Allogeneic Hematopoietic Cell Transplantation for VEXAS Syndrome: Results of a Multicenter Study of the EBMT

Robin M, Porcher R, Zinke-Cerwenka W, van Biezen A, Volin L, Mufti G . Allogeneic haematopoietic stem cell transplant in patients with lower risk myelodysplastic syndrome: a retrospective analysis on behalf of the Chronic Malignancy Working Party of the EBMT. Bone Marrow Transplant. 2016; 52(2):209-215. DOI: 10.1038/bmt.2016.266. View

Bruno A, Gurnari C, Alexander T, Snowden J, Greco R . Autoimmune manifestations in VEXAS: Opportunities for integration and pitfalls to interpretation. J Allergy Clin Immunol. 2023; 151(5):1204-1214. DOI: 10.1016/j.jaci.2023.02.017. View

Gurnari C, Pagliuca S, Durkin L, Terkawi L, Awada H, Kongkiatkamon S . Vacuolization of hematopoietic precursors: an enigma with multiple etiologies. Blood. 2021; 137(26):3685-3689. PMC: 8462399. DOI: 10.1182/blood.2021010811. View

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

van Leeuwen-Kerkhoff N, de Witte M, Heijstek M, Leavis H . Case report: Up-front allogeneic stem cell transplantation in a patient with the VEXAS syndrome. Br J Haematol. 2022; 199(3):e12-e15. DOI: 10.1111/bjh.18424. View

Diarra A, Duployez N, Fournier E, Preudhomme C, Coiteux V, Magro L . Successful allogeneic hematopoietic stem cell transplantation in patients with VEXAS syndrome: a 2-center experience. Blood Adv. 2021; 6(3):998-1003. PMC: 8945317. DOI: 10.1182/bloodadvances.2021004749. View

Mangaonkar A, Langer K, Lasho T, Finke C, Litzow M, Hogan W . Reduced intensity conditioning allogeneic hematopoietic stem cell transplantation in VEXAS syndrome: Data from a prospective series of patients. Am J Hematol. 2022; 98(2):E28-E31. DOI: 10.1002/ajh.26786. View

Gutierrez-Rodrigues F, Kusne Y, Fernandez J, Lasho T, Shalhoub R, Ma X . Spectrum of clonal hematopoiesis in VEXAS syndrome. Blood. 2023; 142(3):244-259. PMC: 10375269. DOI: 10.1182/blood.2022018774. View

Koster M, Lasho T, Olteanu H, Reichard K, Mangaonkar A, Warrington K . VEXAS syndrome: Clinical, hematologic features and a practical approach to diagnosis and management. Am J Hematol. 2023; 99(2):284-299. DOI: 10.1002/ajh.27156. View

10.

Alexander T, Greco R . Hematopoietic stem cell transplantation and cellular therapies for autoimmune diseases: overview and future considerations from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2022; 57(7):1055-1062. PMC: 9109750. DOI: 10.1038/s41409-022-01702-w. View

11.

Al-Hakim A, Poulter J, Mahmoud D, Rose A, Elcombe S, Lachmann H . Allogeneic haematopoietic stem cell transplantation for VEXAS syndrome: UK experience. Br J Haematol. 2022; 199(5):777-781. DOI: 10.1111/bjh.18488. View

12.

Greco R, Labopin M, Badoglio M, Veys P, Furtado Silva J, Abinun M . Allogeneic HSCT for Autoimmune Diseases: A Retrospective Study From the EBMT ADWP, IEWP, and PDWP Working Parties. Front Immunol. 2019; 10:1570. PMC: 6622152. DOI: 10.3389/fimmu.2019.01570. View

13.

Loschi M, Roux C, Sudaka I, Ferrero-Vacher C, Marceau-Renaut A, Duployez N . Allogeneic stem cell transplantation as a curative therapeutic approach for VEXAS syndrome: a case report. Bone Marrow Transplant. 2022; 57(2):315-318. DOI: 10.1038/s41409-021-01544-y. View

14.

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

15.

Gurnari C, Mannion P, Pandit I, Pagliuca S, Voso M, Maciejewski J . Screening in Sweet Syndrome With Hematological Neoplasms Reveals a Novel Association Between VEXAS and Chronic Myelomonocytic Leukemia. Hemasphere. 2022; 6(10):e775. PMC: 9519141. DOI: 10.1097/HS9.0000000000000775. View

16.

Bacigalupo A, Ballen K, Rizzo D, Giralt S, Lazarus H, Ho V . Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009; 15(12):1628-33. PMC: 2861656. DOI: 10.1016/j.bbmt.2009.07.004. View

17.

Arber D, Orazi A, Hasserjian R, Thiele J, Borowitz M, Le Beau M . The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016; 127(20):2391-405. DOI: 10.1182/blood-2016-03-643544. View

18.

Gurnari C, Visconte V . From bone marrow failure syndromes to VEXAS: Disentangling clonal hematopoiesis, immune system, and molecular drivers. Leuk Res. 2023; 127:107038. DOI: 10.1016/j.leukres.2023.107038. View

19.

Gurnari C, Pascale M, Vitale A, Diral E, Tomelleri A, Galossi E . Diagnostic capabilities, clinical features, and longitudinal UBA1 clonal dynamics of a nationwide VEXAS cohort. Am J Hematol. 2023; 99(2):254-262. DOI: 10.1002/ajh.27169. View

20.

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