Allogeneic haematopoietic stem cell transplantation for VEXAS syndrome: UK experience

Adam Al-Hakim¹, James A Poulter², Dina Mahmoud¹, Ailsa M S Rose¹, Suzanne Elcombe³, Helen Lachmann⁴, Catherine Cargo⁵, Christopher J A Duncan^{6

7}, Mark Bishton^{8

9}, Venetia Bigley^{6

7}, Anjum Khan¹⁰, Sinisa Savic^{2

11

12}

Affiliations

¹ Department of Clinical Immunology and Allergy, St James's University Hospital, Leeds, UK.
² Leeds Institute of Medical Research, University of Leeds, Leeds, UK.
³ Department of Immunology, Newcastle upon Tyne NHS Trust, Newcastle upon Tyne, UK.
⁴ National Amyloidosis Centre Royal Free London NHS Foundation Trust and Division of Medicine University, College London, London, UK.
⁵ Haematological Malignancy Diagnostic Service, Leeds Cancer Centre, St James's University Hospital, Leeds, UK.
⁶ Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
⁷ Northern Centre for Bone Marrow Transplantation, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
⁸ Department of haematology, Nottingham University Hospitals NHS Trust, UK.
⁹ Translational Medical Sciences, University of Nottingham, UK.
¹⁰ Department of Haematology, St James's University Hospital, Leeds, UK.
¹¹ Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, UK.
¹² NIHR Leeds Biomedical Research Centre, Leeds Teaching Hospitals NHS Trust, Chapel Allerton Hospital, Leeds, UK.

Free article

Adam Al-Hakim et al. Br J Haematol. 2022 Dec.

Free article

. 2022 Dec;199(5):777-781.

doi: 10.1111/bjh.18488. Epub 2022 Oct 2.

¹ Department of Clinical Immunology and Allergy, St James's University Hospital, Leeds, UK.
² Leeds Institute of Medical Research, University of Leeds, Leeds, UK.
³ Department of Immunology, Newcastle upon Tyne NHS Trust, Newcastle upon Tyne, UK.
⁴ National Amyloidosis Centre Royal Free London NHS Foundation Trust and Division of Medicine University, College London, London, UK.
⁵ Haematological Malignancy Diagnostic Service, Leeds Cancer Centre, St James's University Hospital, Leeds, UK.
⁶ Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
⁷ Northern Centre for Bone Marrow Transplantation, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
⁸ Department of haematology, Nottingham University Hospitals NHS Trust, UK.
⁹ Translational Medical Sciences, University of Nottingham, UK.
¹⁰ Department of Haematology, St James's University Hospital, Leeds, UK.
¹¹ Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, UK.
¹² NIHR Leeds Biomedical Research Centre, Leeds Teaching Hospitals NHS Trust, Chapel Allerton Hospital, Leeds, UK.

No abstract available

Keywords: VEXAS syndrome; allogeneic haematopoietic stem cell transplantation; chronic myelomonocytic leukaemia; myelodysplastic syndrome.

References

1. Beck DB, Ferrada MA, Sikora KA, Ombrello AK, Collins JC, Pei W, et al. Somatic mutations in UBA1 and severe adult-onset autoinflammatory disease. N Engl J Med. 2020;383(27):2628-38.
1. Ferrada MA, Savic S, Ospina Cardona D, Collins JC, Alessi H, Gutierrez-Rodrigues F, et al. Translation of cytoplasmic UBA1 contributes to VEXAS syndrome pathogenesis. Blood. 2022. Online ahead of print.
1. Loschi M, Roux C, Sudaka I, Ferrero-Vacher C, Marceau-Renaut A, Duployez N, et al. Allogeneic stem cell transplantation as a curative therapeutic approach for VEXAS syndrome: a case report. Bone Marrow Transplant. 2022;57(2):315-8.
1. Diarra A, Duployez N, Fournier E, Preudhomme C, Coiteux V, Magro L, et al. Successful allogeneic hematopoietic stem cell transplantation in patients with VEXAS syndrome: a 2-center experience. Blood Adv. 2022;6(3):998-1003.
1. Georgin-Lavialle S, Terrier B, Guedon AF, Heiblig M, Comont T, Lazaro E, et al. 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. 2022;186(3):564-74.