Janus kinase (JAK) inhibitors and interleukin-6 inhibitors may be effective treatments for patients with vacuoles, E1 enzymes, X-linked, autoinflammatory, somatic (VEXAS) syndrome, according to a press release from the American College of Rheumatology (ACR). Standard treatment with high-dose corticosteroids often leads to severe side effects. In a retrospective study presented by Hadjadj et al at ACR Convergence 2023, investigators used the French national VEXAS registry to analyze the outcomes of 110 patients with VEXAS syndrome from November 2020 to August 2023. The patients received either the JAK inhibitor ruxolitinib, the interleukin-6 inhibitor tocilizumab, or other targeted therapies such as the interleukin-1 inhibitor anakinra or tumor necrosis factor (TNF) blockers. The investigators noted that at baseline, the median C-reactive protein level was 39 mg/L and the median prednisone dose was 20 mg/day. After a follow-up of 3 months, patients who received tocilizumab, ruxolitinib, anakinra, and TNF blockers experienced overall response rates of 32%, 24%, 9%, and 0%, respectively. After 6 months, the overall response rates decreased to 26% for tocilizumab and increased to 30% for ruxolitinib. The investigators reported that there was a 28% rate of discontinuation for ruxolitinib and a 69% rate of discontinuation for tocilizumab after a median delay of 7.2 months and 5.1 months, respectively. “It was … surprising that the overall response at 3 and 6 months was similar between JAK [inhibitors] and [interleukin]-6 inhibitors. Our study shows that tocilizumab could be a good alternative, whereas other drugs are not efficient. We also showed that survival without treatment withdrawal was significantly longer with JAK [inhibitors], mainly because of serious adverse events with other targeted therapies,” concluded lead study author Jerome Hadjadj, MD, PhD, an internist at Assistance Publique–Hôpitaux de Paris.