Beckett Madelaine, Tan Julia, Bonnardeaux Evelyne, Dutz Jan, Shojania Kamran, To Fergus, Obrzut Anthony, Aviña-Zubieta J, Huang Kun
Department of Medicine, University of British Columbia, Vancouver, BC, Canada.
Division of Rheumatology, Department of Medicine, University of British Columbia, Vancouver, BC, Canada.
Rheumatology (Oxford). 2024 May 2;63(5):1432-1436. doi: 10.1093/rheumatology/kead404.
To evaluate the efficacy and safety of tofacitinib in treatment-refractory inflammatory myositis in a real-world clinical setting.
All patients with refractory inflammatory myositis treated with tofacitinib from a single urban centre in Vancouver, British Columbia, Canada, were included from June 2016 to December 2022. The medical records of these patients were retrospectively reviewed.
A total of 41 patients were included, 23 with classic DM, 12 with amyopathic DM (ADM) and six with PM phenotype. The patients failed an average of four to five non-steroidal immunosuppressants before initiation of tofacitinib. In the classic DM and ADM group, tofacitinib offered clinically and statistically significant cutaneous improvement. In all myositis patients including the PM phenotype, no meaningful muscle strength response to tofacitinib was observed. 53.7% of the patients discontinued tofacitinib due to lack of benefit or death. Of the 19 patients who remained on tofacitinib at the conclusion of this study, tofacitinib demonstrated clinically and statistically significant improvement in cutaneous disease activity.
Tofacitinib appears to be highly effective in targeting cutaneous manifestations in classic DM and ADM; however, minimal benefit in muscle strength in the DM or PM phenotype were observed.
在真实临床环境中评估托法替布治疗难治性炎性肌病的疗效和安全性。
纳入2016年6月至2022年12月期间在加拿大不列颠哥伦比亚省温哥华市一个城市中心接受托法替布治疗的难治性炎性肌病患者。对这些患者的病历进行回顾性审查。
共纳入41例患者，其中23例为典型皮肌炎（DM），12例为无肌病性皮肌炎（ADM），6例为多发性肌炎（PM）表型。患者在开始使用托法替布之前平均使用了四到五种非甾体类免疫抑制剂均治疗失败。在典型DM和ADM组中，托法替布在临床和统计学上均使皮肤状况得到显著改善。在所有肌炎患者（包括PM表型）中，未观察到托法替布对肌肉力量有明显反应。53.7%的患者因缺乏疗效或死亡而停用托法替布。在本研究结束时仍在使用托法替布的19例患者中，托法替布在临床和统计学上均使皮肤疾病活动度得到显著改善。
托法替布似乎对典型DM和ADM的皮肤表现具有高效性；然而，在DM或PM表型中对肌肉力量的益处甚微。
