Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Jun 3;9(6):e70156.
doi: 10.1002/hem3.70156. eCollection 2025 Jun.

Efficacy of erythroid-stimulating agent and luspatercept in VEXAS syndrome: A multicenter retrospective study by the FRENVEX group

Maël Heiblig  1 , Vincent Jachiet  2 , Jérôme Hadjadj  2 , Lin Pierre Zhao  3 , Thibault Comont  4 , Hervé Lobbes  5 , Valentin Lacombe  6 , Anne Blandine Boutin  7 , Joris Galland  8 , Benjamin Terrier  9 , Sophie Georgin-Lavialle  10 , Pierre Fenaux  3 , Arsène Mekinian  2 ; FRENVEX Group
Affiliations

Efficacy of erythroid-stimulating agent and luspatercept in VEXAS syndrome: A multicenter retrospective study by the FRENVEX group

Maël Heiblig et al. Hemasphere. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

The authors do not have any conflicts of interest to declare for this study.

Figures

Figure 1
Figure 1
Erythroid hematological improvement: (A) at 16 week and (B) 24 weeks. (C) Serum endogenous erythropoietin (EPO) levels at erythropoietin‐stimulating agent (ESA) according to HI‐E response at week 16. (D) Cumulative incidence of ESA failure in responding patients. (E) Baseline predictive factors of HI‐E response at week 16 on ESA. (F) Overall survival according to baseline red blood cell transfusion burden before ESA initiation.
See this image and copyright information in PMC

References

    1. Beck DB, Ferrada MA, Sikora KA, et al. Somatic mutations in UBA1 and severe adult‐onset autoinflammatory disease. N Engl J Med. 2020;383:NEJMoa2026834. - PMC - PubMed
    1. Bourbon E, Heiblig M, Gerfaud‐Valentin M, et al. Therapeutic options in Vexas syndrome: insights from a retrospective series. Blood. 2021;137:3682‐3684. - PubMed
    1. Obiorah IE, Patel BA, Groarke EM, et al. Benign and malignant hematologic manifestations in patients with VEXAS syndrome due to somatic mutations in UBA1. Blood Adv. 2021;5(16):3203‐3215. - PMC - PubMed
    1. Gutierrez‐Rodrigues F, Kusne Y, Fernandez J, et al. Spectrum of clonal hematopoiesis in VEXAS syndrome. Blood. 2023; 142(3):244‐259. - PMC - PubMed
    1. Heiblig M, Ferrada MA, Koster MT, et al. Ruxolitinib is more effective than other JAK inhibitors to treat VEXAS syndrome: a retrospective multicenter study. Blood. 2022;140(8):927‐931. - PMC - PubMed

LinkOut - more resources