Background: Adult-onset Still’s disease (AOSD) is a rare multigenic systemic autoinflammatory disease with unknown etiology. It has highly heterogeneous manifestations with different clinical phenotypes and an unclear pathology. About 30% are refractory to conventional therapy with corticosteroids and conventional synthetic disease-modifying antirheumatic drugs, including methotrexate and cyclosporine. New successful therapeutic options are needed to ameliorate systemic inflammation and arthritis. Patients with AOSD present high levels of pro-inflammatory cytokines that signal through the JAK-STAT pathway and JAK inhibitors could further improve the AOSD therapeutic strategy.

Case presentation: We report two patients with refractory AOSD that were successfully treated with tofacitinib. Patient 1 had a systemic form with high disease activity and did not respond adequately to corticosteroids and methotrexate, however, the early addition of tofacitinib resulted in substantial improvement. Patient 2 presented a chronic overlapping pattern, with systemic and chronic erosive arthritis course, also had an insufficient response to corticosteroids, methotrexate-leflunomide, and adalimumab. He was rescued with long-term tofacitinib monotherapy and showed improvement of their articular synovitis and inhibition of radiographic progression.

Learning points for clinical practice: Both patients were rescued with tofacitinib, showing an improved prognosis without major side effects. The patient 1 completed 2 years with therapy, while patient with chronic arthritis completed 3.8 years with tofacitinib therapy. The results support that tofacitinib can be effective for patients refractory to conventional treatment and contribute to disease remission.

REFERENCES: NIL.

Acknowledgements: We thank all the patients who participated in this study.

Disclosure of Interests: None declared.