Costagliola Giorgio, Cappelli Susanna, Consolini Rita
Laboratory of Immunology, Department of Clinical and Experimental Medicine, Division of Pediatrics, University of Pisa, Pisa, Italy.
Ther Clin Risk Manag. 2020 Jun 11;16:495-507. doi: 10.2147/TCRM.S232660. eCollection 2020.
Behçet's Disease (BD) is an inflammatory disease of unknown etiology with multisystemic involvement, being the main clinical manifestations represented by recurrent oral and genital ulcerations and uveitis. The disease has typically a chronic-relapsing course and may cause significant morbidity and mortality due to eye, vascular and neurological involvement. Although BD is more frequently diagnosed in adulthood, the disease onset can also be in pediatric age. Pediatric-onset BD is commonly featured by an incomplete clinical picture, and therefore the diagnosis represents a considerable clinical challenge for the physicians. The first classification criteria for pediatric BD, based on a scoring system, have been proposed few years ago. This work focuses on the main difficulties concerning both the diagnostic approach and the treatment of BD in pediatric age. The recommendation for the treatment of pediatric BD has been recently updated and allowed a considerable improvement of the therapeutic strategies. In particular, the use of anti-TNFα drugs as a second-line option for refractory BD, and as a first-line treatment in severe ocular and neurological involvement, has demonstrated to be effective in improving the outcome of BD patients. The knowledge about the molecular pathogenesis is progressively increasing, showing that BD shares common features with autoimmune and autoinflammatory disorders, and thus leading to the use of new biologic agents targeting the main mediators involved in the determination of BD. Anti-IL-17, anti-IL-23, anti-IL-1 and anti-IL-6 agents have shown promising results for the treatment of refractory BD in clinical trials and will represent an important alternative for the therapeutic approach to the disease.
白塞病(BD)是一种病因不明的炎症性疾病,可累及多系统,主要临床表现为复发性口腔和生殖器溃疡以及葡萄膜炎。该病通常呈慢性复发病程,可因眼部、血管和神经受累而导致显著的发病率和死亡率。虽然BD在成年期更常被诊断,但发病也可能在儿童期。儿童期发病的BD通常临床表现不完整,因此诊断对医生来说是一项相当大的临床挑战。几年前提出了基于评分系统的首个儿童BD分类标准。这项工作聚焦于儿童期BD诊断方法和治疗方面的主要困难。儿童BD的治疗建议最近已更新,使治疗策略有了显著改进。特别是,使用抗TNFα药物作为难治性BD的二线选择,以及在严重眼部和神经受累时作为一线治疗,已证明对改善BD患者的预后有效。关于分子发病机制的认识正在逐步增加,表明BD与自身免疫性和自身炎症性疾病有共同特征,从而导致使用针对BD发病主要介质的新型生物制剂。抗IL-17、抗IL-23、抗IL-1和抗IL-6药物在临床试验中对难治性BD的治疗已显示出有前景的结果,并将成为该疾病治疗方法的重要替代方案。