐药，因此接受了利妥昔单抗治疗。在接受利妥昔单抗治疗的患者中，97%在一个疗程（第1天和第14天静脉注射1g）后实现完全缓解，但24%的患者需要重复治疗。关键循环抗体，即皮肤基底膜抗体（SBMA）、BP180/230、胶原蛋白VII和层粘连蛋白332的检测率较低，无法识别对霉酚酸酯耐药的患者。两个患者组的不良反应，包括感染并发症，都很少。
在我们对大多为局限性黏膜MMP患者的研究中，患者对利妥昔单抗单疗程治疗反应良好，缓解持久且无重大不良并发症。
