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. 2025 Sep 22.
doi: 10.1002/art.43384. Online ahead of print.

Comparative Efficacy and Safety of Anakinra and Canakinumab in Patients With VEXAS Syndrome: An International Multicenter Study

Tali Eviatar  1   2 , Dafne Capelusnik  1   2   3 , Corrado Campochiaro  4 , Valentin Lacombe  5   6 , Vincent Jachiet  7 , Michael Zisapel  1   2 , Iftach Sagy  8   9 , Oshrat E Tayer-Shifman  2   10 , David Ozeri  11 , Shaye Kivity  2   10 , Alessandro Tomelleri  4 , Benjamin Terrier  12 , Hagit Peleg  13   14 , Thibault Comont  15 , Karim Sacre  16 , Pascal Woaye-Hune  17 , Laurent Arnaud  18 , Estibaliz Lazaro  19 , Vincent Grobost  20 , Francois Lifermann  21 , Maxime Samson  22   23 , Samuel Ardois  24 , Alice Garnier  25 , Alexandre Maria  26 , Alain Cantagrel  27 , Aurore Meyer  28 , Jean-David Bouaziz  29 , Mael Heiblig  30 , Lorenzo Dagna  4 , Elisa Diral  4 , Olivier Kosmider  31 , Ori Elkayam  1   2 , Jérôme Hadjadj  7 , Sophie Georgin-Lavialle  32 , Olivier Fain  7 , Arsene Mekinian  7
Affiliations

Comparative Efficacy and Safety of Anakinra and Canakinumab in Patients With VEXAS Syndrome: An International Multicenter Study

Tali Eviatar et al. Arthritis Rheumatol. .

Abstract

Objective: The aim of this study was to compare differences in clinical response, drug survival, and adverse event rates between anakinra and canakinumab in VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome.

Methods: This multicenter international study includes patients with VEXAS from France, Israel, and Italy treated with interleukin-1 inhibition. Global response (GR) was defined as the absence of inflammatory symptoms and ≥50% decrease in steroid dose and C-reactive protein. Multiple regression analysis was performed to identify associated variables. Drug survival was analyzed using Kaplan-Meier plots and log-rank test, with Cox regression models for associated factors.

Results: We included 47 male patients with VEXAS; 44 received anakinra and 9 received canakinumab, with 6 patients using both at different time points. GR at 1 month was 34% for anakinra and 100% for canakinumab (P < 0.001) and 22% and 78% at 3 months, respectively (P = 0.001). Treatment with canakinumab was associated with a higher odds ratio (OR) of achieving GR at 3 months (OR 28.8, 95% confidence interval 3.0-273.9; P = 0.004) in a multivariable analysis. Median drug survival was 54 (interquartile range [IQR] 30-56) months for canakinumab at 300 mg/month compared with 7 (IQR 4-8) months for canakinumab 150 mg/month and 1 (IQR 1-2.5) months for anakinra (P = 0.01). Injection-site reactions were only recorded for the anakinra group (47 vs 0%; P = 0.006), whereas infections were more frequent in the anakinra group (31% and 11%; P = 0.3).

Conclusion: Canakinumab demonstrated superior clinical response and drug survival with fewer adverse events compared with anakinra. Monthly canakinumab 300 mg may be considered as an effective steroid-sparing therapeutic option for patients with VEXAS.

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References

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