Work

Myeloid neoplasm inspired intensive therapy in VEXAS syndrome: A single‐centre experience

Year: 2025

Type: article

Abstract: There is still no standard of care and unmet medical needs in refractory/advanced VEXAS (vacuoles in myeloid progenitors, E1 ubiquitin activating enzyme, X-linked, autoinflammatory manifestations and somatic) syndrome with or without associated haematological neoplasm. We report the clinical outcome of four multirefractory/advanced VEXAS patients treated with acute myeloid leukaemia-like therapeutic approaches. All patients responded to inflammatory/haematological VEXAS-related features, which were associated with measurable residual disease response (partial or complete). Prospective studies evaluating new and effective therapeutic strategies in order to reduce clonal burden in VEXAS patients are warranted.

Source: British Journal of Haematology

DOI: 10.1111/bjh.20067

Authors: Maël Heiblig, Adriana Plesa, Juliet Tantot, Yvan Jamilloux, Hélène Labussière‐Wallet, Pierre Sujobert