Tofacitinib in refractory adult-onset Still’s disease: 14 cases from a single centre in China

Adult-onset Still’s disease (AOSD) is an autoinflammatory disease characterised by spiking fever, rash, polyarthralgia, sore throat and even life-threatening complications, such as macrophage activation syndrome and fulminant hepatitis. Excessive and inappropriate production of cytokines is a cornerstone in AOSD pathogenesis.1 Unlike anakinra and tocilizumab, Janus kinases (JAK) inhibitors block the proinflammatory effect of a wide range of cytokines. This range of activity could be beneficial in AOSD patients who are refractory to or intolerant of treatment with biologicals. Anti-interleukin 1 (IL-1) agents are not available in mainland China. Tofacitinib, a JAK1/3 inhibitor, has been proven efficacious in several inflammatory diseases, such as rheumatoid arthritis, systemic lupus erythematosus and psoriasis arthritis.2 To our interest, a case report observed that tofacitinib could ameliorate arthritis in a 13-year-old girl with recalcitrant systemic juvenile idiopathic arthritis,3 which is the juvenile counterpart of AOSD.4 Moreover, a JAK1/2 inhibitor, baricitinib has been reported effective in a 43-year-old patient with refractory AOSD.5 Therefore, JAK inhibitors may be a novel therapeutic approach for refractory AOSD. In our study, we aim to describe, to our knowledge for the first time, the efficacy of tofacitinib in 14 …