Evidence map›Paper›PMID 40046741›Full record

ArticleFrontiers in pharmacology2025

Efficacy and safety profile of biotechnological agents and Janus kinase inhibitors in VEXAS syndrome: data from the international AIDA Network VEXAS registry.

Antonio Vitale, Valeria Caggiano, Flavia Leone, Andrea Hinojosa-Azaola, Eduardo Martín-Nares, Guillermo Arturo Guaracha-Basañez, Jiram Torres-Ruiz, Perla Ayumi Kawakami-Campos, Pravin Hissaria, Alicia Callisto and 49 more

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In one paragraph

Article in Frontiers in pharmacology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. VEXAS syndrome.Nature reviews. Disease primers · 2026
    Review
  4. Article
  5. Article
  6. Review
  7. Article
  8. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

59 authors.

Antonio Vitale *Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.
Valeria Caggiano *Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.
Flavia LeoneUOC di Reumatologia, Fondazione Policlinico Universitario A Gemelli IRCCS, Università Cattolica del Sacro Cuore, Roma, Italy.
Andrea Hinojosa-AzaolaDepartment of Immunology and Rheumatology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Eduardo Martín-NaresDepartment of Immunology and Rheumatology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Guillermo Arturo Guaracha-BasañezDepartment of Immunology and Rheumatology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Jiram Torres-RuizDepartment of Immunology and Rheumatology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Perla Ayumi Kawakami-CamposDepartment of Ophthalmology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Pravin HissariaDepartment of Clinical Immunology and Allergy, Royal Adelaide Hospital, Adelaide, SA, Australia.
Alicia CallistoDepartment of Clinical Immunology and Allergy, Royal Adelaide Hospital, Adelaide, SA, Australia.
Mark BeecherDepartment of Clinical Immunology and Allergy, Royal Adelaide Hospital, Adelaide, SA, Australia.
Lorenzo DagnaDivision of Immunology, Transplants and Infectious Diseases, Università Vita-Salute San Raffaele, Milan, Italy.
Corrado CampochiaroDivision of Immunology, Transplants and Infectious Diseases, Università Vita-Salute San Raffaele, Milan, Italy.
Alessandro TomelleriDivision of Immunology, Transplants and Infectious Diseases, Università Vita-Salute San Raffaele, Milan, Italy.
Micol FrassiRheumatology and Clinical Immunology, Spedali Civili and Department of Clinical and Experimental Sciences, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Brescia, Brescia, Italy.
Franco FranceschiniRheumatology and Clinical Immunology, Spedali Civili and Department of Clinical and Experimental Sciences, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Brescia, Brescia, Italy.
Francesca CrisafulliRheumatology and Clinical Immunology, Spedali Civili and Department of Clinical and Experimental Sciences, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Brescia, Brescia, Italy.
José Hernández-RodríguezClinical Unit of Autoinflammatory Diseases, Department of Autoimmune Diseases, Institut d'Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Hospital Clínic of Barcelona, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Barcelona, Barcelona, Spain.
Verónica Gómez-CaverzaschiClinical Unit of Autoinflammatory Diseases, Department of Autoimmune Diseases, Institut d'Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Hospital Clínic of Barcelona, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Barcelona, Barcelona, Spain.
Olga AraújoClinical Unit of Autoinflammatory Diseases, Department of Autoimmune Diseases, Institut d'Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Hospital Clínic of Barcelona, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Barcelona, Barcelona, Spain.
Paolo SfrisoRheumatology Unit, Department of Medicine, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Padua, Padua, Italy.
Sara BindoliRheumatology Unit, Department of Medicine, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Padua, Padua, Italy.
Chiara BaggioRheumatology Unit, Department of Medicine, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, University of Padua, Padua, Italy.
Jurgen SotaDepartment of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.
Abdurrahman TufanDepartment of Internal Medicine, Division of Rheumatology, Gazi University Hospital, Ankara, Türkiye.
Hamit KucukDepartment of Internal Medicine, Division of Rheumatology, Gazi University Hospital, Ankara, Türkiye.
Matteo PigaDepartment of Medical Sciences and Public Health, University of Cagliari, Cagliari, Italy.
Alberto CauliDepartment of Medical Sciences and Public Health, University of Cagliari, Cagliari, Italy.
Maria Antonietta D'AgostinoUOC di Reumatologia, Fondazione Policlinico Universitario A Gemelli IRCCS, Università Cattolica del Sacro Cuore, Roma, Italy.
Amato De PaulisDepartment of Translational Medical Sciences, Section of Clinical Immunology, University of Naples Federico II, Naples, Italy.
Ilaria MormileDepartment of Translational Medical Sciences, Section of Clinical Immunology, University of Naples Federico II, Naples, Italy.
Henrique A Mayrink GiardiniRheumatology Division, Faculdade de Medicina, Hospital das Clínicas, Universidade de São Paulo, São Paulo, Brazil.
Rafael Alves CordeiroRheumatology Division, Faculdade de Medicina, Hospital das Clínicas, Universidade de São Paulo, São Paulo, Brazil.
Giuseppe LopalcoDepartment of Precision and Regenerative Medicine and Ionian Area (DiMePRe-J) Policlinic Hospital, University of Bari, Bari, Italy.
Florenzo IannoneDepartment of Precision and Regenerative Medicine and Ionian Area (DiMePRe-J) Policlinic Hospital, University of Bari, Bari, Italy.
Sara MontiDivision of Rheumatology, Fondazione IRCCS Policlinico San Matteo, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, Pavia, Italy.
Carlomaurizio MontecuccoDivision of Rheumatology, Fondazione IRCCS Policlinico San Matteo, European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center, Pavia, Italy.
Guillermo Ruiz-IrastorzaFaculty of Medicine and Nursery, University of the Basque Country, UPV/EHU, Leioa, Biscay, Spain.
Adriana Soto-PeleteiroFaculty of Medicine and Nursery, University of the Basque Country, UPV/EHU, Leioa, Biscay, Spain.
Paola TriggianeseRheumatology, Allergology and Clinical Immunology, Department of Systems Medicine, University of Rome Tor Vergata, Rome, Italy.
Carmelo GurnariDepartment of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy.
Ombretta ViapianaRheumatology Unit, Department of Medicine, University and Azienda Ospedaliera Universitaria Integrata of Verona, Verona, Italy.
Riccardo BixioRheumatology Unit, Department of Medicine, University and Azienda Ospedaliera Universitaria Integrata of Verona, Verona, Italy.
Rosetta VitettaUnit of Rheumatology, ASL VC Sant' Andrea Hospital, Vercelli, Italy.
Guido RoveraUnit of Rheumatology, ASL VC Sant' Andrea Hospital, Vercelli, Italy.
Edoardo ConticiniDepartment of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.
Francesco La TorreDepartment of Pediatrics, Giovanni XXIII Pediatric Hospital, University of Bari, Bari, Italy.
Piero PortincasaClinica Medica "A. Murri", Division of Internal Medicine, Department of Precision and Regenerative Medicine and Ionian Area (DiMePre-J), University of Bari Aldo Moro, Bari, Italy.
Nour JaberClinica Medica "A. Murri", Division of Internal Medicine, Department of Precision and Regenerative Medicine and Ionian Area (DiMePre-J), University of Bari Aldo Moro, Bari, Italy.
Gaafar RagabRheumatology and Clinical Immunology Unit, Internal Medicine Department, Faculty of Medicine, Cairo University, Giza, Egypt.
Amina MaherRheumatology and Clinical Immunology Unit, Internal Medicine Department, Faculty of Medicine, Cairo University, Giza, Egypt.
Ezgi Deniz BatuDepartment of Pediatric Rheumatology, Faculty of Medicine, Hacettepe University, Ankara, Türkiye.
Seza OzenDepartment of Pediatric Rheumatology, Faculty of Medicine, Hacettepe University, Ankara, Türkiye.
Ewa Wiesik-SzewczykDepartment of Internal Medicine, Pneumonology, Allergology and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defense, Military Institute of Medicine, National Research Institute, Warsaw, Poland.
Alejandra de-la-TorreNeuroscience Research Group (NEUROS), NeuroVitae Center, Escuela de Medicina y Ciencias de la Salud, Universidad del Rosario, Bogotá, Colombia.
Alberto BalistreriDepartment of Immunology and Rheumatology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Bruno FredianiDepartment of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.
Claudia FabianiAutoinflammatory and Autoimmune Diseases (RITA) Center Siena, Azienda Ospedaliero-Universitaria Senese European Reference Network (ERN) for Rare Immunodeficiency, Siena, Italy.
Luca CantariniDepartment of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: VEXAS syndrome, a recently identified systemic autoinflammatory disorder, poses new diagnostic and management challenges. Based on experience with other autoinflammatory diseases, anti-interleukin (IL)-1, anti-IL-6, anti-tumor necrosis factor (TNF) biotechnological agents, and Janus kinase inhibitors (JAKis) have been widely employed in VEXAS patients. The aim of this study is to evaluate the global effectiveness and safety of biotechnological agents and JAKis using data from the real-world context. Methods: Clinical, laboratory, and therapeutic data from VEXAS patients were obtained from the international AIDA Network VEXAS registry. Results: In total, 69 VEXAS patients were enrolled in the study. Among them, 12 patients (13 treatment courses) received IL-1 inhibitors, 12 patients (13 treatment courses) were administered anti-IL-6 agents, 8 patients (9 treatment courses) were treated with anti-TNF agents, and 16 patients (17 treatment courses) were treated with JAKis. A complete response was observed in 3 patients (23%) treated with anti-IL-1 agents, 2 patients (15%) receiving IL-6 inhibitors, 1 patient (11%) receiving TNF inhibitors, and 4 patients (23.5%) treated with JAKis. The mean prednisone (or equivalent) dosage significantly decreased during anti-IL-1 treatment (p = 0.01), while glucocorticoids changed during anti-IL-6, anti-TNF, and JAKi treatment in a non-significant fashion. A total of 21 patients experienced adverse events, 3 of which led to death (gut perforation, Legionnaires' disease, and infectious pneumonia) while on JAKis; treatment withdrawal was required for 8 out of 21 patients. Conclusion: IL-1 and IL-6 inhibitors, along with JAKis, represent promising therapeutic options for VEXAS patients, albeit careful monitoring is mandatory to control disease activity and ensure safety.

Indexed as

anakinraanti-TNFcanakinumabJAK inhibitorstocilizumabtreatment

Identifiers

PMID40046741
PMCPMC11879931

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