TY - JOUR
T1 - Efficacy and safety profile of biotechnological agents and Janus kinase inhibitors in VEXAS syndrome
T2 - data from the international AIDA Network VEXAS registry
AU - Vitale, Antonio
AU - Caggiano, Valeria
AU - Leone, Flavia
AU - Hinojosa-Azaola, Andrea
AU - Martín-Nares, Eduardo
AU - Guaracha-Basañez, Guillermo Arturo
AU - Torres-Ruiz, Jiram
AU - Kawakami-Campos, Perla Ayumi
AU - Hissaria, Pravin
AU - Callisto, Alicia
AU - Beecher, Mark
AU - Dagna, Lorenzo
AU - Campochiaro, Corrado
AU - Tomelleri, Alessandro
AU - Frassi, Micol
AU - Franceschini, Franco
AU - Crisafulli, Francesca
AU - Hernández-Rodríguez, José
AU - Gómez-Caverzaschi, Verónica
AU - Araújo, Olga
AU - Sfriso, Paolo
AU - Bindoli, Sara
AU - Baggio, Chiara
AU - Sota, Jurgen
AU - Tufan, Abdurrahman
AU - Kucuk, Hamit
AU - Piga, Matteo
AU - Cauli, Alberto
AU - D’Agostino, Maria Antonietta
AU - De Paulis, Amato
AU - Mormile, Ilaria
AU - Giardini, Henrique A.Mayrink
AU - Cordeiro, Rafael Alves
AU - Lopalco, Giuseppe
AU - Iannone, Florenzo
AU - Monti, Sara
AU - Montecucco, Carlomaurizio
AU - Ruiz-Irastorza, Guillermo
AU - Soto-Peleteiro, Adriana
AU - Triggianese, Paola
AU - Gurnari, Carmelo
AU - Viapiana, Ombretta
AU - Bixio, Riccardo
AU - Vitetta, Rosetta
AU - Rovera, Guido
AU - Conticini, Edoardo
AU - La Torre, Francesco
AU - Portincasa, Piero
AU - Jaber, Nour
AU - Ragab, Gaafar
AU - Maher, Amina
AU - Batu, Ezgi Deniz
AU - Ozen, Seza
AU - Wiesik-Szewczyk, Ewa
AU - de-la-Torre, Alejandra
AU - Balistreri, Alberto
AU - Frediani, Bruno
AU - Fabiani, Claudia
AU - Cantarini, Luca
N1 - Publisher Copyright:
Copyright © 2025 Vitale, Caggiano, Leone, Hinojosa-Azaola, Martín-Nares, Guaracha-Basañez, Torres-Ruiz, Kawakami-Campos, Hissaria, Callisto, Beecher, Dagna, Campochiaro, Tomelleri, Frassi, Franceschini, Crisafulli, Hernández-Rodríguez, Gómez-Caverzaschi, Araújo, Sfriso, Bindoli, Baggio, Sota, Tufan, Kucuk, Piga, Cauli, D’Agostino, De Paulis, Mormile, Giardini, Cordeiro, Lopalco, Iannone, Monti, Montecucco, Ruiz-Irastorza, Soto-Peleteiro, Triggianese, Gurnari, Viapiana, Bixio, Vitetta, Rovera, Conticini, La Torre, Portincasa, Jaber, Ragab, Maher, Batu, Ozen, Wiesik-Szewczyk, de-la-Torre, Balistreri, Frediani, Fabiani and Cantarini.
PY - 2025
Y1 - 2025
N2 - 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.
AB - 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.
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UR - http://www.scopus.com/inward/citedby.url?scp=86000473669&partnerID=8YFLogxK
U2 - 10.3389/fphar.2025.1462254
DO - 10.3389/fphar.2025.1462254
M3 - Research Article
AN - SCOPUS:86000473669
SN - 1663-9812
VL - 16
JO - Frontiers in Pharmacology
JF - Frontiers in Pharmacology
M1 - 1462254
ER -