Tofacitinib Treatment in Children With Rheumatic Diseases: Single-Center Experience

The study evaluated the safety and efficacy of tofacitinib (TOF) in 17 children with immune-mediated diseases, including juvenile idiopathic arthritis (JIA), autoinflammatory diseases (AID), and juvenile dermatomyositis (JDM). The mean duration of TOF treatment was 25.4 months. TOF was used as monotherapy in 3 cases, in combination with methotrexate in 6, and with other biologics in 3. Nine patients received corticosteroids (CS). TOF induced hair growth in two JIA patients with alopecia and controlled joint inflammation. The treatment outcomes were 9 complete responses (CR), 7 partial responses (PR), and 1 no response (NR). TOF allowed for the discontinuation or reduction of CS in several patients. Four patients experienced side effects that did not require discontinuation of treatment. The study concluded that TOF is a promising treatment for pediatric rheumatic diseases, especially in AID patients with rare alleles in interferon pathway genes, patients with arthritis and alopecia, and children with JDM. Further studies are needed to establish clear indications for JAK-inhibitor treatment in this population.