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

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.