# AZA-001: azacitidine versus conventional care in higher-risk myelodysplastic syndromes

Source: https://onco.cc/key-papers/paper-aza-001-fenaux-lancet-oncol-2009/  
OnCo record `paper-aza-001-fenaux-lancet-oncol-2009` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Summary

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.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: The Lancet Oncology
- Year: 2009
- DOI: 10.1016/S1470-2045(09)70003-8
- Authors: Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al.
- Findings: Median overall survival 24.5 vs 15.0 months; hazard ratio 0.58.; Two-year survival 50.8 percent vs 26.2 percent.
- What it means: 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.
- Caveats: 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.

## Sources

- Lancet Oncol 2009: https://doi.org/10.1016/S1470-2045(09)70003-8
- PubMed: https://pubmed.ncbi.nlm.nih.gov/19230772/

## Connected records

- cancers: [Higher-risk myelodysplastic syndromes](https://onco.cc/cancers/mds-higher-risk/)
- drugs: [Azacitidine](https://onco.cc/drugs/azacitidine/)
- trials: [AZA-001](https://onco.cc/trials/aza-001/)
- journals: [The Lancet Oncology](https://onco.cc/journals/lancet-oncology/)

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