| Objective To assess the efficacy and safety of tofacitinib in the treatment of dermatomyositis (DM). Methods This retrospective cohort analysis included 153 patients with DM who were hospitalized at Nanjing Drum Tower Hospital between January 2018 and May 2023. According to the use of tofacitinib or not, patients were categorized into the observation group (treated with tofacitinib, n=65) and the control group (not treated with tofacitinib, n=88), with all patients followed up. The efficacy and safety of tofacitinib in the treatment of DM were assessed based on quantitative analysis of high-resolution computed tomography (HRCT) to evaluate the regression in interstitial lung disease (ILD) in the two groups. The survival rate was compared, the mortality rate of patients with different demographic data was counted, and the incidence of post-treatment adverse effects was recorded. Results Except for the positive rate of anti-melanoma differentiation-associated gene 5 (MDA5) antibody, there was no statistically significant difference in the general information between the two groups (P>0.05). KaplanMeier survival curves showed that the five-year survival rate was higher in the observation group than in the control group (P=0.041). Especially in patients with skin involvement (HR=0.238, 95%CI: 0.068-0.830, P=0.024), muscle involvement (HR=0.127, 95%CI: 0.016-0.989, P= 0.049), and higher erythrocyte sedimentation rate (HR=0.219, 95%CI: 0.062-0.771, P=0.018), the use of tofacitinib was effective in reducing the mortality rate. Based on quantitative HRCT analysis to assess ILD condition, the difference in the percentage of baseline whole lung lesion volume between the two groups was statistically significant (P=0.024). The decreased percentage of whole-lung lesions volume after treatment was higher in the observation group than in the control group (P=0.015). However, tofacitinib increased the risk of EBV infection (P=0.027). Conclusion Tofacitinib is effective in controlling interstitial pneumonia and improving survival rates in patients with DM, and may be a potential therapeutic option for the treatment of DM. However, attention should be paid to the occurrence of infections during treatment. Quantitative HRCT analysis can be used to assess the severity of lung lesions in patients with DM-ILD. |