Li Jingjing, Wang Ran, Chen Jie, Xu Antao, Fu Yakai, Lin Yanwei, Wang Xiaodong, Ye Shuang, Yuan Ye, Du Fang, Fu Qiong
Department of Rheumatology, Renji Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
State Key Laboratory of Biopharmaceutical Preparation and Delivery, Institute of Process Engineering, Chinese Academy of Sciences, Beijing, China.
Front Immunol. 2025 Jun 26;16:1604648. doi: 10.3389/fimmu.2025.1604648. eCollection 2025.
Rheumatic diseaseassociated macrophage activation syndrome (RD-MAS) is a rare and life-threatening complication of rheumatic diseases, with approximately 30% of cases being refractory to conventional therapeutic protocols. Ruxolitinib, a Janus kinase 1/2 inhibitor, has emerged as a potential therapy for refractory RD-MAS. This study aimed to evaluate its efficacy and safety in patients with refractory RD-MAS.
A meticulous chart review was conducted on 20 refractory RD-MAS patients treated with ruxolitinib. Data from no ruxolitinib treatment RD-MAS patients served as historical controls. Clinical and laboratory parameters, therapeutic response, and survival outcomes were analyzed. Ruxolitinib's efficacy and safety were evaluated, and survival rates were compared to historical controls.
The cohort included 20 refractory RD-MAS patients (17 females, 3 males) with underlying conditions: adult-onset Still's disease (n = 13), systemic lupus erythematosus (n = 4), and other connective tissue diseases (CTDs) (n = 3). All patients displayed active disease at baseline. By week 8, 50% (10/20) of patients achieved partial remission, while 30% (6/20) attained complete remission. The ruxolitinib group had a significantly higher survival rate (19/20, 95%) compared to historical controls (13/21, 62%) (P = 0.011). By week 8, the median daily glucocorticoid dose dropped from 2.7 mg/kg to 0.5 mg/kg. Cytomegalovirus infection occurred in 20% (4/20) of patients.
Ruxolitinib demonstrated substantial efficacy and tolerability in refractory RD-MAS, improving clinical outcomes and reducing glucocorticoid dependence. Although limited by its retrospective nature and small cohort size, this study suggests that ruxolitinib may serve as a potential therapy for refractory RD-MAS, warranting further investigation.
风湿性疾病相关巨噬细胞活化综合征（RD-MAS）是风湿性疾病一种罕见且危及生命的并发症，约30%的病例对传统治疗方案无效。鲁索替尼，一种Janus激酶1/2抑制剂，已成为难治性RD-MAS的一种潜在治疗方法。本研究旨在评估其在难治性RD-MAS患者中的疗效和安全性。
对20例接受鲁索替尼治疗的难治性RD-MAS患者进行了详细的病历回顾。未接受鲁索替尼治疗的RD-MAS患者的数据作为历史对照。分析了临床和实验室参数、治疗反应及生存结果。评估了鲁索替尼的疗效和安全性，并将生存率与历史对照进行比较。
该队列包括20例难治性RD-MAS患者（17例女性，3例男性），其基础疾病为：成人斯蒂尔病（n = 13）、系统性红斑狼疮（n = 4）和其他结缔组织病（CTD）（n = 3）。所有患者在基线时均表现为疾病活动。到第8周时，50%（10/20）的患者实现部分缓解，而30%（6/20）达到完全缓解。与历史对照（13/21，62%）相比，鲁索替尼组的生存率显著更高（19/20，95%）（P = 0.011）。到第8周时，糖皮质激素的每日中位剂量从2.7 mg/kg降至0.5 mg/kg。20%（4/20）的患者发生了巨细胞病毒感染。
鲁索替尼在难治性RD-MAS中显示出显著疗效和耐受性，改善了临床结局并降低了对糖皮质激素的依赖。尽管本研究受限于其回顾性性质和小样本量，但提示鲁索替尼可能为难治性RD-MAS的一种潜在治疗方法，值得进一步研究。
