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EBMT 2025 | The role of allogeneic transplantation in VEXAS syndrome
In this video, Carmelo Gurnari, MD, University of Rome Tor Vergata, Rome, Italy, and Cleveland Clinic, Cleveland, OH, discusses the role of allogeneic stem cell transplantation (alloSCT) in VEXAS syndrome, stating that it is the only curative approach for the disease. Dr Gurnari highlights that despite the high morbidity burden of these patients, alloSCT has shown to been effective in eradicating the underlying mutation responsible for the VEXAS phenotype. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.
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Transcript
Allogeneic hematopoietic stem cell transplant is the only curative approach so far for VEXAS. And together with the EBMT and the Chronic Malignancy and Autoimmune Disease Working Party, we have published the largest cohort of patients so far reported for VEXAS. It was a cohort of 19 cases, mostly transplanted in a VEXAS agnostic fashion because of the recalcitrant inflammatory status, as well as the concomitant presence of myelodysplastic syndrome that was in need of a transplantation because of it’s non-responsiveness to the treatment that we could offer the patient for the myelodysplastic syndrome...
Allogeneic hematopoietic stem cell transplant is the only curative approach so far for VEXAS. And together with the EBMT and the Chronic Malignancy and Autoimmune Disease Working Party, we have published the largest cohort of patients so far reported for VEXAS. It was a cohort of 19 cases, mostly transplanted in a VEXAS agnostic fashion because of the recalcitrant inflammatory status, as well as the concomitant presence of myelodysplastic syndrome that was in need of a transplantation because of it’s non-responsiveness to the treatment that we could offer the patient for the myelodysplastic syndrome. The results have been good, considering the very high morbidity burden that this patient carry. The median line of treatment was five before transplant, which is like high, considering like a myelodysplastic syndrome patient. And obviously these also include the rheumatologic treatment that was needed to control the hyper-inflammatory status. And at two years of follow-up, 75% that was the overall survival with the transplant-related mortality of 25% mostly due to infectious disease, which are in fact a complication that are like somehow typical of patient that have been highly immunosuppressed.
One thing that is important if I can add is that after transplant molecular studies have shown that the culprit mutation leading to VEXAS was completely eradicated after the procedure with a complete reversion of the VEXAS phenotype.
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