First ACR Guidance Statement for VEXAS Released

Like all ACR guidance documents, this initial VEXAS guidance will evolve as new information becomes available. For example, a study of the JAK inhibitor pacritinib in patients with VEXAS is now open—the first randomized, double-blind, placebo-controlled trial for the disease.⁴

“In less than five years we’ve gone from an initial index cohort to rapid escalation of knowledge, with national databases and an international, multispecialty consensus document,” says Dr. Koster. “That’s phenomenal. We’re going into a hyper-accelerated timeframe of identifying diseases, collaborating internationally and moving toward dedicated clinical trials.”

Ruth Jessen Hickman, MD, a graduate of the Indiana University School of Medicine, is a medical and science writer in Bloomington, Ind.

References

1. Mekinian AM, Georgin-Lavaille S, Ferrada MA, et al. American College of Rheumatology guidance statement for diagnosis and management of VEXAS developed by the international VEXAS working group expert panel. Arthritis Rheumatol. 2025 Aug 11. Online ahead of print.
2. Beck DB, Ferrada MA, Sikora KA, et al. Somatic mutations in UBA1 and severe adult-onset autoinflammatory disease. N Engl J Med. 2020 Dec 31;383(27):2628–2638.
3. Jachiet V, Kosmider O, Beydon M, et al. Efficacy and safety of azacitidine for VEXAS syndrome: A large-scale retrospective study from FRENVEX. Blood. 2025 Sep 18;146(12):1450–1461.
4. Beck DB, Heiblig M, Savic S, et al. POS0379 PAXIS: A randomized, double-blind, placebo-controlled dose finding phase 2 study (part 1) followed by an open-label period (part 2) to assess the efficacy and safety of pacritinib in patients with VEXAS syndrome [abstract]. Ann Rheum Dis. June 2025;84(S1):623–624.