Department of Rheumatology, Guang'anmen Hospital, China Academy of Chinese Medical Sciences, Beijing 100032, PR China; Beijing University of Chinese Medicine, Beijing 100029, PR China.
Peking University China-Japan Friendship school of clinical medicine, Beijing, PR China.
Semin Arthritis Rheum. 2024 Oct;68:152474. doi: 10.1016/j.semarthrit.2024.152474. Epub 2024 Jun 3.
To evaluate the efficacy and safety of Janus kinase inhibitors (JAKi) in the treatment of refractory anti-synthetase syndrome (ASS) in real-world clinical settings.
The medical records of all refractory ASS patients who were treated with JAKi from October 2020 to June 2023 were retrospectively reviewed.
Twenty patients were included, and all (100 %) patients had interstitial lung disease (ILD). After treatment with JAKi, 14 (70 %) of the refractory ASS patients showed significant improvement in clinical manifestations, including arthritis (56.3 % vs. 6.3 %, p = 0.002), rash (77.8 % vs. 27.8 %, p = 0.012), shortness of breath (55.6 % vs. 16.7 %, p = 0.039), cough (61.1 % vs. 11.1 %, p = 0.012). Improvement was noted for myalgia (50 % vs. 11.1 %, p = 0.016) and muscular weakness (61.1 % vs. 11.1 %, p = 0.012), while creatine kinase (CK) levels, which were abnormally elevated in five patients prior treatment, were significantly lowered (1096 ± 1042.98 IU/L vs. 199.2 ± 144.66 IU/L, p = 0.043). A decrease in levels of inflammatory markers, including erythrocyte sedimentation rate (ESR) (p = 0.001) and C-reactive protein (CRP) (p = 0.023) was observed in the patients. In ASS-ILD, the CT score reduced (188.75 ± 69.67 vs. 156.35 ± 74.62, p = 0.001). Furthermore, the glucocorticoid dose significantly reduced (21.42 ± 13.26 mg vs. 11.32 ± 8.59 mg; p = 0.001).
JAKi were effective in most refractory ASS patients as evidenced by improved skin rash, myositis, and ILD. However, larger prospective controlled studies are required to evaluate its efficacy.
评估 Janus 激酶抑制剂 (JAKi) 在真实临床环境中治疗难治性抗合成酶综合征 (ASS) 的疗效和安全性。
回顾性分析 2020 年 10 月至 2023 年 6 月期间接受 JAKi 治疗的所有难治性 ASS 患者的病历。
共纳入 20 例患者，所有患者（100%）均存在间质性肺病 (ILD)。接受 JAKi 治疗后，14 例（70%）难治性 ASS 患者的临床表现有显著改善，包括关节炎（56.3% vs. 6.3%，p=0.002）、皮疹（77.8% vs. 27.8%，p=0.012）、呼吸急促（55.6% vs. 16.7%，p=0.039）、咳嗽（61.1% vs. 11.1%，p=0.012）。肌痛（50% vs. 11.1%，p=0.016）和肌无力（61.1% vs. 11.1%，p=0.012）也有改善，而治疗前 5 例患者异常升高的肌酸激酶 (CK) 水平显著降低（1096±1042.98 IU/L vs. 199.2±144.66 IU/L，p=0.043）。患者的炎症标志物水平，包括红细胞沉降率 (ESR)（p=0.001）和 C 反应蛋白 (CRP)（p=0.023）也有所下降。在 ASS-ILD 中，CT 评分降低（188.75±69.67 vs. 156.35±74.62，p=0.001）。此外，糖皮质激素剂量显著减少（21.42±13.26 mg vs. 11.32±8.59 mg；p=0.001）。
JAKi 对大多数难治性 ASS 患者有效，表现为皮疹、肌炎和 ILD 改善。然而，需要更大规模的前瞻性对照研究来评估其疗效。
