Seymour, John FDöhner, HartmutButrym, AleksandraWierzbowska, AgnieszkaSelleslag, DominikJang, Jun HoKumar, RajatCavenagh, JamesSchuh, Andre CCandoni, AnnaRecher, ChristianSandhu, IrwindeepBernal-del-Castillo, TeresaAl-Ali, Haifa KathrinFalantes, JoseStone, Richard MMinden, Mark DWeaver, JerrySonger, SteveBeach, C LDombret, Herve2023-01-252023-01-252017-12-14Seymour JF, Döhner H, Butrym A, Wierzbowska A, Selleslag D, Jang JH, et al. Azacitidine improves clinical outcomes in older patients with acute myeloid leukaemia with myelodysplasia-related changes compared with conventional care regimens. BMC Cancer. 2017 Dec 14;17(1):852.http://hdl.handle.net/10668/11907Compared with World Health Organization-defined acute myeloid leukaemia (AML) not otherwise specified, patients with AML with myelodysplasia-related changes (AML-MRC) are generally older and more likely to have poor-risk cytogenetics, leading to poor response and prognosis. More than one-half of all older (≥65 years) patients in the phase 3 AZA-AML-001 trial had newly diagnosed AML-MRC. We compared clinical outcomes for patients with AML-MRC treated with azacitidine or conventional care regimens (CCR; induction chemotherapy, low-dose cytarabine, or supportive care only) overall and within patient subgroups defined by cytogenetic risk (intermediate or poor) and age (65-74 years or ≥75 years). The same analyses were used to compare azacitidine with low-dose cytarabine in patients who had been preselected to low-dose cytarabine before they were randomized to receive azacitidine or CCR (ie, low-dose cytarabine). Median overall survival was significantly prolonged with azacitidine (n = 129) versus CCR (n = 133): 8.9 versus 4.9 months (hazard ratio 0.74, [95%CI 0.57, 0.97]). Among patients with intermediate-risk cytogenetics, median overall survival with azacitidine was 16.4 months, and with CCR was 8.9 months (hazard ratio 0.73 [95%CI 0.48, 1.10]). Median overall survival was significantly improved for patients ages 65-74 years treated with azacitidine compared with those who received CCR (14.2 versus 7.3 months, respectively; hazard ratio 0.64 [95%CI 0.42, 0.97]). Within the subgroup of patients preselected to low-dose cytarabine before randomization, median overall survival with azacitidine was 9.5 months versus 4.6 months with low-dose cytarabine (hazard ratio 0.77 [95%CI 0.55, 1.09]). Within the low-dose cytarabine preselection group, patients with intermediate-risk cytogenetics who received azacitidine had a median overall survival of 14.1 months versus 6.4 months with low-dose cytarabine, and patients aged 65-74 years had median survival of 14.9 months versus 5.2 months, respectively. Overall response rates were similar with azacitidine and CCR (24.8% and 17.3%, respectively), but higher with azacitidine versus low-dose cytarabine (27.2% and 13.9%). Adverse events were generally comparable between the treatment arms. Azacitidine may be the preferred treatment for patients with AML-MRC who are not candidates for intensive chemotherapy, particularly patients ages 65-74 years and those with intermediate-risk cytogenetics.enAttribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/AMLAML-MRCAcute myeloid leukaemiaAzacitidineInduction chemotherapyLow-dose cytarabineMyelodysplasia-related changesResponseSurvivalAge FactorsAgedAged, 80 and overAntimetabolites, AntineoplasticAzacitidineCytarabineFemaleHumansKaplan-Meier EstimateLeukemia, Myeloid, AcuteMaleMyelodysplastic SyndromesPrognosisTreatment Outcomeresearch article29241450open accessPacientesAzacitidinaCitarabinaDosificaciónSobrevidaAncianoPronósticoQuimioterapiaQuimioterapia de inducción10.1186/s12885-017-3803-61471-2407PMC5731212https://doi.org/10.1186/s12885-017-3803-6https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5731212/pdf