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Publications

Journal Article Ahead of print

PubMed: 42439317 DOI: 10.1002/acr.80117

Development of a Disease Activity Index for the Assessment of VEXAS Syndrome (VEXAS-DAI).

Arthritis Care Res (Hoboken)

Authors: Kevin Byram, Heřman Mann, Danielle Hammond, Onima Chowdhury, Sinisa Savic, Yohei Kirino, Carmelo Gurnari, Maël Heiblig, Thibault Comont, Arsène Mekinian, Mrinal M Patnaik, Lachelle D Weeks, Gary Ho, Adam Al-Hakim, Scott Goldberg, Marcela A Ferrada, Sophie Georgin-Lavialle, Peter C Grayson, Emma M Groarke, Bhavisha Patel, Megan Sullivan, Sarah A Buckley, Bryan G Harder, Raman Garcha, David B Beck, Matthew J Koster

Affiliations

  • Vanderbilt University Medical Center, Nashville, Tennessee.
  • Institute of Rheumatology and Department of Rheumatology, 1st Faculty of Medicine, Charles University, Prague, Czech Republic.
  • The University of Texas MD Anderson Cancer Center, Houston, Texas.
  • Oxford University Hospitals' NHS Foundation Trust, Oxford, United Kingdom.
  • Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, United Kingdom.
  • University of Leeds, Leeds Institute of Rheumatic and Musculoskeletal Medicine, Leeds, United Kingdom.
  • Yokohama City University Graduate School of Medicine, Yokohama, Japan.
  • Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy.
  • Translational Hematology and Oncology Research Department, Taussig Cancer Center, Cleveland Clinic, Cleveland, Ohio.
  • Lyon-Sud Hospital, Hospices Civils de Lyon, Paris, France.
  • Université Claude Bernard, Lyon, France.
  • University Toulouse, CHU Toulouse, Toulouse, France.
  • AP-HP.Sorbonne Université - Hôpital Saint-Antoine, Paris, France.
  • Mayo Clinic, Rochester, Minnesota.
  • Dana-Farber Cancer Institute, Boston, Massachusetts.
  • NYU Langone Health, New York, New York.
  • NYU Langone Medical Center, New York, New York.
  • University of Maryland School of Medicine, Baltimore, Maryland.
  • Sorbonne University, Tenon Hospital, DMU3ID, FHU INFLAMME, CEREMAIA, ERN RITA, Paris, France.
  • National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Maryland.
  • National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland.
  • Mayo Clinic, Scottsdale, Arizona.
  • Sobi Inc. Waltham, Massachusetts.
  • NYU School of Medicine, New York, New York.

Abstract

OBJECTIVE

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 (CTCAE) criteria. 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, neurological, 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 syndrome. Prospective validation within a clinical trial setting is ongoing.

MeSH

Arthritis, Research