article · EJC Paediatric Oncology
NRAS alone 4.7% at diagnosis) with UBA1 mutation arising second (NRAS+UBA1: 27.3%).The NRAS+UBA1-mutant clone demonstrated myeloid bias compared to NRAS-only and wildtype cells.Purified CD34+ cells showed enrichment of both UBA1 and NRAS mutations, suggesting preferential expansion within hematopoietic stem/progenitor cells.Conclusions: We describe the first pediatric case of VEXAS MDS showing excellent response to CPX-351 followed by transplantation.We also show that the UBA1 mutation can arise secondary to Ras pathway activation.
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DOI: 10.1016/j.ejcped.2025.100405
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