Department of internal medicine and clinical immunology, Centre de compétence des maladies auto-immunes systémiques rares, CHU de Nancy, Université de Lorraine, 5 Rue du Morvan, Vandoeuvre-lès-Nancy 54500, France.
Department of internal medicine and clinical immunology, Centre de compétence des maladies auto-immunes systémiques rares, CHU de Nancy, Université de Lorraine, 5 Rue du Morvan, Vandoeuvre-lès-Nancy 54500, France; UMR 7365, IMoPA, Université de Lorraine, CNRS, Nancy, France.
Autoimmun Rev. 2022 Oct;21(10):103168. doi: 10.1016/j.autrev.2022.103168. Epub 2022 Aug 6.
Systemic sclerosis (SSc) is a systemic autoimmune disease with heterogeneous clinical presentation and prognosis. JAK inhibitors reduced cutaneous and pulmonary fibrosis in mice models of SSc. Clinical data regarding the efficacy and safety of JAK inhibitors in SSc patients are scarce.
We performed a systematic literature review of patients with SSc defined by the 2013 ACR/EULAR criteria and treated with JAK inhibitors, searching in Medline, Cochrane library and Embase databases.
Fifty-nine patients (mean age 47 ± 15 years) were included. Median treatment duration was 12 [6-12] months. JAK inhibitors (tofacitinib in 47 patients and baricitinib in 12 patients) were prescribed as first line therapy in 35 patients (59%). A significant cutaneous response (decrease in the mRSS - modified Rodnan skin score - of >5 points and ≥ 25% from baseline) was reported in 52 patients (88%). Among patients with interstitial lung disease (ILD) (n = 31), 28/29 patients had no ILD progression during follow-up time (missing data in 2 patients). Only 2 patients had a disease progression during treatment (including one patient with progressive skin fibrosis). Cutaneous response was more frequently observed in treatment naïve SSc patients. Decrease of the mRSS after treatment initiation was more significant in treatment naïve SSc patients. Eighteen non-serious side-effects were noted in 12 patients (20%), without treatment interruption: 6 infections, 6 gastrointestinal disorders, 4 hepatitis and 3 dyslipidemias.
JAK inhibitors could represent a safe and effective treatment option for skin fibrosis and ILD in systemic sclerosis.
系统性硬化症（SSc）是一种系统性自身免疫性疾病，具有异质性的临床表现和预后。JAK 抑制剂可减少 SSc 小鼠模型的皮肤和肺纤维化。关于 JAK 抑制剂在 SSc 患者中的疗效和安全性的临床数据很少。
我们对 2013 年 ACR/EULAR 标准定义的接受 JAK 抑制剂治疗的 SSc 患者进行了系统的文献复习，检索了 Medline、Cochrane 图书馆和 Embase 数据库。
共纳入 59 例患者（平均年龄 47±15 岁）。中位治疗时间为 12[6-12]个月。JAK 抑制剂（托法替尼 47 例，巴瑞替尼 12 例）一线治疗 35 例（59%）。52 例（88%）患者报告皮肤明显改善（mRSS-改良 Rodnan 皮肤评分-较基线下降>5 分且≥25%）。31 例间质性肺病（ILD）患者中，28/29 例在随访期间无 ILD 进展（2 例数据缺失）。仅 2 例患者在治疗期间疾病进展（包括 1 例皮肤纤维化进展）。治疗初治的 SSc 患者更常出现皮肤反应。治疗初治的 SSc 患者治疗后 mRSS 下降更显著。12 例（20%）患者发生 18 例非严重不良反应，无治疗中断：6 例感染，6 例胃肠道疾病，4 例肝炎和 3 例血脂异常。
JAK 抑制剂可能是系统性硬化症皮肤纤维化和ILD 的安全有效治疗选择。
