DOAJ Open Access 2025

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

Antonio Vitale Antonio Vitale Valeria Caggiano Valeria Caggiano Flavia Leone +79 lainnya

Abstrak

BackgroundVEXAS 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.MethodsClinical, laboratory, and therapeutic data from VEXAS patients were obtained from the international AIDA Network VEXAS registry.ResultsIn 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.ConclusionIL-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.

Topik & Kata Kunci

Therapeutics. Pharmacology

Penulis (84)

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

Mark Beecher

Lorenzo Dagna

Corrado Campochiaro

Alessandro Tomelleri

Micol Frassi

Franco Franceschini

Francesca Crisafulli

José Hernández-Rodríguez

Verónica Gómez-Caverzaschi

Olga Araújo

Paolo Sfriso

Sara Bindoli

Chiara Baggio

Jurgen Sota

Abdurrahman Tufan

Hamit Kucuk

Matteo Piga

Alberto Cauli

Maria Antonietta D’Agostino

Amato De Paulis

Ilaria Mormile

Henrique A. Mayrink Giardini

Rafael Alves Cordeiro

Giuseppe Lopalco

Florenzo Iannone

Sara Monti

Carlomaurizio Montecucco

Guillermo Ruiz-Irastorza

Adriana Soto-Peleteiro

Paola Triggianese

Carmelo Gurnari

Ombretta Viapiana

Riccardo Bixio

Rosetta Vitetta

Guido Rovera

Edoardo Conticini

Francesco La Torre

Piero Portincasa

Nour Jaber

Gaafar Ragab

Amina Maher

Ezgi Deniz Batu

Seza Ozen

Ewa Wiesik-Szewczyk

Alejandra de-la-Torre

Alberto Balistreri

Bruno Frediani

Claudia Fabiani

Luca Cantarini

Format Sitasi

APA MLA BibTeX

Vitale, A., Vitale, A., Caggiano, V., Caggiano, V., Leone, F., Hinojosa-Azaola, A. et al. (2025). Efficacy and safety profile of biotechnological agents and Janus kinase inhibitors in VEXAS syndrome: data from the international AIDA Network VEXAS registry. https://doi.org/10.3389/fphar.2025.1462254

@article{vitale2025, author = {Antonio Vitale and Antonio Vitale and Valeria Caggiano and Valeria Caggiano and Flavia Leone and Andrea Hinojosa-Azaola and Eduardo Martín-Nares and Guillermo Arturo Guaracha-Basañez and Jiram Torres-Ruiz and Perla Ayumi Kawakami-Campos and Pravin Hissaria and Pravin Hissaria and Alicia Callisto and Alicia Callisto and Mark Beecher and Mark Beecher and Lorenzo Dagna and Lorenzo Dagna and Corrado Campochiaro and Corrado Campochiaro and Alessandro Tomelleri and Alessandro Tomelleri and Micol Frassi and Franco Franceschini and Francesca Crisafulli and José Hernández-Rodríguez and Verónica Gómez-Caverzaschi and Olga Araújo and Paolo Sfriso and Sara Bindoli and Chiara Baggio and Jurgen Sota and Jurgen Sota and Abdurrahman Tufan and Hamit Kucuk and Matteo Piga and Matteo Piga and Alberto Cauli and Alberto Cauli and Maria Antonietta D’Agostino and Amato De Paulis and Amato De Paulis and Ilaria Mormile and Henrique A. Mayrink Giardini and Rafael Alves Cordeiro and Giuseppe Lopalco and Florenzo Iannone and Sara Monti and Sara Monti and Carlomaurizio Montecucco and Carlomaurizio Montecucco and Guillermo Ruiz-Irastorza and Guillermo Ruiz-Irastorza and Adriana Soto-Peleteiro and Adriana Soto-Peleteiro and Paola Triggianese and Paola Triggianese and Carmelo Gurnari and Carmelo Gurnari and Ombretta Viapiana and Riccardo Bixio and Rosetta Vitetta and Guido Rovera and Edoardo Conticini and Edoardo Conticini and Francesco La Torre and Piero Portincasa and Nour Jaber and Gaafar Ragab and Gaafar Ragab and Amina Maher and Amina Maher and Ezgi Deniz Batu and Seza Ozen and Ewa Wiesik-Szewczyk and Alejandra de-la-Torre and Alberto Balistreri and Alberto Balistreri and Bruno Frediani and Bruno Frediani and Claudia Fabiani and Claudia Fabiani and Luca Cantarini and Luca Cantarini}, title = {Efficacy and safety profile of biotechnological agents and Janus kinase inhibitors in VEXAS syndrome: data from the international AIDA Network VEXAS registry}, year = {2025}, journal = {DOAJ}, doi = {10.3389/fphar.2025.1462254}, url = {https://www.frontiersin.org/articles/10.3389/fphar.2025.1462254/full}, }

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Informasi Jurnal

Tahun Terbit: 2025
Sumber Database: DOAJ
DOI: 10.3389/fphar.2025.1462254
Akses: Open Access ✓