AZA-001: azacitidine versus conventional care in higher-risk myelodysplastic syndromes
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.
Overview
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.
- Median overall survival 24.5 vs 15.0 months; hazard ratio 0.58.
- Two-year survival 50.8 percent vs 26.2 percent.
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.
- Median time to response is several cycles, so treatment must continue for at least four to six cycles before judging failure.
- Responses are not durable; outcomes after hypomethylating failure are poor.
Similar pages
not linked directly; found by shared links- TreatmentPevonedistat
- TrialVERONA
- TrialA Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia
- TermHypomethylating agents (azacitidine, decitabine)
- TermIPSS-R and IPSS-M (myelodysplastic syndrome risk scores)
- TreatmentDecitabine