Xie Weilin, Liu Qingyan, Tang Yanchun, Li Yao, Zhang Yue, Wang Shuhua, Wang Lin
Department of Rheumatology and Nephrology, The First Hospital of Changsha, Changsha, 410005, China.
Department of Rheumatology and Immunology, The Qingdao University Medical College Affiliated Yantai Yuhuangding Hospital, Yudong Road 20th, Yantai, 264000, China.
Clin Rheumatol. 2025 May 8. doi: 10.1007/s10067-025-07465-1.
Our pilot study investigated the efficacy and safety of tofacitinib (TOF) in patients with macrophage activation syndrome (MAS), providing a new therapeutic option.
This was a historical comparator, single-center study. Patients enrolled in our study were screened using the 2016 Pediatric Rheumatology International Trials Organization (PRINTO) criteria of MAS. MAS patients deemed suitable for TOF therapy were enrolled in the experimental group. The control group included MAS patients from Yantai Yuhuangding Hospital who had received biological and/or conventional synthetic disease-modifying anti-rheumatic drugs (b/csDMARDs) treatment in a similar period. Clinical characteristics, laboratory test results, MAS treatment response rates, and glucocorticoid discontinuation rates were compared between the two groups before and after treatment. Serious adverse events such as thrombosis or severe infection were recorded.
A total of 36 MAS patients were included-17 in the TOF treatment group and 19 in the b/csDMARDs control group. Baseline demographic and clinical characteristics were comparable between groups. After six weeks, the proportion of febrile patients was significantly lower in the TOF group compared to the control group (11.8% [2/17] vs. 47.3% [9/19], P = 0.021). Significant reductions were also observed in C-reactive protein levels at week 6 (5.9% [1/17] vs. 36.8% [7/19], P = 0.026) and ferritin levels at week 12 (100.0% [17/17] vs. 26.3% [5/19], P = 0.023). The MAS treatment response rate was significantly higher in the TOF group at weeks 2 and 6 (P = 0.029 and P = 0.023, respectively). Glucocorticoid discontinuation was more frequent in the TOF group, with a 12-week discontinuation rate of 100.0% (17/17) compared to 57.9% (11/19) in the control group (P = 0.002). No serious adverse events, including thrombosis or severe infections, were reported.
Our results suggest that TOF may be as effective as or even better than b/csDMARDs, with a quicker and higher response rate in patients with MAS without severe adverse events. Key Points •Tofacitinib may be as effective or better than b/csDMARDs in MAS. •Tofacitinib may act at a quicker and higher response rate in MAS. •Tofacitinib might be an alternative therapeutic option for patients with MAS.
我们的初步研究调查了托法替布（TOF）在巨噬细胞活化综合征（MAS）患者中的疗效和安全性，提供了一种新的治疗选择。
这是一项历史性对照单中心研究。我们研究中纳入的患者采用2016年国际儿科风湿病试验组织（PRINTO）的MAS标准进行筛查。被认为适合TOF治疗的MAS患者纳入实验组。对照组包括同期在烟台毓璜顶医院接受生物和/或传统合成改善病情抗风湿药物（b/csDMARDs）治疗的MAS患者。比较两组治疗前后的临床特征、实验室检查结果、MAS治疗反应率和糖皮质激素停用率。记录血栓形成或严重感染等严重不良事件。
共纳入36例MAS患者，TOF治疗组17例，b/csDMARDs对照组19例。两组间基线人口统计学和临床特征具有可比性。六周后，TOF组发热患者的比例显著低于对照组（11.8%[2/17]对47.3%[9/19]，P = 0.021）。在第6周时C反应蛋白水平也显著降低（5.9%[1/17]对36.8%[7/19]，P = 0.026），在第12周时铁蛋白水平显著降低（100.0%[17/17]对26.3%[5/19]，P = 0.023）。在第2周和第6周时，TOF组的MAS治疗反应率显著更高（分别为P = 0.029和P = 0.023）。TOF组糖皮质激素停用更频繁，12周停用率为100.0%（17/17），而对照组为57.9%（11/19）（P = 0.002）。未报告包括血栓形成或严重感染在内的严重不良事件。
我们的结果表明，TOF可能与b/csDMARDs一样有效甚至更好，在MAS患者中反应更快、更高，且无严重不良事件。要点•托法替布在MAS中可能与b/csDMARDs一样有效或更好。•托法替布在MAS中可能作用更快、反应率更高。•托法替布可能是MAS患者的一种替代治疗选择。
