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Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study
Open accessAbstract
Background Drug treatments for patients with high-risk myelodysplastic syndromes provide no survival advantage. In this trial, we aimed to assess the effect of azacitidine on overall survival compared with the three commonest conventional care regimens.Methods In a phase III, international, multicentre, controlled, parallel-group, open-label trial, patients with higher-risk myelodysplastic syndromes were randomly assigned one-to-one to receive azacitidine (75 mg/m(2) per day for 7 days every 28 days) or conventional care (best supportive care, low-dose cytarabine, or intensive chemotherapy as selected by investigators before randomisation). Patients were stratified by French-American-British and international prognostic scoring system classifications; randomisation was done with a block size of four. The primary endpoint was overall survival. Efficacy analyses were by intention to treat for all patients assigned to receive treatment. This study is registered with ClinicalTrials.gov, number NCT00071799.Findings Between Feb 13, 2004, and Aug 7, 2006, 358 patients were randomly assigned to receive azacitidine (n=179) or conventional care regimens (n=179). Four patients in the azacitidine and 14 in the conventional care groups received no study drugs but were included in the intention-to-treat efficacy analysis. After a median follow-up of 21.1 months (IQR 15.1-26.9), median overall survival was 24.5 months (9.9-not reached) for the azacitidine group versus 15.0 months (5.6-24.1) for the conventional care group (hazard ratio 0.58; 95% CI 0.43-0.77; stratified log-rank p=0.0001). At last follow-up, 82 patients in the azacitidine group had died compared with 113 in the conventional care group. At 2 years, on the basis of Kaplan-Meier estimates, 50.8% (95% CI 42.1-58.8) of patients in the azacitidine group were alive compared with 26.2% (18.7-34.3) in the conventional care group (p<0.0001). Peripheral cytopenias were the most common grade 3-4 adverse events for all treatments.Interpretation Treatment with azacitidine increases overall survival in patients with higher-risk myelodysplastic syndromes relative to conventional care.
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“Hypomethylating agents (HMAs), such as azacitidine and decitabine, yield statistically similar complete remission (CR) rates of approximately 15%-20% in patients with either TP53-mutant or wild-type MDS.12-14”
From Eprenetapopt (APR-246) and Azacitidine in TP53 -Mutant Myelodysplastic Syndromes (2021) · cites this paper as [14]
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“Adverse events were similar to those reported for azacitidine or eprenetapopt monotherapy, with the most common grade ≥ 3 adverse events being febrile neutropenia (33%), leukopenia (29%), and neutropenia (29%).”
From Eprenetapopt (APR-246) and Azacitidine in TP53 -Mutant Myelodysplastic Syndromes (2021) · cites this paper as [14]
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“Whilst response rates of up to 60% to azacytidine have been reported in MDS 125,129 ,”
From The roles of DNA, RNA and histone methylation in ageing and cancer (2019) · cites this paper as [129]
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Kantarjian, H. M., Short, N. J., Fathi, A. T., Marcucci, G., Ravandi, F., Tallman, M. S., Wang, E. S., & Wei, A. H. (2021) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2021.05.016 · Cited by 56
Prediction of early (4‐week) mortality in acute myeloid leukemia with intensive chemotherapy
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Regulation of epigenetic homeostasis in uveal melanoma and retinoblastoma
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Role of Epigenetics in Chronic Myeloid Leukemia
Poláková, K. M., Koblihova, J., & Stopka, T. (2013) · Current Hematologic Malignancy Reports · doi:10.1007/s11899-012-0152-z · Cited by 52
“Additionally, they have shown promising results in combination with cytotoxic chemotherapy or alone in the treatment of several solid and hematologic neoplasias, including MDS [37, 38].”
BackgroundInfluentialIncidence of Myelodysplastic Syndrome in Japan
Chihara, D., Ito, H. M., Katanoda, K., Shibata, A., Matsuda, T., Sobue, T., & Matsuo, K. (2014) · Journal of Epidemiology · doi:10.2188/jea.je20140042 · Cited by 52
on behalf of the German MDS Study Group, Platzbecker, U., Braulke, F., Kündgen, A., Götze, K. S. S., Bug, G., Schönefeldt, C., Shirneshan, K., Röllig, C., Bornhäuser, M., et al. (2013) · Leukemia · doi:10.1038/leu.2013.26 · Cited by 53
Self-citationAzacytidine and erlotinib exert synergistic effects against acute myeloid leukemia
Lainey, É., Wolfromm, A., Marie, N., Enot, D. P., Scoazec, M., Bouteloup, C., Leroy, C., Micol, J.-B., de Botton, S., Galluzzi, L., et al. (2012) · Oncogene · doi:10.1038/onc.2012.469 · Cited by 51
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“), has recently been approved by the Food and Drug Administration (FDA) for the treatment of patients with Myelodysplastic Syndromes (MDS) and leukemia [13,14].”
Oral Clofarabine in the Treatment of Patients With Higher-Risk Myelodysplastic Syndrome
Faderl, S. H., Garcia‐Manero, G., Estrov, Z. E., Ravandi, F., Borthakur, G. M., Cortes, J. E., O'Brien, S. M., Gandhi, V. V., Plunkett, W. K., Byrd, A. L., et al. (2010) · Journal of Clinical Oncology · doi:10.1200/jco.2009.26.3509 · Cited by 51
“This is a single-arm, open-label, phase II clinical trial to evaluate the efficacy and safety of oral clofarabine in patients with higher-risk MDS.”
BackgroundInfluentialvan der Helm, L. H., Scheepers, E. R. M., Veeger, N. J. G. M., Daenen, S. M. G. J., Mulder, A. B., van den Berg, E., Vellenga, E., & Huls, G. A. (2013) · Journal of Hematology & Oncology · doi:10.1186/1756-8722-6-29 · Cited by 52
“A superior OS has been demonstrated in AML patients with 20–30% bone marrow blasts treated with azacitidine compared to conventional treatment [18,19].”
BackgroundEpigenetic regulators as promising therapeutic targets in acute myeloid leukemia
Gallipoli, P., Giotopoulos, G., & Huntly, B. J. P. (2015) · Therapeutic Advances in Hematology · doi:10.1177/2040620715577614 · Cited by 53
Cabrero, M., Jabbour, E. J., Ravandi, F., Bohannan, Z., Pierce, S. A., Kantarjian, H. M., & Garcia‐Manero, G. (2015) · Leukemia Research · doi:10.1016/j.leukres.2015.03.006 · Cited by 52
“This approach is based on the design of the AZA-001 trial [3] and clinical experience indicating that early interruption of these agents is associated with treatment failure.”
MethodNew insights into transfusion-related iron toxicity: Implications for the oncologist
Porter, J. B., de Witte, T., Cappellini, M. D., & Gattermann, N. (2015) · Critical Reviews in Oncology/Hematology · doi:10.1016/j.critrevonc.2015.11.017 · Cited by 55
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“[8,9] Effective clinical development of drugs targeting HDAC has been more challenging but”
BackgroundSelf-citationThe role of DNA-demethylating agents in cancer therapy
Mehdipour, P., Murphy, T., & Carvalho, D. D. D. (2019) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2019.107416 · Cited by 55
Schlafen 11 predicts response to platinum-based chemotherapy in gastric cancers
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Epigenetic Mechanisms in DNA Double Strand Break Repair: A Clinical Review
Fernandez, A., O’Leary, C. G., O'Byrne, K. J., Burgess, J. T., Richard, D. J., & Suraweera, A. (2021) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2021.685440 · Cited by 51
Warta, R., Herold‐Mende, C. C., Chaisaingmongkol, J., Popanda, O., Möck, A., Mogler, C., Osswald, F., Herpel, E., Küstner, S., Eckstein, V., et al. (2014) · International Journal of Cancer · doi:10.1002/ijc.28906 · Cited by 51
“This is of special interest because efficacy of demethylating agents is tested in several ongoing studies in cancer therapy.(31,32) However, due to the oncogenic nature of CSPG4 future clinical trials should consider that especially highly methylated tumors could become more aggressive in such a clinical setting.”
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“[3] Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al.”
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“Among patients who were RBC transfusion dependent at baseline, significantly more azacitidine than conventional care recipients achieved RBC transfusion independence (table IV).([46]) Rates of any, complete or partial remission and rates of any, major erythroid or major platelet improvement were significantly higher i…”
InfluentialXie, M., Jiang, Q., & Xie, Y. (2014) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2014.04.010 · Cited by 51
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“The overall response rate to azacytidine in the pivotal multicenter trial was 29%.(1) However, there is limited information about response rates to azacytidine in patients with previously treated MDS, in patients with acute myeloid leukemia (AML) that has relapsed after chemotherapy, and in patients with specific mole…”
BackgroundMyelodysplastic Syndromes and Acute Myeloid Leukemia in the Elderly
Klepin, H. D. (2015) · Clinics in Geriatric Medicine · doi:10.1016/j.cger.2015.08.010 · Cited by 53
“For example, the core binding factor leukemias (inv 16, t(8;21), t(16;16)), and acute promyelocytic leukemia (t(15;17)) are associated with better prognosis.”
BackgroundPleyer, L., Döhner, H., Dombret, H., Seymour, J. F., Schuh, A. C., Beach, C. L., Swern, A. S., Burgstaller, S., Stauder, R., Girschikofsky, M., et al. (2017) · International Journal of Molecular Sciences · doi:10.3390/ijms18020415 · Cited by 51
“This assumption was based on the significant improvement of overall survival (OS) obtained in the AZA-MDS-001 trial [7] and the Cancer and Leukemia Group B (CALGB) protocols, in which 32% [8] and 38% [9,10] of the trial population had AML with 20%–30% BM blasts, respectively.”
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Sébert, M., Renneville, A., Bally, C., Péterlin, P., Beyne‐Rauzy, O., Legros, L., Gourin, M.-P., Sanhès, L., Wattel, E., Gyan, E., et al. (2019) · Haematologica · doi:10.3324/haematol.2018.207118 · Cited by 51
“and the median overall survival (OS) of 18-24 months obtained with azacitidine remains modest.(1) Moreover, median survival after HMA failure is only approximately six months.”
BackgroundSelf-citationMyelodysplastic syndromes: 2011 update on diagnosis, risk‐stratification, and management
Garcia‐Manero, G. (2011) · American Journal of Hematology · doi:10.1002/ajh.22047 · Cited by 50
Moving toward individualized target-based therapies in acute myeloid leukemia
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“or these patients, a new strategy includes molecules with ifferentiating or proapoptotic activity [2–4].”
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“Compared with CCR, both AZA and DAC have shown delayed progression to AML (Fenaux et al., 2009; Kantarjian et al., 2006; Lubbert et al., 2011; Silverman et al., 2002).”
BackgroundMethodInfluentialTreatment of chronic myelomonocytic leukemia with 5-Azacitidine: A case series and literature review
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“Hypomethylating agents such as the DNA methyl transferase inhibitors 5-azacitidine (AZA) and 2-deoxy-5-azacytidine (decitabine) are active in patients with high-risk myelodysplastic syndrome (MDS), [6] but to a lesser degree in those with highly proliferative AML.”
BackgroundWhat's different about atypical CML and chronic neutrophilic leukemia?
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Metadata
- Authors
- Fenaux, P., Mufti, G. J., Hellstrom-Lindberg, E., Santini, V., Finelli, C., Giagounidis, A., Schoch, R., Gattermann, N., Sanz, G., List, A., Gore, S. D., Seymour, J. F., Bennett, J. M., Byrd, J., Backstrom, J., Zimmerman, L., McKenzie, D., Beach, C., & Silverman, L. R.
- Affiliations
- Université Sorbonne Paris Nord (FR)
- King's College Hospital (GB)
- Azienda Ospedaliero-Universitaria Careggi (IT)
- University Hospital Schleswig-Holstein (DE)
- Düsseldorf University Hospital (DE)
- Hospital Universitari i Politècnic La Fe (ES)
- Moffitt Cancer Center (US)
- Johns Hopkins University (US)
- Karolinska University Hospital (SE)
- University of Rochester Medicine (US)
- The Ohio State University (US)
- Celgene (United States) (US)
- Icahn School of Medicine at Mount Sinai (US)
- Corresponding author
- Fenaux, Pierre
- ORCID
- Venue
- The Lancet Oncology
- Publisher
- Elsevier BV
- Published
- 2009-03
- Volume
- 10
- Issue
- 3
- Pages
- 223-232
- ISSN
- 1470-2045 (ISSN-L)
- PMC
- PMC4086808
- OpenAlex
- W2118771330
- Cited by
- 2,663
- References
- 45
- Citations by year
- 2026: 80 · 2025: 99 · 2024: 103 · 2023: 153 · 2022: 149 · 2021: 182 · 2020: 148 · 2019: 149 · 2018: 132 · 2017: 164 · 2016: 178 · 2015: 173 · 2014: 201 · 2013: 182 · 2012: 178
- FWCI
- 124.66
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- The Lancet Oncology (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Florence Research (University of Florence) (submittedVersion)
- IRIS UNIMORE (University of Modena and Reggio Emilia) (submittedVersion, cc-by)
- Research Portal (King's College London) (submittedVersion)
- Funders
- Celgene
- National Cancer Institute (K24CA111717)
- Topics
- Health Sciences › Medicine › Hematology › Acute Myeloid Leukemia Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinoids in leukemia and cellular processes
- Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
- Keywords
- azacitidine, myelodysplastic syndrome, high-risk myelodysplastic syndrome, overall survival, low-dose cytarabine, intensive chemotherapy, best supportive care, hypomethylating agents, treatment efficacy comparison, hematologic malignancies
- MeSH terms
- Adult, Aged, Aged, 80 and over, Antimetabolites, Antineoplastic, Azacitidine, Female, Humans, Male, Middle Aged, Myelodysplastic Syndromes, Proportional Hazards Models
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