{"entity":{"id":"paper-aza-001-fenaux-lancet-oncol-2009","kind":"paper","name":"AZA-001: azacitidine versus conventional care in higher-risk myelodysplastic syndromes","aka":[],"tldr":"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).\n\nMedian 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.","asOf":"2026-09-17","links":[{"label":"Lancet Oncol 2009","url":"https://doi.org/10.1016/S1470-2045(09)70003-8"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/19230772/"}],"tags":[],"related":[],"cancers":["mds-higher-risk"],"sections":[],"technologies":[],"targets":[],"drugs":["azacitidine"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["aza-001"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["lancet-oncology"],"dependsOn":[],"notes":[],"journal":"The Lancet Oncology","year":2009,"doi":"10.1016/S1470-2045(09)70003-8","pmid":"19230772","authors":"Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al.","paperType":"rct","findings":["Median overall survival 24.5 vs 15.0 months; hazard ratio 0.58.","Two-year survival 50.8 percent vs 26.2 percent."],"whatItMeans":"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."],"changedPractice":true,"participants":358},"route":"/key-papers/paper-aza-001-fenaux-lancet-oncol-2009/","neighbours":{"cancer":[{"id":"mds-higher-risk","kind":"cancer","name":"Higher-risk myelodysplastic syndromes","route":"/cancers/mds-higher-risk/"}],"drug":[{"id":"azacitidine","kind":"drug","name":"Azacitidine","route":"/drugs/azacitidine/"}],"trial":[{"id":"aza-001","kind":"trial","name":"AZA-001","route":"/trials/aza-001/"}],"journal":[{"id":"lancet-oncology","kind":"journal","name":"The Lancet Oncology","route":"/journals/lancet-oncology/"}]}}