This multicenter retrospective cohort study compared allogeneic hematopoietic stem cell transplantation (alloHSCT, n=31) versus hypomethylating agents (HMA, n=35) in 66 patients with VEXAS syndrome, with a median follow-up of 18 months from therapy initiation.
AlloHSCT achieved molecular remission in 100% of evaluable patients and glucocorticoid discontinuation in 58%, versus 22% molecular remission and 6% glucocorticoid discontinuation with HMA. On multivariable analysis, alloHSCT was associated with significantly improved overall survival (HR=0.20, 95% CI: 0.05–0.81; p=0.024), with 3 deaths in the alloHSCT group vs. 11 in the HMA group.
- Retrospective design limits causal inference. - HMA group was older on average, which may bias survival comparisons despite multivariable adjustment. - Small sample size (n=66) restricts statistical power and generalizability.
In eligible patients with VEXAS syndrome, alloHSCT appears to offer superior molecular remission rates and a meaningful survival advantage over HMA — consider it early as a potentially curative strategy. HMA carries a high real-world discontinuation rate due to toxicity or lack of response and should be viewed as a bridge or option for transplant-ineligible patients.
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