Emerging treatments

Janus kinase (JAK) inhibitors

JAK inhibitors are increasingly used under specialist management in people with refractory AOSD, although this depends on local expertise. There is as yet limited evidence for their efficacy with trials currently planned or underway.[4][73]​​​​​​ In one observational study of 14 patients with refractory AOSD, tofacitinib achieved complete remission in 50% and significantly reduced the average daily dose of corticosteroid required.​[95]​ Baricitinib has been found to induce clinical remission in patients with AOSD who had been refractory to interleukin (IL)-1 and IL-6 inhibitors.[96] JAK inhibitors are associated with an increased risk of malignancy, major cardiovascular events, venous thromboembolism, infections, and mortality.[97][98][99]

Interleukin (IL)-18 inhibitors

IL-18 inhibitors such as tadekinig alfa (a recombinant IL-18 binding protein with a high affinity for IL-18) have been found to have a favourable safety profile and produced a positive response in 50% of participants in a phase 2 trial.[100]​ There is as yet limited evidence for their efficacy with trials currently planned or underway.[4][73]​​

Rituximab

Rituximab, an anti-CD20 monoclonal antibody, has been shown in a few case studies to have potential effectiveness for refractory AOSD. It blocks T-cell activation and the generation of pro-inflammatory cytokines.[4]

Use of this content is subject to our disclaimer