Department of Medicine, Oregon Health & Science University, 3181 SW Sam Jackson Park Rd. Mail Code OP09, Portland, OR, 97239, USA.
Division of Pulmonary and Critical Care Medicine, Tuality Healthcare, Hillsboro, OR, 97123, USA.
Lung. 2021 Apr;199(2):147-153. doi: 10.1007/s00408-021-00436-8. Epub 2021 Apr 7.
This is a prospective, open-label, proof-of-concept study of tofacitinib, a Janus kinase inhibitor, as a steroid-sparing therapy in corticosteroid-dependent pulmonary sarcoidosis. Five patients with corticosteroid-dependent pulmonary sarcoidosis were treated with tofacitinib 5 mg twice daily. The primary endpoint was a ≥ 50% reduction in corticosteroids at week 16 with no worsening in pulmonary function or respiratory symptoms. 60% of patients (3/5) met the primary endpoint. One patient was lost to follow up prior to steroid taper, and another was withdrawn due to worsening of known neurosarcoidosis. The three patients who met the primary endpoint each tapered to ≤ 5 mg/day prednisone, respiratory symptoms improved, and spirometry remained stable. In this proof-of-concept study, the addition of a JAK-inhibitor allowed 60% of patients with pulmonary sarcoidosis to successfully taper corticosteroids. JAK-inhibitors are a promising therapy for pulmonary sarcoidosis, which require further investigation in randomized trials.Trial Registration clinicaltrials.gov NCT03793439; registered Jan 4, 2019.
这是一项托法替布（一种 Janus 激酶抑制剂）作为皮质类固醇依赖型肺结节病的皮质类固醇节约治疗的前瞻性、开放标签、概念验证研究。5 例皮质类固醇依赖型肺结节病患者接受托法替布 5mg，每日两次治疗。主要终点是在第 16 周时皮质类固醇减少≥50%，且肺功能或呼吸症状无恶化。60%的患者（3/5）达到主要终点。1 例患者在开始皮质类固醇减量前失访，另 1 例因已知神经结节病恶化而退出。达到主要终点的 3 例患者的泼尼松剂量均降至≤5mg/天，呼吸症状改善，肺功能检查保持稳定。在这项概念验证研究中，添加 JAK 抑制剂使 60%的肺结节病患者成功减少皮质类固醇的用量。JAK 抑制剂是一种有前途的肺结节病治疗方法，需要在随机试验中进一步研究。试验注册临床Trials.gov NCT03793439；2019 年 1 月 4 日注册。
