De-escalation of Corticosteroids and Clonal Remission in Mutation-driven VEXAS Syndrome with 5-azacytidine

Overview

Journal Haematologica

Specialty Hematology

Date 2024 Jun 13

PMID 38867587

Authors

Roochi Trikha

Kar Lok Kong

James Galloway

Tanya N Basu

Lynn Quek

Jamie Wilson

Louise Gamble

Henna Wong

Steven Best

Austin Kulasekararaj

Affiliations

Soon will be listed here.

Citing Articles

VEXAS, Chediak-Higashi syndrome and Danon disease: myeloid cell endo-lysosomal pathway dysfunction as a common denominator?.

Savy C, Bourgoin M, Cluzeau T, Jacquel A, Robert G, Auberger P Cell Mol Biol Lett. 2025; 30(1):12.

PMID: 39865233 PMC: 11765923. DOI: 10.1186/s11658-025-00691-0.

VEXAS Syndrome: A Comprehensive Review of Current Therapeutic Strategies and Emerging Treatments.

Alqatari S, Alqunais A, Alali S, Alharbi M, Hasan M, Al Shubbar M J Clin Med. 2024; 13(22).

PMID: 39598114 PMC: 11594742. DOI: 10.3390/jcm13226970.

Understanding Myelodysplasia and Inflammation Through the Lense of VEXAS Syndrome: A Review.

Wolff L, Caratsch L, Zhao L, Blum S, Comte D Cells. 2024; 13(22).

PMID: 39594638 PMC: 11593025. DOI: 10.3390/cells13221890.

[Treatment of VEXAS syndrome].

Krusche M Z Rheumatol. 2024; 83(9):766-769.

PMID: 39340559 PMC: 11527926. DOI: 10.1007/s00393-024-01577-w.

References

Kulasekararaj A, Kordasti S, Basu T, Salisbury J, Mufti G, du Vivier A . Chronic relapsing remitting Sweet syndrome--a harbinger of myelodysplastic syndrome. Br J Haematol. 2015; 170(5):649-56. DOI: 10.1111/bjh.13485. View

Al-Hakim A, Savic S . An update on VEXAS syndrome. Expert Rev Clin Immunol. 2022; 19(2):203-215. DOI: 10.1080/1744666X.2023.2157262. View

Mekinian A, Zhao L, Chevret S, Desseaux K, Pascal L, Comont T . A Phase II prospective trial of azacitidine in steroid-dependent or refractory systemic autoimmune/inflammatory disorders and VEXAS syndrome associated with MDS and CMML. Leukemia. 2022; 36(11):2739-2742. DOI: 10.1038/s41375-022-01698-8. View

Bourbon E, Heiblig M, Valentin M, Barba T, Durel C, Lega J . Therapeutic options in VEXAS syndrome: insights from a retrospective series. Blood. 2021; 137(26):3682-3684. DOI: 10.1182/blood.2020010177. View

Raaijmakers M, Hermans M, Aalbers A, Rijken M, Dalm V, van Daele P . Azacytidine Treatment for VEXAS Syndrome. Hemasphere. 2021; 5(12):e661. PMC: 8598219. DOI: 10.1097/HS9.0000000000000661. View

Heiblig M, Ferrada M, Koster M, Barba T, Gerfaud-Valentin M, Mekinian A . Ruxolitinib is more effective than other JAK inhibitors to treat VEXAS syndrome: a retrospective multicenter study. Blood. 2022; 140(8):927-931. PMC: 9412002. DOI: 10.1182/blood.2022016642. View

Sockel K, Gotze K, Ganster C, Bill M, Georgi J, Balaian E . VEXAS syndrome: complete molecular remission after hypomethylating therapy. Ann Hematol. 2024; 103(3):993-997. PMC: 10866742. DOI: 10.1007/s00277-023-05611-w. View

Comont T, Heiblig M, Riviere E, Terriou L, Rossignol J, Bouscary D . Azacitidine for patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) and myelodysplastic syndrome: data from the French VEXAS registry. Br J Haematol. 2021; 196(4):969-974. DOI: 10.1111/bjh.17893. View

Beck D, Ferrada M, Sikora K, Ombrello A, Collins J, Pei W . Somatic Mutations in and Severe Adult-Onset Autoinflammatory Disease. N Engl J Med. 2020; 383(27):2628-2638. PMC: 7847551. DOI: 10.1056/NEJMoa2026834. View

10.

Georgin-Lavialle S, Terrier B, Guedon A, Heiblig M, Comont T, Lazaro E . Further characterization of clinical and laboratory features in VEXAS syndrome: large-scale analysis of a multicentre case series of 116 French patients. Br J Dermatol. 2021; 186(3):564-574. DOI: 10.1111/bjd.20805. View

11.

Beck D, Werner A, Kastner D, Aksentijevich I . Disorders of ubiquitylation: unchained inflammation. Nat Rev Rheumatol. 2022; 18(8):435-447. PMC: 9075716. DOI: 10.1038/s41584-022-00778-4. View

12.

Gurnari C, Koster L, Baaij L, Heiblig M, Yakoub-Agha I, Collin M . Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT. Blood Adv. 2024; 8(6):1444-1448. PMC: 10955646. DOI: 10.1182/bloodadvances.2023012478. View

13.

Gurnari C, McLornan D . Update on VEXAS and role of allogeneic bone marrow transplant: Considerations on behalf of the Chronic Malignancies Working Party of the EBMT. Bone Marrow Transplant. 2022; 57(11):1642-1648. DOI: 10.1038/s41409-022-01774-8. View