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Azacitidine was the first drug shown to lengthen survival in higher-risk myelodysplastic syndromes, adding about nine months of median survival compared with the usual supportive care or chemotherapy.
Phase 3 trial of 358 patients with higher-risk MDS randomised to azacitidine or a pre-selected conventional care regimen (best supportive care, low-dose cytarabine or intensive chemotherapy).
Median overall survival was 24.5 months with azacitidine against 15.0 months with conventional care (hazard ratio 0.58), with two-year survival roughly doubled and fewer transfusions and infections.
Azacitidine (and decitabine) became the standard for higher-risk MDS in patients not going straight to transplant, and the backbone for combination trials that have so far failed to beat it.