Spanish cohort of VEXAS syndrome: clinical manifestations, outcome of treatments and novel evidences about


Journal

Annals of the rheumatic diseases
ISSN: 1468-2060
Titre abrégé: Ann Rheum Dis
Pays: England
ID NLM: 0372355

Informations de publication

Date de publication:
12 2023
Historique:
received: 07 06 2023
accepted: 14 08 2023
medline: 13 11 2023
pubmed: 5 9 2023
entrez: 4 9 2023
Statut: ppublish

Résumé

The vacuoles, E1-enzyme, X linked, autoinflammatory and somatic (VEXAS) syndrome is an adult-onset autoinflammatory disease (AID) due to postzygotic To investigate the presence of VEXAS syndrome among patients with adult-onset undiagnosed AID. Additional studies evaluated the mosaicism distribution and the circulating cytokines. Gene analyses were performed by both Sanger and amplicon-based deep sequencing. Patients' data were collected from their medical charts. Cytokines were quantified by Luminex. Genetic analyses of enrolled patients (n=42) identified 30 patients carrying Thirty patients with adult-onset AID were definitively diagnosed with VEXAS syndrome through genetic analyses. Despite minor interindividual differences, their main characteristics were in concordance with previous reports. We detected for the first time the

Sections du résumé

BACKGROUND
The vacuoles, E1-enzyme, X linked, autoinflammatory and somatic (VEXAS) syndrome is an adult-onset autoinflammatory disease (AID) due to postzygotic
OBJECTIVES
To investigate the presence of VEXAS syndrome among patients with adult-onset undiagnosed AID. Additional studies evaluated the mosaicism distribution and the circulating cytokines.
METHODS
Gene analyses were performed by both Sanger and amplicon-based deep sequencing. Patients' data were collected from their medical charts. Cytokines were quantified by Luminex.
RESULTS
Genetic analyses of enrolled patients (n=42) identified 30 patients carrying
CONCLUSION
Thirty patients with adult-onset AID were definitively diagnosed with VEXAS syndrome through genetic analyses. Despite minor interindividual differences, their main characteristics were in concordance with previous reports. We detected for the first time the

Identifiants

pubmed: 37666646
pii: ard-2023-224460
doi: 10.1136/ard-2023-224460
pmc: PMC10646843
doi:

Substances chimiques

Cytokines 0
Ferritins 9007-73-2
Glucocorticoids 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1594-1605

Informations de copyright

© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: None declared.

Références

Br J Haematol. 2022 Dec;199(5):777-781
pubmed: 36184391
J Allergy Clin Immunol. 2022 Jan;149(1):432-439.e4
pubmed: 34048852
Nat Rev Rheumatol. 2021 Jul;17(7):405-425
pubmed: 34035534
JAMA. 2023 Jan 24;329(4):318-324
pubmed: 36692560
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
Arthritis Rheumatol. 2016 Dec;68(12):3035-3041
pubmed: 27273849
Blood Adv. 2018 Nov 27;2(22):3404-3410
pubmed: 30482770
Blood Adv. 2022 Feb 8;6(3):998-1003
pubmed: 34714914
Front Immunol. 2017 Oct 31;8:1410
pubmed: 29163488
Blood. 2015 Jul 2;126(1):9-16
pubmed: 25931582
Arthritis Rheumatol. 2022 Apr;74(4):692-699
pubmed: 34672126
Blood. 2021 Jul 1;137(26):3591-3594
pubmed: 33971000
Bone Marrow Transplant. 2022 Feb;57(2):315-318
pubmed: 34999727
N Engl J Med. 2020 Dec 31;383(27):2628-2638
pubmed: 33108101
Br J Dermatol. 2022 Mar;186(3):564-574
pubmed: 34632574
Rheumatology (Oxford). 2021 Dec 1;60(12):e435-e437
pubmed: 34213531
J Allergy Clin Immunol. 2019 Jan;143(1):359-368
pubmed: 30273710
Rheumatology (Oxford). 2022 May 5;61(5):e117-e119
pubmed: 34894213
Nature. 2015 Oct 1;526(7571):68-74
pubmed: 26432245
Blood. 2021 Jul 1;137(26):3676-3681
pubmed: 33690815
JAMA Dermatol. 2021 Nov 01;157(11):1349-1354
pubmed: 34495287
Mol Genet Genomic Med. 2019 Aug;7(8):e791
pubmed: 31397119
Br J Haematol. 2022 Feb;196(4):969-974
pubmed: 34651299
Arthritis Rheumatol. 2015 Sep;67(9):2482-6
pubmed: 25988971

Auteurs

Jose Manuel Mascaro (JM)

Department of Dermatology, Hospital Clínic, Barcelona, Spain.
Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.

Ignasi Rodriguez-Pinto (I)

Department of Internal Medicine, Hospital Mútua de Terrassa, Terrassa, Spain.

Gabriela Poza (G)

Department of Internal Medicine, Hospital Universitario Virgen de la Arrixaca, Murcia, Spain.

Anna Mensa-Vilaro (A)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
Department of Immunology, Hospital Clínic, Barcelona, Spain.

Julian Fernandez-Martin (J)

Department of Internal Medicine, Hospital Álvaro Cunqueiro, Vigo, Spain.

Luis Caminal-Montero (L)

Department of Internal Medicine, Hospital Universitario Central de Asturias, Oviedo, Spain.
Instituto de Investigación Sanitaria del Principado de Asturias, Oviedo, Spain.

Gerard Espinosa (G)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Autoimmune Diseases, Hospital Clinic de Barcelona, Barcelona, Spain.

Jose Hernández-Rodríguez (J)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Autoimmune Diseases, Hospital Clinic de Barcelona, Barcelona, Spain.

Marina Diaz (M)

Department of Haematology, Hospital Clínic, Barcelona, Spain.

Joana Rita-Marques (J)

Department of Internal Medicine, Hospital Universitari Vall d'Hebron, Barcelona, Spain.

Raimon Sanmarti (R)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Rheumatology, Hospital Clínic, Barcelona, Spain.

Santos Castañeda (S)

Department of Rheumatology, Hospital Universitario La Princesa, Madrid, Spain.
Instituto de Investigaciones Sanitarias Princesa (IIS-Princesa), Madrid, Spain.
Cátedra UAM-Roche, EPID-Future, Department of Medicine, School of Medicine, Universidad Autónoma de Madrid, Madrid, Spain.

Dolores Colunga (D)

Department of Internal Medicine, Hospital Universitario Central de Asturias, Oviedo, Spain.

Rubén Coto-Hernández (R)

Department of Internal Medicine, Hospital Universitario Central de Asturias, Oviedo, Spain.
Instituto de Investigación Sanitaria del Principado de Asturias, Oviedo, Spain.

Patricia Fanlo (P)

Department of Internal Medicine, Hospital Universitario de Navarra, Pamplona, Spain.

Jose Ignacio Elejalde (JI)

Department of Internal Medicine, Hospital Universitario de Navarra, Pamplona, Spain.

Segundo Bujan (S)

Department of Internal Medicine, Hospital Universitari Vall d'Hebron, Barcelona, Spain.

Ignasi Figueras (I)

Department of Dermatology, Hospital de Bellvitge, Barcelona, Spain.

Francisco Manuel Marco (FM)

Department of Immunology, Hospital General Universitario de Alicante Dr Balmis, Alicante, Spain.
Alicante Institute for Health and Biomedical Research, Alicante, Spain.

Mariano Andrés (M)

Alicante Institute for Health and Biomedical Research, Alicante, Spain.
Seccion de Reumatologia, Hospital General Universitario de Alicante, Alicante, Spain.
Department of Clinic Medicine, Universidad Miguel Hernandez de Elche, Elche, Spain.

Silvia Suárez (S)

Department of Internal Medicine, Hospital Valle del Nalón, Langreo, Spain.

Andres Gonzalez-Garcia (A)

Systemic Autoimmune Diseases Unit, Department of Internal Medicine, Hospital Universitario Ramón y Cajal | IRYCIS, Madrid, Spain.

Xavier Fustà-Novell (X)

Department of Dermatology, Althaia Foundation of Manresa, Manresa, Spain.

Clara Garcia-Belando (C)

Department of Rheumatology, Hospital Universitario Virgen de la Arrixaca, Murcia, Spain.

Ana Granados (A)

Department of Internal Medicine, Hospital Universitari Parc Taulí, Sabadell, Spain.

Maria Teresa Fernandez-Figueras (MT)

Department of Pathology, Hospital Universitari General de Catalunya San Cugat del Valles, Sant Cugat del Valles, Spain.

Neus Quilis (N)

Department of Rheumatology, Hospital Universitari de Vinalopo, Elche, Spain.

Maria Orriols-Caba (M)

Department of Internal Medicine, Hospital comarcal Alt Penedès, Consorci Sanitari Alt Penedès-Garraf, Vilafranca del Penedès, Spain.

Ricardo Gómez de la Torre (R)

Department of Autoimmune Diseases, Hospital Universitario Central de Asturias, Oviedo, Spain.

Maria Cinta Cid (MC)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Autoimmune Diseases, Hospital Clinic de Barcelona, Barcelona, Spain.

Georgina Espígol-Frigolé (G)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Autoimmune Diseases, Hospital Clinic de Barcelona, Barcelona, Spain.

Alba Alvarez-Abella (A)

Department of Dermatology, Hospital Mútua de Terrassa, Terrassa, Spain.

Eztizen Labrador (E)

Department of Rheumatology, Hospital San Pedro, Logrono, Spain.

Maria Rozman (M)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Haematology, Hospital Clínic, Barcelona, Spain.

Monica Lopez-Guerra (M)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
Department of Pathology, Hospital Clínic, Barcelona, Spain.

Paola Castillo (P)

Department of Pathology, Hospital Clínic, Barcelona, Spain.

Jose R Alamo-Moreno (JR)

Department of Pathology, Hospital Clínic, Barcelona, Spain.

Eva Gonzalez-Roca (E)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
Department of Immunology, Hospital Clínic, Barcelona, Spain.

Susana Plaza (S)

Department of Immunology, Hospital Clínic, Barcelona, Spain.

Virginia Fabregat (V)

Department of Immunology, Hospital Clínic, Barcelona, Spain.

Rocio Lara (R)

Department of Immunology, Hospital Clínic, Barcelona, Spain.

Esther F Vicente-Rabaneda (EF)

Department of Rheumatology, Hospital Universitario La Princesa, Madrid, Spain.
Instituto de Investigaciones Sanitarias Princesa (IIS-Princesa), Madrid, Spain.
Department of Medicine, School of Medicine, Universidad Autónoma de Madrid, Madrid, Spain.

Sonia Tejedor-Vaquero (S)

Institut Hospital del Mar d'Investigacions Mèdiques, Barcelona, Spain.

Giuliana Magri (G)

Institut Hospital del Mar d'Investigacions Mèdiques, Barcelona, Spain.
Immunology Unit, Department of Biomedical Sciences, Faculty of Medicine and Health Science, University of Barcelona, Barcelona, Spain.

Nuria Bonet (N)

Genomics Core Facility, Departament de Medicina i Ciències de la Vida, Universitat Pompeu Fabra, Barcelona, Spain.

Manuel Solis-Moruno (M)

Genomics Core Facility, Departament de Medicina i Ciències de la Vida, Universitat Pompeu Fabra, Barcelona, Spain.

Andrea Cerutti (A)

Hospital del Mar Medical Research Institute, PRBB, Barcelona, Spain.
Catalan Institute for Research and Advanced Studies (ICREA), Barcelona, Spain.

Oscar Fornas (O)

Flow Cytometry Unit, Universitat Pompeu Fabra - Center of Genomic Regulation, Barcelona, Spain.

Ferran Casals (F)

Departament de Genètica, Microbiologia i Estadística, Faculty of Biology, University of Barcelona, Barcelona, Spain.
Institut de Biomedicina de la Universitat de Barcelona (IBUB), Universitat de Barcelona, Barcelona, Spain.

Jordi Yagüe (J)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Immunology, Hospital Clínic, Barcelona, Spain.

Juan I Aróstegui (JI)

Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain jiaroste@clinic.cat.
School of Medicine, Universitat de Barcelona, Barcelona, Spain.
Department of Immunology, Hospital Clínic, Barcelona, Spain.

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