Department of Rheumatology and Clinical Immunology, Charité - Universitätsmedizin Berlin, Berlin, Germany.
III. Department of Medicine, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Rheumatology (Oxford). 2023 Nov 2;62(11):3518-3525. doi: 10.1093/rheumatology/kead240.
To evaluate the effectiveness and safety of current treatment strategies for the vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome.
A protocolized systematic review according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines was performed. Three databases were searched for reports on treatment strategies for VEXAS. Data from the included publications was extracted and a narrative synthesis was performed. Treatment response was recorded as complete (CR), partial (PR) or none (NR) depending on changes in clinical symptoms and laboratory parameters. Patient characteristics, safety data and previous treatments were analysed.
We identified 36 publications with a total of 116 patients; 113 (98.3%) were male. The identified reports included azacytidine (CR 9/36, 25%; PR 14/36, 38.9%), Janus kinase inhibitors (JAKi) (CR 11/33, 33%; PR 9/33, 27.3%), tocilizumab (CR 3/15, 20%; PR 6/15, 40%), allogeneic stem cell transplantation (CR 6/7, 85.7%; one patient died), anakinra (CR 4/5, 80%; NR 1/5, 20%), canakinumab (CR 1/2, 50%; PR 1/2, 50%) and glucocorticoid monotherapy (CR 1/6, 16.7%; PR 4/6, 66.7%). Individual reports were available for TNF inhibitors, rituximab and MTX. Data on adverse events were available for 67 patients (67/116, 57.8%) and included: pneumonia (12/67, 17.9%), other infections (9/67, 13.4%), venous thromboembolisms (6/67, 8.9%), cytopenias (4/67, 5.9%), and acute (4/67, 5.9%) and chronic graft-vs-host-disease (2/67, 2.9%).
Current data on VEXAS treatment are limited and inhomogeneous. Treatment decisions should be individualized. For the devolvement of treatment algorithms clinical trials are needed. Adverse events remain a challenge, especially an elevated risk for venous thromboembolism associated to JAKi treatment should be carefully considered.
评估 VEXAS 综合征（空泡、E1 酶、X 连锁、自身炎症、体细胞）当前治疗策略的有效性和安全性。
根据 Preferred Reporting Items for Systematic Reviews and Meta-Analyses（PRISMA）指南进行了有方案的系统评价。检索了三个数据库中关于 VEXAS 治疗策略的报告。提取纳入出版物的数据，并进行叙述性综合。根据临床症状和实验室参数的变化，将治疗反应记录为完全缓解（CR）、部分缓解（PR）或无缓解（NR）。分析患者特征、安全性数据和既往治疗情况。
我们确定了 36 篇报告，共纳入 116 例患者；其中 113 例（98.3%）为男性。确定的报告包括阿扎胞苷（CR 36/36，25%；PR 36/36，38.9%）、Janus 激酶抑制剂（JAKi）（CR 33/33，33%；PR 33/33，27.3%）、托珠单抗（CR 15/15，20%；PR 15/15，40%）、异基因造血干细胞移植（CR 7/7，85.7%；1 例死亡）、阿那白滞素（CR 5/5，80%；NR 5/5，20%）、卡那单抗（CR 2/2，50%；PR 2/2，50%）和糖皮质激素单药治疗（CR 6/6，16.7%；PR 6/6，66.7%）。单独的报告可用于 TNF 抑制剂、利妥昔单抗和 MTX。有 67 例患者（116/116，57.8%）的不良事件数据，包括肺炎（12/67，17.9%）、其他感染（9/67，13.4%）、静脉血栓栓塞（6/67，8.9%）、细胞减少症（4/67，5.9%）、急性（4/67，5.9%）和慢性移植物抗宿主病（2/67，2.9%）。
目前关于 VEXAS 治疗的数据有限且不一致。治疗决策应个体化。为了制定治疗方案，需要进行临床试验。不良事件仍然是一个挑战，特别是 JAKi 治疗相关的静脉血栓栓塞风险增加应仔细考虑。
