Alibaz-Öner Fatma, Direskeneli Haner
Division of Rheumatology, Department of Internal Medicine, Marmara University School of Medicine, İstanbul, Turkey.
Eur J Rheumatol. 2021 Oct;8(4):217-222. doi: 10.5152/eurjrheum.2020.20138.
Behçet's disease (BD) significantly increases morbidity and mortality, especially in young men. While vascular involvement is the most frequent cause of mortality, ocular involvement, which can cause visual loss, is the most important cause of morbidity in BD. Immunosuppressive treatment is the mainstay for major organ involvement. However, despite optimal immunosuppressive treatment, relapses and disease-related damage develop in a subgroup of patients, especially among those with ocular or vascular involvement. With the recent understanding of the immuno-pathogenesis, biologic treatments targeting potential pathogenic cells, cytokines or pathways are better optimized in BD. Data from large series showed that tumor necrosis factor-α inhibitors and interferon-α are effective and safe treatment options for the treatment of refractory and major organ involvement, such as ocular, neurologic, vascular, and gastrointestinal. Anakinra and ustekinumab also seem to be promising agents for refractory mucocutaneous disease. IL-1 inhibitors and tocilizumab may be alternatives for the treatment of patients with refractory eye involvement. Still, randomized controlled trials of biologic agents, especially for the treatment of major organ involvement, are insufficient, and further prospective, long-term follow-up studies are needed to clarify the efficacy, safety, and optimal treatment duration of biologic agents in BD.
白塞病（BD）显著增加发病率和死亡率，尤其是在年轻男性中。虽然血管受累是最常见的死亡原因，但可导致视力丧失的眼部受累是BD中最重要的发病原因。免疫抑制治疗是主要器官受累的主要治疗方法。然而，尽管进行了最佳的免疫抑制治疗，仍有一部分患者会复发并出现与疾病相关的损害，尤其是那些有眼部或血管受累的患者。随着对免疫发病机制的最新认识，针对潜在致病细胞、细胞因子或信号通路的生物治疗在BD中得到了更好的优化。大量数据表明，肿瘤坏死因子-α抑制剂和干扰素-α是治疗难治性和主要器官受累（如眼部、神经、血管和胃肠道）的有效且安全的治疗选择。阿那白滞素和乌司奴单抗似乎也是治疗难治性黏膜皮肤疾病的有前景的药物。IL-1抑制剂和托珠单抗可能是治疗难治性眼部受累患者的替代药物。尽管如此，生物制剂的随机对照试验，尤其是用于治疗主要器官受累的试验并不充分，需要进一步进行前瞻性、长期随访研究，以阐明生物制剂在BD中的疗效、安全性和最佳治疗持续时间。
