Allogeneic haematopoietic stem cell transplantation for VEXAS syndrome: UK experience.

Previous studies have reported patients with VEXAS being transplanted in the absence of a prior diagnosis, including one of the patients we report here (P4). Keywords: allogeneic haematopoietic stem cell transplantation; chronic myelomonocytic leukaemia; myelodysplastic syndrome; VEXAS syndrome EN allogeneic haematopoietic stem cell transplantation chronic myelomonocytic leukaemia myelodysplastic syndrome VEXAS syndrome 777 781 5 11/24/22 20221201 NES 221201 Vacuoles, E1-ligase, X-linked, Auto-inflammatory, Somatic (VEXAS) syndrome, is an acquired, progressive systemic auto-inflammatory disorder with overlapping rheumatological and haematological features.1,2 It is caused by myeloid-restricted somatic mutations in ubiquitin-like modifier-activating enzyme 1 ( I UBA1 i ), the gene that encodes E1 ubiquitin ligase. Very few patients had any inflammatory symptoms or disease manifestations classically associated with VEXAS (Table 2).2,5 The MDS features were also different. [Extracted from the article]