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Randomized trial demonstrates overall survival benefits of azacitidine in higher-risk MDS, suggesting optimism in patient counseling.
Azacitidine is the only drug proven to prolong overall survival (OS) in patients with higher-risk myelodysplastic syndromes (HR-MDS) in the large randomized trial AZA-001 with a median OS of 24.5 months among azacitidinetreated patients vs. 15.0 months in the conventional care regimens (CCR) arm, with a hazard ratio of 0.58; p = 0.0001 1 . Two-year OS probability was 51% in azacitidine arm compared with 26% in the CCR arm (p < 0.0001). These numbers are often quoted to counsel HR-MDS patients regarding expected benefits with azacitidine therapy.
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Zeidan et al. (2018) studied this question.
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