MMP case series, supplementary material

Background: The variable clinical severity of MMP often leads to a diagnostic and therapeutic delay. Objective: To describe the characteristics in a large cohort of patients with MMP. Methods: A retrospective review study of clinical and diagnostic characteristics and treatment response in 145 patients with MMP. Results: Monosite involvement was seen in 41.4% and multisite involvement in 58.6% patients. The oral mucosa was affected in 86.9%, followed by the ocular mucosa (30.3%), skin (26.2%), genital mucosa (25.5%), nasal mucosa (23.4%) and pharyngeal and/or laryngeal mucosa (17.2%). Ocular disease developed during disease course in 41.7% of patients with initially other mucosal site involvement. The malignancy rate was significantly higher in patients with autoantibodies against laminin-332 compared to MMP patients without laminin-332 autoantibodies (35.3% vs. 10.9%, p=0.007). Systemic immunosuppressive or immunomodulatory therapy were administered in 77.1% of the patients, mainly in patients with multisite involvement (p<0.001), ocular involvement (p<0.001) and pharyngeal and laryngeal involvement (p=0.002). The remaining patients (22.9%) received topical therapy. Adverse events were frequently reported. Limitations: retrospective design. Conclusion: MMP presents with a heterogeneous clinical presentation and new symptoms may develop during the disease course. Cancer screening should be considered in MMP and in particular with autoantibodies against laminin-332.背景：黏膜类天疱疮（MMP）的临床严重程度存在显著异质性，常导致诊疗延误。 目的：描述大样本MMP患者队列的临床特征。 方法：本研究为回顾性分析，纳入145例MMP患者，对其临床、诊断特征及治疗应答情况进行分析。 结果：41.4%的患者表现为单部位受累，58.6%的患者存在多部位受累。受累部位以口腔黏膜最为常见（86.9%），其次为眼黏膜（30.3%）、皮肤（26.2%）、生殖器黏膜（25.5%）、鼻黏膜（23.4%）以及咽和/或喉黏膜（17.2%）。初始累及其他黏膜部位的患者中，有41.7%在病程中出现眼部受累。抗层黏连蛋白-332自身抗体阳性的MMP患者，其恶性肿瘤发生率显著高于阴性患者（35.3% vs. 10.9%，p=0.007）。77.1%的患者接受了系统性免疫抑制或免疫调节治疗，此类治疗主要应用于多部位受累（p<0.001）、眼部受累（p<0.001）以及咽和喉受累的患者（p=0.002）。剩余22.9%的患者仅接受局部治疗，不良事件报告较为常见。 局限性：本研究为回顾性设计，存在相关局限。 结论：MMP的临床表现具有显著异质性，病程中可能出现新发症状。对于MMP患者，尤其是抗层黏连蛋白-332自身抗体阳性者，应考虑开展恶性肿瘤筛查。