Internal Medicine, The University of British Columbia Faculty of Medicine, Vancouver, British Columbia, Canada.
Dermatology, The University of British Columbia Faculty of Medicine, Vancouver, British Columbia, Canada.
RMD Open. 2024 Jan 18;10(1):e003837. doi: 10.1136/rmdopen-2023-003837.
To evaluate the effectiveness and safety of upadacitinib in treatment-refractory inflammatory myositis.
Patients with refractory inflammatory myositis treated with upadacitinib from a single urban centre in Vancouver, British Columbia, Canada, were included from September 2020 to June 2023. The medical records of these patients were retrospectively reviewed.
10 total patients were identified for review, including 5 classic dermatomyositis (DM), 3 amyopathic DM (ADM) and 2 antisynthetase syndrome. The patients failed an average of four immunosuppressants before initiation of upadacitinib. Three had prior Janus kinase inhibitor therapy with tofacitinib. In the classic DM and ADM aggregate group, upadacitinib offered clinically and statistically significant cutaneous improvement. Lack of active muscle disease at baseline precluded analysis of the effect of upadacitinib on muscle weakness. Nine patients remained on upadacitinib at the end of the study period. One patient discontinued upadacitinib due to severe facial acne.
Upadacitinib appears to be effective in targeting cutaneous manifestations of refractory inflammatory DM. Further research is still needed to validate its efficacy on a broader population scale.
评估乌帕替尼在治疗难治性炎症性肌病中的疗效和安全性。
纳入 2020 年 9 月至 2023 年 6 月期间，在加拿大不列颠哥伦比亚省温哥华市的一家单一城市中心接受乌帕替尼治疗的难治性炎症性肌病患者。回顾性分析这些患者的病历。
共 10 例患者接受了审查，包括 5 例经典皮肌炎（DM）、3 例无肌病性皮肌炎（ADM）和 2 例抗合成酶综合征。这些患者在开始使用乌帕替尼之前平均失败了四种免疫抑制剂。其中 3 例曾接受过托法替尼的 Janus 激酶抑制剂治疗。在经典 DM 和 ADM 综合组中，乌帕替尼在皮肤方面提供了显著的临床和统计学改善。由于基线时无活动性肌肉疾病，因此无法分析乌帕替尼对肌肉无力的影响。研究结束时，9 例患者仍在接受乌帕替尼治疗。1 例患者因严重面部痤疮而停止使用乌帕替尼。
乌帕替尼似乎对难治性炎症性 DM 的皮肤表现有效。仍需要进一步的研究来验证其在更广泛人群中的疗效。
