A WINDOW OF OPPORTUNITY IN STILL’S DISEASE: THE EARLY INTRODUCTION OF IL-1 INHIBITORS FOR ACHIEVING CLINICAL INACTIVE DISEASE
Abstract format and assignment number: Poster 0377
Presenting author: S. Bindoli (Italy)
Date: Saturday, 14 June 2025, 10:15 – 10:21
Early and late treatment with IL-1 inhibition were compared in patients with adult-onset Still’s disease. Treatment included anakinra or canakinumab. Among 42 patients, 57% were treated within 6 months from disease onset and 43% thereafter. Clinically inactive disease was reached in 67% of the early treatments and in 38% of the late treatments. This difference was not statistically significant.
EFFICACY AND SAFETY OF EMAPALUMAB IN PATIENTS WITH MACROPHAGE ACTIVATION SYNDROME IN STILL’S DISEASE: RESULTS FROM A POOLED ANALYSIS OF TWO PROSPECTIVE TRIALS
Abstract format and assignment number: Oral presentation 0207
Presenting author: MD, PhD Fabrizio De Benedetti (Italy)
Date: Thursday, 12 June 2025, 10:40 – 10:50
The efficacy and safety of emapalumab in patients with macrophage activation syndrome due to adult-onset Still’s disease were assessed, using data from two prospective trials. Emapalumab is an antibody binding free and receptor-bound interferon-gamma. Among 39 patients with a median age of 12 years and a lack of response to high-dose steroids and other biologicals, 85% achieved complete remission at any time, and 82% achieved investigator-assessed clinical remission at any time. Serious treatment-related adverse events were reported in 4 patients.
DEVELOPMENT OF A DISEASE ACTIVITY INDEX FOR THE ASSESSMENT OF VEXAS SYNDROME (VEXAS-DAI)
Abstract format and assignment number: Poster 0381
Presenting author: H. Mann (Czechia)
Date: Saturday, 14 June 2025, 10:39 – 10:45
As an improvement of the VEXASCAF score, a new disease activity index was presented as more sensitive to changes in disease severity. This VEXAS-DAI was developed based on a systematic literature review and expert feedback. It included 12 scored domains, in total ranging from 0 to 40. It is yet to be validated.
HETEROGENEITY OF THE CLINICAL PRESENTATION IN VEXAS SYNDROME: INSIGHTS FROM 318 CASES FROM THE FRENCH VEXAS STUDY GROUP (FRENVEX)
Abstract format and assignment number: Oral presentation 0287
Presenting author: S. Georgin-Lavialle (France)
Date: Friday, 13 June 2025, 11:10 – 11:20
The main presentation of VEXAS at diagnosis in a large multicentric cohort was described. In a large cohort of 318 patients, of whom 97% men, the occurrence of a wide range of different clinical features was counted. Of note, recurrent chondritis and myelodysplastic syndrome were not always present.
ERYTHROID STIMULATING AGENTS IN VEXAS SYNDROME: A MULTICENTER RETROSPECTIVE STUDY OF AN ITALIAN VEXAS COHORT
Abstract format and assignment number: Poster 1108
Presenting author: C. Campochiaro (Italy)
Date: Friday, 13 June 2025, 14:45 – 15:45
Since the VEXAS syndrome manifests with macrocytic anaemia, the efficacy and safety of erythropoiesis stimulating agents (ESAs) were assessed. Among 32 patients treated with ESAs for symptomatic anaemia, response was achieved in 63% and correlated with lower endogenous erythropoietin levels. Unfortunately, 6 patients lost response after a median time of one year. During treatment, 13% experienced deep vein thrombosis.
CAN VARIANTS OF UNKNOWN SIGNIFICANCE (VUS) BE ASSOCIATED WITH A SPECIFIC CLINICAL PHENOTYPE IN AUTOINFLAMMATORY DISEASES
Abstract format and assignment number: Poster 1076
Presenting author: S. Bindoli (Italy)
Date: Friday, 13 June 2025, 14:45 – 15:45
Genetic variants of unknown significance (VUS) were assessed in patients with autoinflammatory diseases. Patients with signs of autoinflammatory disease in one hospital were retrospectively reviewed. Among 103 patients, 34 carried at least one VUS. Two distinct clusters of patients were identified with VUS on NOD2. Patients carrying two VUS across the same or different genes generally experienced a higher burden of inflammatory symptoms and responded well to colchicine or IL-1 inhibitors.
Jacob J.E. Koopman
Jacob is a clinical immunologist who was trained at Erasmus Medical Centre in Rotterdam, Netherlands. He has worked at University Medical Centre Utrecht. His clinical practice and research focus mainly on systemic lupus erythematosus, but additionally, he has interests in inflammatory myopathies, vasculitis, and rare immunological diseases.
He is a member of EMEUNET Newsletter Sub-committee.