ESH MDS 2024 | A retrospective study assessing efficacy and safety of ESAs in VEXAS syndrome with or without MDS

Maël Heiblig, MD, Hôpital Lyon Sud, Lyon, France, discusses the findings of a retrospective study that aimed to assess the clinical efficacy and safety of two erythropoiesis-stimulating agents (ESAs), erythropoietin (EPO) and luspatercept, in patients with VEXAS syndrome with or without myelodysplastic syndromes (MDS). Patients with a lower transfusion burden were more likely to respond to EPO, and EPO level at baseline was the only factor predictive of response. However, Dr Heiblig highlights that achieving a response did not translate to an increase in overall survival (OS). Luspatercept also showed encouraging efficacy and safety, although the cohort size was limited, and a larger prospective study is necessary for robust conclusions to be drawn. This interview took place at the European School of Hematology 9th Translational Research Conference on Myelodysplastic Syndromes (ESH MDS) in Budapest, Hungary.

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