Objective To investigate efficacy and safety of tofacitinib combined with Disease-modifying Antirhrumatic Drugs (DMARDs) in treatment of patients with refractory moderate-to-severe rheumatoid arthritis (RA).

Methods The clinical data of forty-seven patients with moderate-to-severe RA who had been treated with two or multiple kinds of DMARDs or more than one biological DMARDs for more than six months previously were analyzed. All patients were orally given tofacitinib for 5 mg every time, twice daily, the patients were given low-dose hormones or one or two kinds of DMARDs for treatment according to their conditions. The general data of the patients, relevant laboratory indicators, disease assessment indicators and the occurrence of adverse reactions before treatment, after 4, 12 and 24 weeks of treatment were recorded and observed.

Results After 4, 12 and 24 weeks of treatment, total cholesterol (TC) and high-density lipoprotein (HDL) were higher than before treatment, and platelet (PLT) after 24 weeks of treatment was lower than before treatment, and the differences were statistically significant (P < 0.05). Compared with before treatment, the swollen joint number (SJC), tender joint Number (TJC), C-reactive protein (CRP), Simplified Disease Activity Index (SDAI), Rheumatoid Arthritis Disease Activity Score in 28 Joints (DAS28) score and Clinical Disease Activity Index (CDAI) after 4, 12 and 24 weeks of treatment were statistically significant (P < 0.05). Compared with before treatment, erythrocyte sedimentation rate (ESR) after 12 and 24 weeks of treatment showed statistically significant differences(P < 0.05). Only 5 patients were treated with hormones after 24 weeks of treatment, and the hormone dosage decreased compared with before treatment, and the difference was statistically significant (P < 0.05). After 4, 12 and 24 weeks of treatment, the number of DMARDs combination usage decreased compared with before treatment, and the difference was statistically significant (P < 0.05). Only one patient developed herpes zoster after 24 weeks of treatment, three patients developed mildly elevated transaminase after 4 weeks of treatment, four patients developed mildly elevated transaminase after 12 weeks of treatment, and eight patients developed mildly elevated transaminase after 24 weeks of treatment. But their liver function improved after hepatoprotective treatment with appropriate drugs. No serious adverse reactions such as leukopenia, thrombocytopenia, cardiac insufficiency and allergy occurred.

Conclusion For patients with refractory moderate to severe RA, the application of tofacitinib can significantly improve clinical symptoms, signs, disease activity and other indicators, reduce the incidence of adverse reactions, and have a high safety. Therefore, tofacitinibis is a new choice for the treatment of refractory RA.