OBJECTIVES: To investigate the effects of thalidomide in preventing disease relapse with 'zero' glucocorticoids (GCs) usage in IgG4-related disease (IgG4-RD). METHODS: This was a multicentre, randomised, double-blinded, placebo-controlled study, in which eligible patients in disease active status were randomised into 2 groups (group 1: GCs+Thalidomide
group 2: GCs+Placebo) at a 1:1 ratio. The primary outcome of this trial was the disease relapse rate at month 12, whereas the secondary outcomes were the disease remission rate at month 12 and the incidence of adverse events (AEs). RESULTS: A total of 60 patients were randomised, and 57 patients (GCs+Thalidomide: 29
GCs+Placebo: 28) finished the study per protocol. The relapse rates of the GCs+Thalidomide and GCs+Placebo groups at month 12 were 13.8% and 67.8%, respectively. A 100% response rate was observed in both treatment group, while the remission rates of the GCs+Thalidomide and GCs+Placebo groups were 75.8% and 32.1%, respectively. In total. 49 AEs were recorded in 35 participants, in which 4 were graded as moderate, and 45 were graded as mild. The risk-benefit analysis based on the evaluation of rates of disease relapse and moderate AEs within the 12-month follow-up showed a NNT (number needed to treat) of 2 and a NNH (number needed to harm) of 8 for thalidomide treatment. CONCLUSIONS: Thalidomide can effectively prevent relapse in IgG4-RD outweighing its side effects, which indicating that thalidomide can be a potential safe therapeutic option for disease relapse prevention in parallel with steroid sparing in IgG4-RD.