Citations 2
Development of a Disease Activity Index for the Assessment of VEXAS Syndrome (VEXAS‐DAI)
Byram, K, Mann, Heřman , Hammond, Danielle, Chowdhury, Onima , Savic, Sinisa , Kirino, Yohei , Heiblig, Maël , Comont, Thibault , Mékinian, A. , Patnaik, Mrinal M., Weeks, Lachelle D., Ho, Gary , Al‐Hakim, Adam , Goldberg, Scott, Grayson, Peter C. , Patel, Bhavisha , Buckley, Sarah A., Harder, Bryan G., Garcha, Raman, Beck, David B. , Mann, H. , Hammond, Danielle , Byram, Kevin W , Hammond, Danielle , Gurnari, Carmelo , Heiblig, Mael , Comont, Thibault , Mékinian, Arsène , Weeks, Lachelle Dawn , Ho, Gary , Al-Hakim, Adam , Ferrada, Marcela A , Georgin-Lavialle, Sophie , Grayson, Peter C , Groarke, Emma M , Sullivan, Megan M , Buckley, Sarah A , Harder, Bryan G , Garcha, Raman , Beck, David B , Koster, Matthew J , Kirino, Yohei , Savic, Sinisa , Gurnari, Carmelo , Patnaik, Mrinal M. , Weeks, Lachelle D. , Ferrada, Marcela A. , Georgin‐Lavialle, Sophie , Sullivan, Megan M. , Harder, Bryan , Koster, Matthew J.
Arthritis Care & Research | July 13, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available
Objective Vacuoles, E1 enzyme, X‐linked, autoinflammatory, somatic syndrome (VEXAS) syndrome is characterized by a complex spectrum of inflammatory and hematologic manifestations. Clinical research to identify effective therapies is urgently needed but is hindered by the lack of validated outcome measures. A VEXAS‐specific disease activity index (DAI) is an essential tool for reliably capturing changes in disease activity over time, thereby supporting clinical research and informing patient care in this emerging disease. This study aimed to develop a comprehensive DAI to assess disease‐related inflammation across affected organ systems in patients with VEXAS syndrome. Methods Inflammatory manifestations of VEXAS were previously identified through a systematic literature review and expert input. A multinational expert advisory committee developed the VEXAS‐DAI using modified Delphi methodology until consensus (defined as ≥75% concurrence) was reached. Item grading was adapted from the Common Terminology Criteria for Adverse Events. Scoring criteria were developed based on 158 test cases using the Physician Global Assessment of Inflammation. Results Consensus on the VEXAS‐DAI was achieved after four Delphi rounds. The index assesses inflammatory activity at the time of evaluation across 12 domains: inflammatory rash, chondritis, periorbital, ophthalmologic, joint, pulmonary, cardiovascular, genitourinary, neurologic, oral and gastrointestinal, renal, and constitutional symptoms. The total score ranges from 0 to 40. Conclusion The VEXAS‐DAI is a novel instrument designed to quantify active inflammation over time in patients with VEXAS. Prospective validation within a clinical trial setting is ongoing. image
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
Beck, David B. , Heiblig, Maël , Savic, S., Ferrada, Marcela A. , Mekinian, Arsène, Chowdhury, Onima , Hammond, Danielle E., Weeks, Lachelle D., Gurnari, Carmelo , Kirino, Yohei , Georgin-Lavialle, Sophie, Buckley, Sarah A., Garcha, Raman, Harder, Bryan G., Harder, Bryan , Weeks, Lachelle D. , Mékinian, A. , Savic, Sinisa , Hammond, Danielle , Georgin‐Lavialle, Sophie , Savic, Sinisa , Mékinian, Arsène , Hammond, Danielle , Weeks, Lachelle Dawn , Georgin-Lavialle, Sophie , Buckley, Sarah A , Harder, Bryan G , Koster, Matthew J , Gurnari, Carmelo , Kirino, Yohei , Heiblig, Mael , Ferrada, Marcela A , Koster, Matthew J.
Journal of Clinical Medicine | February 11, 2026 | Volume: 15 | Issue: 4 | Pages: 1426
VEXAS (Vacuoles, E1 ubiquitin-activating enzyme, X-linked, Autoinflammatory, Somatic) syndrome is a systemic disorder characterized by an overlap of hematologic and inflammatory features. Most patients require chronic use of moderate-to-high doses of glucocorticoids (GCs) to maintain disease control. Data on GC-sparing therapies is limited, and there have been no prospective pharmacotherapeutic trials in VEXAS syndrome published to date. Pacritinib, an oral inhibitor of IRAK1, JAK2, and ACVR1, has emerged as a promising therapeutic option for VEXAS syndrome. The PAXIS trial is the first prospective, randomized pharmacotherapeutic study conducted in this rare and severe disease. Utilizing a novel study design and disease-specific endpoints, the trial will evaluate the efficacy and safety of two dose levels of pacritinib compared with placebo in patients with VEXAS syndrome (NCT06782373, EUCTR: 2024-516347-41-00).