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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]
Experimental — accuracy not yet measuredIn the citing paper
“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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Self-citationSvensson, T., Chowdhury, O., Garelius, H. G., Lorenz, F., Saft, L., Jacobsen, S. E. W., Hellström‐Lindberg, E., & Cherif, H. (2014) · European Journal Of Haematology · doi:10.1111/ejh.12383 · Cited by 34
“This in contrast to reports from the pivotal studies with Aza, where progression of grade I–II thrombocytopenia to severe thrombocytopenia was evident in the majority of the thrombocytopenic patients (6, 21).”
BackgroundResultvan der Helm, L. H., Veeger, N. J. G. M., Kooy, M. V. M., Beeker, A., de Weerdt, O., de Groot, M., Alhan, C., Hoogendoorn, M., Laterveer, L., van de Loosdrecht, A. A., et al. (2013) · Leukemia Research · doi:10.1016/j.leukres.2013.03.022 · Cited by 34
Current therapeutic approaches for patients with myelodysplastic syndromes
Greenberg, P. L. (2010) · British Journal of Haematology · doi:10.1111/j.1365-2141.2010.08226.x · Cited by 33
Risks for infection in patients with myelodysplasia and acute leukemia
Pagano, L., & Caira, M. (2012) · Current Opinion in Infectious Diseases · doi:10.1097/qco.0b013e328358b000 · Cited by 34
“New drugs, such as demethylating agents (azacytidine or decitabine) and lenalidomide, have been recently introduced for the treatment of high-risk MDS, but they do not seem to modify the infectious risk in this subset of patients [12,13].”
BackgroundSmith, B. D., Beach, C. L., Mahmoud, D., Weber, L., & Henk, H. J. (2014) · Experimental Hematology and Oncology · doi:10.1186/2162-3619-3-10 · Cited by 34
Self-citationDrug Repurposing for Epigenetic Targets Guided by Computational Methods
Naveja, J. J., Dueñas‐González, A., & Medina‐Franco, J. L. (2016) · Elsevier eBooks · doi:10.1016/b978-0-12-802808-7.00012-5 · Cited by 37
Jabbour, E. J., Faderl, S. H., Sasaki, K., Kadia, T. M., Daver, N. G., Pemmaraju, N., Patel, K. P., Khoury, J. D., Bueso-Ramos, C., Bohannan, Z. S., et al. (2016) · Cancer · doi:10.1002/cncr.30383 · Cited by 34
“Partial response 1 (1) Hematologic improvement 2 (3)”
BackgroundUnnikrishnan, A., Vo, A. N. Q., Pickford, R., Raftery, M. J., Nunez, A. C., Verma, A., Hesson, L. B., & Pimanda, J. E. (2017) · Leukemia · doi:10.1038/leu.2017.340 · Cited by 34
Voso, M. T., Niscola, P., Piciocchi, A., Fianchi, L., Maurillo, L., Musto, P., Pagano, L., Mansueto, G., Criscuolo, M., Spiriti, M. A. A., et al. (2015) · European Journal Of Haematology · doi:10.1111/ejh.12595 · Cited by 34
“The study included 196 consecutive patients, affected by MDS (n = 184 patients), and low blast count AML (n = 12 patients), retrospectively collected by the Italian cooperative groups ‘Gruppo Romano MDS (GROM)’ and Basilicata MDS Registry.”
BackgroundInfluentialBethge, N., Lothe, R. A., Honne, H., Andresen, K., Trøen, G., Eknæs, M., Liestøl, K., Holte, H., Delabie, J., Smeland, E. B., et al. (2013) · Epigenetics · doi:10.4161/epi.27554 · Cited by 34
5-Azacytidine in myelodysplastic syndromes: A clinical practice guideline
Buckstein, R. J., Yee, K. W. L., & Wells, R. A. (2010) · Cancer Treatment Reviews · doi:10.1016/j.ctrv.2010.05.006 · Cited by 34
Maesako, Y., Izumi, K., Okamori, S., Takeoka, K., Kishimori, C., Okumura, A., Honjo, G., Akasaka, T., & Ohno, H. (2013) · International Journal of Hematology · doi:10.1007/s12185-013-1482-x · Cited by 34
“The use of azacitidine for this case was based upon a phase III study, in which the drug was proved to be active for high-risk MDS and CMML defined by the FrenchAmerican-British classification [14, 15].”
MethodSinghal, D., Kutyna, M., Hahn, C. N., Shah, M. V., & Hiwase, D. (2024) · Blood Cancer Discovery · doi:10.1158/2643-3230.bcd-24-0103 · Cited by 39
“Furthermore, published experience is largely based on retrospective analysis of relatively small numbers of t-MN cases (127, 144), and the median number of completed HMA cycles ranges from three to six cycles (127, 144), lower than that reported for p-MDS (143, 145).”
BackgroundAlhan, C., Westers, T. M., van der Helm, L. H., Eeltink, C., Huls, G. A., Lissenberg-Witte, B. I., Buchi, F., Santini, V., Ossenkoppele, G. J., & van de Loosdrecht, A. A. (2014) · Cytometry Part B Clinical Cytometry · doi:10.1002/cyto.b.21160 · Cited by 33
Self-citationLaille, E. J., Savona, M. R., Scott, B. L., Boyd, T. E., Dong, Q., & Skikne, B. (2013) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.251 · Cited by 33
“Stable disease in patients with higher‐risk MDS receiving SC azacitidine has been associated with prolonged overall survival in the absence of complete remission.(25,27) It remains to be Table 3.”
BackgroundResultZeidan, A. M., Lee, J., Prébet, T., Greenberg, P. L., Sun, Z., Juckett, M., Smith, M. R., Paietta, E. M., Gabrilove, J. L., Erba, H. P., et al. (2014) · British Journal of Haematology · doi:10.1111/bjh.12884 · Cited by 33
Self-citationGetta, B. M., Kishtagari, A., Hilden, P. D., Tallman, M. S., Maloy, M., Gonzales, P. K., Castro-Malaspina, H. R., Perales, M., Giralt, S., Tamari, R., et al. (2017) · Transplantation and Cellular Therapy · doi:10.1016/j.bbmt.2017.03.020 · Cited by 35
“Results Cohort 362 patients with a median (range) age at diagnosis of 65 years (20-75) and median followup of 17 (0-94) months in those alive at last review were included (table 1).”
BackgroundAlmeida, A. M., Prébet, T., Itzykson, R., Ramos, F., Al‐Ali, H. K., Shammo, J. M., Pinto, R., Maurillo, L., Wetzel, J. L., Musto, P., et al. (2017) · International Journal of Molecular Sciences · doi:10.3390/ijms18040837 · Cited by 33
“This reclassifies almost all cases of AEL into myelodysplasia (MDS) or AML subtypes [7,8].”
BackgroundInfluentialSelf-citationBoutault, R., Péterlin, P., Boubaya, M., Sockel, K., Chevallier, P., Garnier, A., Guillaume, T., Bourgeois, A. L., Debord, C., Godon, C., et al. (2018) · British Journal of Haematology · doi:10.1111/bjh.15626 · Cited by 33
“…prompt initiation of appropriate medical care can benefit some of these patients, as new treatments have been emerging in the last few years which may improve the management of MDS, both in terms of survival and quality of life (Fenaux et al, 2009, 2011; Platzbecker et al, 2017; List et al, 2018).”
BackgroundTranscriptional networks in acute myeloid leukemia
Thoms, J. A. I., Beck, D., & Pimanda, J. E. (2019) · Genes Chromosomes and Cancer · doi:10.1002/gcc.22794 · Cited by 33
Recent Advances in the Targeting of Epigenetic Regulators in B-Cell Non-Hodgkin Lymphoma
Ribeiro, M. L., Reyes-Garau, D., Armengol, M., Fernández-Serrano, M., & Roué, G. (2019) · Frontiers in Genetics · doi:10.3389/fgene.2019.00986 · Cited by 34
Mahmood, N., Arakelian, A., Cheishvili, D., Moshe Szyf, A., & Rabbani, S. A. (2020) · Journal of Cellular and Molecular Medicine · doi:10.1111/jcmm.15642 · Cited by 32
Bewersdorf, J. P., & Zeidan, A. M. (2021) · Cancers · doi:10.3390/cancers13071610 · Cited by 36
“AZA has been the only agent shown to have a statistically significant OS benefit in randomized clinical trials in MDS based on data from the AZA-001 trial [81].”
BackgroundResultInfluentialInterpreting new molecular genetics in myelodysplastic syndromes
Abdel-Wahab, O. I., & Figueroa, M. E. (2012) · Hematology · doi:10.1182/asheducation.v2012.1.56.3798194 · Cited by 33
Epigenetic therapy in myelodysplastic syndromes
Musolino, C., Sant’Antonio, E., Penna, G., Alonci, A., Russo, S., Granata, A., & Allegra, A. (2010) · European Journal Of Haematology · doi:10.1111/j.1600-0609.2010.01433.x · Cited by 33
Small molecule drugs with immunomodulatory effects in cancer
Murphy, A. G., & Zheng, L. (2015) · Human Vaccines & Immunotherapeutics · doi:10.1080/21645515.2015.1057363 · Cited by 33
“The DNA methyltransferase (DNMT) inhibitors azacitidine and decitabine are FDAapproved in high risk myelodysplastic syndrome.(7) Azacitidine has been shown to have immunomodulatory effects in colon cancer by converting CD11b myeloid cells into antigen presenting cells (APCs).”
BackgroundPan, F., Peng, S., Fleurence, R., Linnehan, J. E., Knopf, K., & Kim, E. J. (2010) · Clinical Therapeutics · doi:10.1016/j.clinthera.2010.12.003 · Cited by 33
Chau, D., Ng, K. K. M., Chan, T. S. Y., Cheng, Y. Y., Fong, B. M.-W., Tam, S., Kwong, Y., & Tse, E. (2015) · Journal of Hematology & Oncology · doi:10.1186/s13045-015-0143-3 · Cited by 32
“Demethylating agents, including azacitidine and decitabine, are a standard medication for the management of myelodysplastic syndrome and elderly subjects with AML [16-19].”
BackgroundBonadies, N., Feller, A., Rovó, A., Ruefer, A., Blum, S., Gerber, B., Stüssi, G., Benz, R. A., Cantoni, N., Holbro, A., et al. (2017) · Cancer Epidemiology · doi:10.1016/j.canep.2016.12.005 · Cited by 33
“I Am Older, Not Elderly,” Said the Patient With Acute Myeloid Leukemia
Schiffer, C. A. (2009) · Journal of Clinical Oncology · doi:10.1200/jco.2009.25.8616 · Cited by 33
Mitotic perturbation is a key mechanism of action of decitabine in myeloid tumor treatment
Yabushita, T., Chinen, T., Nishiyama, A., Asada, S., Shimura, R., Isobe, T., Yamamoto, K., Sato, N., Enomoto, Y., Tanaka, Y., et al. (2023) · Cell Reports · doi:10.1016/j.celrep.2023.113098 · Cited by 32
Narayan, R., Piérola, A. A., Donnellan, W. B., Molero, A., Abdul‐Hay, M., Platzbecker, U., Subklewe, M. S., Kadia, T. M., Alonso‐Domínguez, J. M., McCloskey, J. K., et al. (2024) · Clinical and Translational Science · doi:10.1111/cts.13742 · Cited by 31
“While 40%–50% of patients with higher risk MDS respond to hypometh-ylating agents (HMAs), 6,7 responses are generally transient ( 2 years), and prognoses post-HMA failure are grim.”
BackgroundRole of reactive oxygen species in myelodysplastic syndromes
Jing, Q., Zhou, C., Zhang, J., Zhang, P., Wu, Y., Zhou, J., Tong, X., Li, Y., Du, J., & Wang, Y. (2024) · Cellular & Molecular Biology Letters · doi:10.1186/s11658-024-00570-0 · Cited by 33
“The registration trial (AZA-001) found that patients who received azaciti-dine showed a notable improvement in survival time compared with those who received standard of care, including intensive chemotherapy (24.5 months compared with 15 months) [277].”
ResultInfluentialMenin inhibitors in the treatment of acute myeloid leukemia
Huls, G. A., Woolthuis, C. M., & Schuringa, J. J. (2024) · Blood · doi:10.1182/blood.2024026232 · Cited by 34
Itzykson, R., Thépot, S., Quesnel, B., Dreyfus, F., Récher, C., Wattel, E., Gardin, C., Adès, L., & Fenaux, P. (2012) · Blood · doi:10.1182/blood-2012-04-422204 · Cited by 32
Self-citationWang, H., Li, Y., Lv, N., Li, Y., Wang, L., & Yu, L. (2018) · Annals of Hematology · doi:10.1007/s00277-018-3464-9 · Cited by 32
Diagnosis and treatment of chronic myelomonocytic leukemia
Kwon, J. (2021) · Blood Research · doi:10.5045/br.2021.2020321 · Cited by 32
“The clinical trials that led to the approval of these drugs were conducted on a large number of MDS patients, and the overall response rate (ORR) reached 60% [50, 51].”
BackgroundSOHO State of the Art & Next Questions: Myelodysplastic Syndromes: A New Decade
Volpe, V. O., Garcia‐Manero, G., & Komrokji, R. S. (2021) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2021.07.031 · Cited by 33
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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