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EHA 2025 | The burden of disease in patients with VEXAS syndrome and the need for further research

Fabio Efficace, PhD, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy, comments on the health-related quality of life profile of patients with VEXAS syndrome. He highlights the substantial burden of disease and emphasizes the need for further research to address the unmet needs of these patients. This interview took place at the 30th Congress of the European Hematology Association (EHA) in Milan, Italy.

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Transcript

Yeah, this is a relatively novel disease. So for the first time we wanted to explore the prevalence of clinically important problems and symptoms of these patients. So we have done this in a preliminary analysis of some patients with this disease. We will present additional and final data at the next meeting at ASH. And actually seeing that the burden of the disease is pretty substantial in terms of functional limitation, in terms of symptoms...

Yeah, this is a relatively novel disease. So for the first time we wanted to explore the prevalence of clinically important problems and symptoms of these patients. So we have done this in a preliminary analysis of some patients with this disease. We will present additional and final data at the next meeting at ASH. And actually seeing that the burden of the disease is pretty substantial in terms of functional limitation, in terms of symptoms. So we really need to do a lot of research in this population because we know very little and we expect that this will be the first evidence-based information that might help clinicians to better address unmet needs of these patients.

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