The DOI matches its Crossref record; 45 of the 45 references it cites were found in a registry or an index.
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.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“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]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“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]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Nonparametric Estimation from Incomplete Observations
Kaplan, E. L., & Meier, P. (1992) · Springer series in statistics · doi:10.1007/978-1-4612-4380-9_25 · Cited by 45,236
Regression Models and Life-Tables
Cox, D. R. (1972) · Journal of the Royal Statistical Society Series B: Statistical Methodology · doi:10.1111/j.2517-6161.1972.tb00899.x · Cited by 39,793
Nonparametric Estimation from Incomplete Observations
Kaplan, E. L., & Meier, P. (1958) · Journal of the American Statistical Association · doi:10.1080/01621459.1958.10501452 · Cited by 39,158
Regression Models and Life-Tables
Cox, D. R. (1992) · Springer Series in Statistics · doi:10.1007/978-1-4612-4380-9_37 · Cited by 26,297
Evaluation of survival data and two new rank order statistics arising in its consideration.
Mantel, N. (1966) · PubMed · Cited by 7,516
International scoring system for evaluating prognosis in myelodysplastic syndromes
Greenberg, P., Cox, C., LeBeau, M. M., Fenaux, P., Morel, P., Sanz, G., Sanz, M., Vallespi, T., Hamblin, T., Oscier, D., et al. (1997) · Blood · doi:10.1182/blood.v89.6.2079 · Cited by 4,427
Self-citationProposals for the classification of the myelodysplastic syndromes
Bennett, J. M., Catovsky, D., Daniel, M., Flandrin, G., Galton, D. A. G., Gralnick, H. R., & Sultan, C. (1982) · British Journal of Haematology · doi:10.1111/j.1365-2141.1982.tb08475.x · Cited by 3,619
Self-citationHarris, N. L., Jaffe, E. S., Diebold, J., Flandrin, G., Müller‐Hermelink, H. K., Vardiman, J. W., Lister, T. A., & Bloomfield, C. D. (1999) · Journal of Clinical Oncology · doi:10.1200/jco.1999.17.12.3835 · Cited by 3,261
Cheson, B. D., Bennett, J. M., Kopecky, K. J., Büchner, T., Willman, C. L., Estey, E. H., Schiffer, C. A., Döhner, H., Tallman, M. S., Lister, T. A., et al. (2003) · Journal of Clinical Oncology · doi:10.1200/jco.2003.04.036 · Cited by 2,686
Self-citationDiscrete sequential boundaries for clinical trials
GORDON LAN, K. K., & DEMETS, D. L. (1983) · Biometrika · doi:10.1093/biomet/70.3.659 · Cited by 2,310
Cited by
Sorted by citations
Items 901–1,000 of 1,002
Myelodysplastic syndromes: What do hospitalists need to know?
Zeidan, A. M., Faltas, B. M., Smith, B. D., & Gore, S. D. (2013) · Journal of Hospital Medicine · doi:10.1002/jhm.2049 · Cited by 22
Self-citationHistorical perspectives on myelodysplastic syndromes
Steensma, D. P. (2012) · Leukemia Research · doi:10.1016/j.leukres.2012.08.007 · Cited by 25
Gattermann, N., Kündgen, A., Kellermann, L., Zeffel, M., Paessens, B., & Germing, U. (2013) · European Journal Of Haematology · doi:10.1111/ejh.12196 · Cited by 22
Self-citationÖzbalak, M., Cetiner, M., Beköz, H. S., Ateşoğlu, E. B., Ar, M. C., Salihoğlu, A., Tüzüner, N., & Ferhanoğlu, B. (2011) · Hematological Oncology · doi:10.1002/hon.986 · Cited by 22
“In AZA001, a randomized phase III study on 358 high risk MDS patients, Fenaux et al. concluded that median overall survival in patients receiving Aza-C was longer than those on supportive care (24.5 vs. 15.0 months).”
BackgroundInfluentialThe role of histone methyltransferase EZH2 in myelodysplastic syndromes
Xu, F., & Li, X. (2012) · Expert Review of Hematology · doi:10.1586/ehm.12.5 · Cited by 22
Wu, L., Li, X., Su, J., He, Q., Zhang, X., Chang, C., & Pu, Q. (2011) · Journal of Cancer Research and Clinical Oncology · doi:10.1007/s00432-011-1020-2 · Cited by 22
Promises and challenges of anticancer drugs that target the epigenome
Verbrugge, I., Johnstone, R. W., & Bots, M. L. (2011) · Epigenomics · doi:10.2217/epi.11.82 · Cited by 22
Buchi, F., Masala, E., Rossi, A., Valencia, A., Spinelli, E., Sanna, A., Gozzini, A., & Santini, V. (2013) · Epigenetics · doi:10.4161/epi.27322 · Cited by 22
Self-citationThomas, X., Arthur, C. K., Delaunay, J., Jones, M., Berrak, E., & Kantarjian, H. M. (2013) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2013.09.007 · Cited by 22
Epigenetic Dysregulation in Follicular Lymphoma
Araf, S., Okosun, J., Koniali, L., Fitzgibbon, J., & Heward, J. A. (2015) · Epigenomics · doi:10.2217/epi.15.96 · Cited by 23
Unraveling Myelodysplastic Syndromes: Current Knowledge and Future Directions
Chamseddine, A. N., Jabbour, E. J., Kantarjian, H. M., Bohannan, Z. S., & Garcia‐Manero, G. (2015) · Current Oncology Reports · doi:10.1007/s11912-015-0489-2 · Cited by 22
Kondo, T., Ozawa, S., Ikoma, T., Xy, Y., Kanamori, K., Suzuki, K., Iwabuchi, H., Maehata, Y., Miyamoto, C., Taguchi, T., et al. (2016) · Oncogenesis · doi:10.1038/oncsis.2016.43 · Cited by 22
Prébet, T., Cluzeau, T., Park, S., Sekeres, M. A., Germing, U., Adès, L., Platzbecker, U., Götze, K. S. S., Vey, N., Olíva, E. N., et al. (2017) · Oncotarget · doi:10.18632/oncotarget.18477 · Cited by 21
Self-citationKrejčík, Z., Beličková, M., Hruštincová, A., Votavová, H., Jonasova, A. T., Čermák, J., Dyr, J. E., & Merkerová, M. D. (2018) · Cancer Biomarkers · doi:10.3233/cbm-171029 · Cited by 22
Blood and Marrow Transplant Clinical Trials Network State of the Science Symposium 2014
Appelbaum, F. R., Anasetti, C., Antin, J. H., Atkins, H. L., Davies, S. M., Devine, S. M., Giralt, S., Heslop, H. E., Laport, G. G., Lee, S. J., et al. (2014) · Transplantation and Cellular Therapy · doi:10.1016/j.bbmt.2014.10.003 · Cited by 22
Can we predict responsiveness to hypomethylating agents in AML?
Stomper, J., & Lübbert, M. (2019) · Seminars in Hematology · doi:10.1053/j.seminhematol.2019.02.001 · Cited by 22
Zeidan, A. M., Hu, X. Y., Zhu, W., Stahl, M. F., Wang, R., Huntington, S. F., Giri, S., Bewersdorf, J. P., Podoltsev, N. A., Gore, S. D., et al. (2019) · Leukemia & lymphoma/Leukemia and lymphoma · doi:10.1080/10428194.2019.1663423 · Cited by 22
“0% Months followed from first HMA treatment (median, IQR) 10 [4, 19] Months to First HMA (median, IQR) 3 [1, 13] Dead by the end of study No 251 12.”
BackgroundSelf-citationDecitabine treatment of patients with higher-risk myelodysplastic syndromes
Steensma, D. P. (2009) · Leukemia Research · doi:10.1016/s0145-2126(09)70228-0 · Cited by 22
DNA methylation at enhancer regions Novel avenues for epigenetic biomarker development
Helgason, C. D. (2015) · Frontiers in bioscience · doi:10.2741/4399 · Cited by 24
Vidal, V., Robert, G., Goursaud, L., Durand, L., Ginet, C., Karsenti, J. M., Luciano, F., Gastaud, L., Garnier, G., Braun, T., et al. (2017) · Oncotarget · doi:10.18632/oncotarget.17482 · Cited by 22
“Azacitidine (AZA) is the reference treatment for most higher-risk myelodysplastic syndromes (MDS) patients [1] which also improves overall survival (OS) in elderly acute myeloid leukemia (AML) patients ineligible for intensive chemotherapy (IC) [2].”
Self-citationA clinical guide to TP53 mutations in myeloid neoplasms
Urrutia, S., Wong, T. N., & Link, D. C. (2025) · Blood · doi:10.1182/blood.2025029691 · Cited by 22
Management of Lower-Risk Myelodysplastic Syndromes:The Art and Evidence
Komrokji, R. S., Sekeres, M. A., & List, A. F. (2011) · Current Hematologic Malignancy Reports · doi:10.1007/s11899-011-0086-x · Cited by 21
Self-citationNazha, A., Sekeres, M. A., Gore, S. D., & Zeidan, A. M. (2015) · The Oncologist · doi:10.1634/theoncologist.2015-0067 · Cited by 22
Self-citationCandelaria, M., Burgos, S., Ponce, M., Espinoza, R., & Dueñas‐González, A. (2017) · Annals of Hematology · doi:10.1007/s00277-017-3103-x · Cited by 24
Healthcare expenses for treatment of acute myeloid leukemia
Bewersdorf, J. P., Shallis, R. M., Wang, R., Huntington, S. F., Perreault, S., Ma, X., & Zeidan, A. M. (2019) · Expert Review of Hematology · doi:10.1080/17474086.2019.1627869 · Cited by 21
Richardson, D. R., Green, S. D., Foster, M. C., & Zeidner, J. F. (2021) · Current Hematologic Malignancy Reports · doi:10.1007/s11899-021-00608-6 · Cited by 21
Allogeneic stem cell transplantation for patients with myelodysplastic syndromes
Vittayawacharin, P., Kongtim, P., & Ciurea, S. O. (2022) · American Journal of Hematology · doi:10.1002/ajh.26763 · Cited by 23
The genetics of myelodysplastic syndromes and the opportunities for tailored treatments
Kontandreopoulou, C., Kalopisis, K., Viniou, N., & Diamantopoulos, P. T. (2022) · Frontiers in Oncology · doi:10.3389/fonc.2022.989483 · Cited by 23
Treatment of Acute Myeloid Leukemia in Older Adults
Alsouqi, A., Geramita, E. M., & Im, A. (2023) · Cancers · doi:10.3390/cancers15225409 · Cited by 23
Lim, A. M. L., Candiloro, I. L. M., Wong, N. C., Collins, M. L., Do, H., Takano, E. A., Angel, C. M., Young, R. J., Corry, J. F., Wiesenfeld, D., et al. (2014) · Clinical Epigenetics · doi:10.1186/1868-7083-6-22 · Cited by 21
Wang, X., CHEN, E., Yang, X., WANG, Y., Quan, Z., Wu, X., & Luo, C. (2015) · Oncology Reports · doi:10.3892/or.2015.4492 · Cited by 21
Zeidan, A. M., Wang, R., Davidoff, A. J., Ma, S., Zhao, Y., Gore, S. D., Gross, C. P., & Ma, X. (2016) · Cancer · doi:10.1002/cncr.29945 · Cited by 21
Self-citationEpigenetic therapy of myelodysplastic syndromes and acute myeloid leukemia
Voso, M. T., Lo‐Coco, F., & Fianchi, L. (2015) · Current Opinion in Oncology · doi:10.1097/cco.0000000000000231 · Cited by 21
Cogle, C. R., Kurtin, S. E., Bentley, T. G. K., Broder, M. S., Chang, E. Y., Megaffin, S., Fruchtman, S. M., Petrone, M. E., & Mukherjee, S. (2017) · The Oncologist · doi:10.1634/theoncologist.2016-0211 · Cited by 21
Mądry, K., Lis, K., Biecek, P., Młynarczyk, M., Rytel, J., Górka, M., Kacprzyk, P., Dutka, M., Rodzaj, M., Bołkuń, Ł., et al. (2019) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2019.01.002 · Cited by 21
Ball, B. J., & Stein, E. M. (2019) · Haematologica · doi:10.3324/haematol.2018.208587 · Cited by 21
Treatment approaches in relapsed or refractory peripheral T-cell lymphomas
Foster, C. J., & Kuruvilla, J. G. (2020) · F1000Research · doi:10.12688/f1000research.22257.1 · Cited by 21
Kaito, S., & Iwama, A. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms22010074 · Cited by 21
Adès, L., Duployez, N., Guerci‐Bresler, A., Laribi, K., Peterlin, P., Vey, N., Thepot, S., Wickenhauser, S., Zerazhi, H., Stamatoullas, A., et al. (2022) · British Journal of Haematology · doi:10.1111/bjh.18193 · Cited by 21
Self-citationThépot, S., Boehrer, S., Seegers, V., Prébet, T., Beyne‐Rauzy, O., Wattel, E., Delaunay, J., Raffoux, E., Hunault, M., Jourdan, É., et al. (2014) · Leukemia Research · doi:10.1016/j.leukres.2014.09.014 · Cited by 20
“Current reference treatments for higher risk MDS are hypomethylating agents, mainly azacitidine (AZA), that has demonstrated a survival improvement over conventional treatments [1].”
BackgroundSelf-citationPerspectives and future directions for epigenetics in hematology
Goodell, M. A., & Godley, L. A. (2013) · Blood · doi:10.1182/blood-2013-04-427724 · Cited by 20
Optimizing hypomethylating agents in myelodysplastic syndromes
Itzykson, R., & Fenaux, P. (2012) · Current Opinion in Hematology · doi:10.1097/moh.0b013e32834ff58a · Cited by 20
“Randomized comparison in the AZA-001 trial was hampered by the small number of patients eligible for intensive chemotherapy before randomization, but the CR rate appeared higher with intensive chemotherapy than with AZA [1].”
BackgroundSelf-citationMerkerová, M. D., Remešová, H., Krejčík, Z., Loudová, N., Hruštincová, A., Szikszai, K., Čermák, J., Jonasova, A. T., & Beličková, M. (2018) · Cells · doi:10.3390/cells7090138 · Cited by 20
Bewersdorf, J. P., & Zeidan, A. M. (2021) · Best Practice & Research Clinical Haematology · doi:10.1016/j.beha.2021.101245 · Cited by 18
Paro, R., Grossniklaus, U., Santoro, R., & Wutz, A. (2021) · Learning materials in biosciences · doi:10.1007/978-3-030-68670-3 · Cited by 26
Evolving Therapeutic Approaches for Older Patients with Acute Myeloid Leukemia in 2021
Urbino, I., Secreto, C., Olivi, M., Apolito, V., D’Ardìa, S., Frairia, C., Giai, V., Aydin, S., Freilone, R., Dellacasa, C. M., et al. (2021) · Cancers · doi:10.3390/cancers13205075 · Cited by 20
Wang, L., Zhang, Q., Ye, L., Ye, X., Yang, W., Zhang, H., Zhou, X., Ren, Y., Ma, L., Zhang, X., et al. (2022) · British Journal of Cancer · doi:10.1038/s41416-022-02074-0 · Cited by 20
“The response rate of complete remission + partial remission (CR+ PR) was 19–29% [6, 7], and that of CR+ PR+marrow complete remission (mCR)+ haematological improvement (HI) was 47.”
BackgroundEpigenetic Regulations in Autoimmunity and Cancer: from Basic Science to Translational Medicine
Li, H., Zhao, Z., Chao, L., Yu, B., Li, A., & Wu, H. (2023) · European Journal of Immunology · doi:10.1002/eji.202048980 · Cited by 20
TP53 in AML and MDS: The new (old) kid on the block
Marks, J. A., Wang, X., Fenu, E. M., Bagg, A., & Lai, C. (2023) · Blood Reviews · doi:10.1016/j.blre.2023.101055 · Cited by 20
Rajakumaraswamy, N., Gandhi, M. D., Wei, A. H., Sallman, D. A., Daver, N. G., Mo, S., Iqbal, S., Karalliyadda, R., Chen, M., Wang, Y., et al. (2023) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2023.12.008 · Cited by 20
Haumschild, R. J., Kennerly-Shah, J. M., Barbarotta, L., & Zeidan, A. M. (2024) · Journal of Oncology Pharmacy Practice · doi:10.1177/10781552241238979 · Cited by 20
Myelodysplastic Neoplasms (MDS): The Current and Future Treatment Landscape
Karel, D., Valburg, C., Woddor, N., Nava, V. E., & Aggarwal, A. (2024) · Current Oncology · doi:10.3390/curroncol31040148 · Cited by 20
“There was an improvement in overall survival in participants who received azacitidine when compared to the “conventional care” arm with a median survival of 24.5 months vs. 15 months, respectively [58].”
BackgroundInfluentialDuong, V. H., Lancet, J. E., Alrawi, E., Al‐Ali, N., Perkins, J. B., Field, T. L., Epling‐Burnette, P. K., Zhang, L., List, A. F., & Komrokji, R. S. (2013) · Leukemia Research · doi:10.1016/j.leukres.2012.12.012 · Cited by 20
“5-month median overall survival [7].”
BackgroundResultInfluentialSelf-citationSteensma, D. P., & Stone, R. M. (2010) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2010.02.012 · Cited by 20
Pretransplantation 5-Azacitidine in High-Risk Myelodysplastic Syndrome
Nishihori, T., Perkins, J. B., Mishra, A., Komrokji, R. S., Kim, J., Kharfan‐Dabaja, M. A., Perez, L. E., Lancet, J. E., Fernandez, H. F., List, A. F., et al. (2014) · Transplantation and Cellular Therapy · doi:10.1016/j.bbmt.2014.02.008 · Cited by 20
Self-citationBenjamin, J., Chhabra, S., Kohrt, H. E. K., Lavori, P. W., Laport, G. G., Arai, S., Johnston, L., Miklos, D. B., Shizuru, J. A., Weng, W., et al. (2014) · Transplantation and Cellular Therapy · doi:10.1016/j.bbmt.2014.02.023 · Cited by 20
“Recently, therapies for MDS, such as DNA methyltransferase inhibitors (DNMTi), have been shown to alter the natural course of the disease in 40% to 50% of high-risk patients, yet responses are often incomplete and rarely durable [1,2].”
Pfeilstöcker, M., Tüchler, H., Schönmetzler, A., Nösslinger, T., & Pittermann, E. (2011) · Leukemia Research · doi:10.1016/j.leukres.2011.09.007 · Cited by 20
“007 phase 3 trial [4].”
BackgroundLevy, A. R., Zou, D., Risebrough, N. A., Buckstein, R. J., Kim, T., & Brereton, N. J. (2013) · Current Oncology · doi:10.3747/co.21.1311 · Cited by 20
Buckstein, R. J., Kerbel, R. S., Cheung, M. C., Shaked, Y., Chodirker, L., Lee, C. R., Lenis, M., Davidson, C., Cussen, M., Reis, M. D., et al. (2014) · Leukemia Research · doi:10.1016/j.leukres.2014.03.022 · Cited by 20
Clinical utility of lenalidomide in the treatment of myelodysplastic syndromes
Zeidan, A. M., Zahr, A. A., Aldin, E. S., & Komrokji, R. S. (2014) · Journal of Blood Medicine · doi:10.2147/jbm.s50482 · Cited by 20
Cluzeau, T., Moreilhon, C., Mounier, N., Karsenti, J.-M., Gastaud, L., Garnier, G., Re, D., Montagne, N., Gutnecht, J., Auberger, P., et al. (2013) · Blood Cancer Journal · doi:10.1038/bcj.2013.52 · Cited by 20
Saber, W., Rademacher, J. L., Sekeres, M. A., Logan, B. R., Lewis, M., Mendizabal, A. M., Leifer, E., Appelbaum, F. R., Horowitz, M. M., Nakamura, R., et al. (2014) · Transplantation and Cellular Therapy · doi:10.1016/j.bbmt.2014.06.010 · Cited by 20
Grövdal, M., Karimi, M., Tobiasson, M., Reinius, L. E., Jansson, M., Ekwall, K., Ungerstedt, J. S., Kere, J., Greco, D., & Hellström‐Lindberg, E. (2013) · Leukemia · doi:10.1038/leu.2013.265 · Cited by 20
Tang, H., Tian, E., Liu, C., Wang, Q., & Deng, H. (2013) · PLoS ONE · doi:10.1371/journal.pone.0059610 · Cited by 20
Epigenetic regulation in myelodysplastic syndromes: implications for therapy
Vigna, E., Recchia, A. G., Madeo, A., Gentile, M., Bossio, S., Mazzone, C., Lucia, E., Morabito, L., Gigliotti, V., Stefano, L. D., et al. (2011) · Expert Opinion on Investigational Drugs · doi:10.1517/13543784.2011.559164 · Cited by 20
“More importantly, 5-AZA showed a significant effect on overall survival (OS) in a randomized, Phase III study [75].”
BackgroundThe clinical use of DNA methyltransferase inhibitors in myelodysplastic syndromes
Zahr, A. A., Aldin, E. S., Barbarotta, L., Podoltsev, N. A., & Zeidan, A. M. (2015) · Expert Review of Anticancer Therapy · doi:10.1586/14737140.2015.1061936 · Cited by 20
“patients in the BSC arm fared better than those in the azacitidine trials [13,14].”
BackgroundResultInfluentialTabarroki, A., Saunthararajah, Y., Visconte, V., Cinalli, T., Colaluca, K., Rogers, H. J., Sekeres, M. A., Duong, H. K., Stein, B. L., & Tiu, R. V. (2014) · Leukemia & lymphoma/Leukemia and lymphoma · doi:10.3109/10428194.2014.916805 · Cited by 20
Targeting Splicing in the Treatment of Myelodysplastic Syndromes and Other Myeloid Neoplasms
Brierley, C. K., & Steensma, D. P. (2016) · Current Hematologic Malignancy Reports · doi:10.1007/s11899-016-0344-z · Cited by 20
Therapy-related myeloid neoplasms - what have we learned so far?
Zahid, M. F., Parnes, A. D., Savani, B. N., Litzow, M. R., & Hashmi, S. K. (2016) · World Journal of Stem Cells · doi:10.4252/wjsc.v8.i8.231 · Cited by 20
Nanah, R., McCullough, K. B., Hogan, W. J., Begna, K. H., Patnaik, M. M., Elliott, M. A., Litzow, M. R., & Al‐Kali, A. (2017) · American Journal of Hematology · doi:10.1002/ajh.24780 · Cited by 20
Kubasch, A. S., & Platzbecker, U. (2018) · Cancers · doi:10.3390/cancers10060158 · Cited by 20
“Response rates comprised 49% of AZA-treated patients, including an improvement of blood counts (hematologic improvement (HI) according to IWG criteria), as well as 29% of patients with either complete remission (CR) or partial remission (PR) [14].”
BackgroundMethodInfluentialINFECTIONS IN MYELODYSPLASTIC SYNDROME IN RELATION TO STAGE AND THERAPY
Leone, G. G. M., Pagano, L., & Pagano, L. (2018) · Mediterranean Journal of Hematology and Infectious Diseases · doi:10.4084/mjhid.2018.039 · Cited by 22
“In the recent experience reported by Fenaux et al.44 comparing the behavior of patients at different ages, the adverse events (AEs) in the ≥75 years group were compared with the 65 years group.”
BackgroundInfluentialMorita, Y., Maeda, Y., Yamaguchi, T., Urase, F., Kawata, S., Hanamoto, H., Tsubaki, K., Ishikawa, J., Shibayama, H., Matsumura, I., et al. (2018) · Cancer Science · doi:10.1111/cas.13739 · Cited by 20
“A hypomethylating agent (HMA), azacitidine, was reported to improve overall survival (OS) for higher‐risk MDS (including acute myeloid leukemia by WHO classification) with a 7‐day administration schedule (AZA‐7) compared with conventional therapies.(2) In addition, a phase 1/2 AZA‐7 study in Japan demonstrated that AZ…”
BackgroundAl‐Kali, A., Zblewski, D., Foran, J. M., Patnaik, M. M., Larrabee, B. R., Gangat, N., Begna, K. H., Elliott, M. A., Hogan, W. J., Tefferi, A., et al. (2019) · Mayo Clinic Proceedings · doi:10.1016/j.mayocp.2019.02.029 · Cited by 22
Wu, P., Weng, J., Li, M., Lu, Z., Deng, C., Sun, Q., Xu, R., Geng, S., & Du, X. (2019) · Europe PMC (PubMed Central) · Cited by 20
Oral Azacitidine and Cedazuridine Approximate Parenteral Azacitidine Efficacy in Murine Model
Ramsey, H. E., Oganesian, A., Gorska, A. E., Fuller, L., Arrate, M. P., Boyd, K. L., Keer, H. N., Azab, M., & Savona, M. R. (2020) · Targeted Oncology · doi:10.1007/s11523-020-00709-x · Cited by 20
Talati, C., Goldberg, A. D., Przespolewski, A., Chan, O., Ali, N. E. A., Kim, J., Famulare, C., Sallman, D. A., Padron, E., Kuykendall, A. T., et al. (2020) · Leukemia Research · doi:10.1016/j.leukres.2020.106367 · Cited by 20
Renneville, A., Patnaik, M. M., Chan, O., Padron, E., & Solary, É. (2021) · Leukemia · doi:10.1038/s41375-021-01330-1 · Cited by 21
Saliba, A. N., Litzow, M. R., Gangat, N., Al‐Kali, A., Foran, J. M., Hogan, W. J., Palmer, J. M., Mangaonkar, A. A., Tefferi, A., & Patnaik, M. M. (2021) · American Journal of Hematology · doi:10.1002/ajh.26334 · Cited by 20
“Food and Drug Administration (FDA) approved therapies for CMML, largely based on the inclusion of a limited number of predominantly dysplastic CMML patients in MDS centered trials.(4) The prospective DACOTA trial was the first to randomize higher risk proliferative CMML patients to decitabine versus hydroxyurea and sh…”
BackgroundSadashiv, S. K., Hilton, C. J., Khan, C., Rossetti, J. M., Benjamin, H. L., Fazal, S., Sahovic, E., Shadduck, R. K., & LISTER, J. W. (2014) · Cancer Medicine · doi:10.1002/cam4.321 · Cited by 20
“Hence, many patients aged >65 years may not be candidates for intensive chemotherapy regimens [13, 14].”
BackgroundResultInfluentialGore, S. D. (2009) · Leukemia Research · doi:10.1016/s0145-2126(09)70226-7 · Cited by 20
Self-citationLeisch, M., Weiss, L., Lindlbauer, N., Jungbauer, C., Egle, A., Rohde, E., Greil, R., Grabmer, C., & Pleyer, L. (2017) · Leukemia Research · doi:10.1016/j.leukres.2017.05.006 · Cited by 20
The impact of antimicrobial prophylaxis in morbidity and infections during azacitidine treatment
Lorenzana-Blanco, N., Avila, L. F., Alonso, S., Colado, E., & Bernal, T. (2017) · Annals of Hematology · doi:10.1007/s00277-017-3091-x · Cited by 20
Davidoff, A. J., Hu, X. Y., Bewersdorf, J. P., Wang, R., Podoltsev, N. A., Huntington, S. F., Gore, S. D., Ma, X., & Zeidan, A. M. (2019) · Leukemia & lymphoma/Leukemia and lymphoma · doi:10.1080/10428194.2019.1703970 · Cited by 20
“5months when compared to conventional care among HR-MDS patients [2].”
BackgroundResultSelf-citationLübbert, M., & Kuendgen, A. (2014) · Cancer · doi:10.1002/cncr.29083 · Cited by 19
Treatment of Unfit Patients With Acute Myeloid Leukemia: A Still Open Clinical Challenge
Ferrara, F. (2011) · Clinical Lymphoma Myeloma & Leukemia · doi:10.3816/clml.2011.n.001 · Cited by 19
Almasri, J., Alkhateeb, H. B., Firwana, B., Sonbol, M. B., Damlaj, M., Wang, Z., Murad, M. H., & Al‐Kali, A. (2018) · Systematic Reviews · doi:10.1186/s13643-018-0805-7 · Cited by 20
Ishiyama, K., Aoki, J., Itonaga, H., Uchida, N., Takahashi, S., Ohno, Y., Matsuhashi, Y., Sakura, T., Onizuka, M., Miyakoshi, S., et al. (2019) · Blood Cancer Journal · doi:10.1038/s41408-019-0192-x · Cited by 19
Modi, D., Kim, S., Singh, V., Ayash, L. J., Alavi, A., Ratanatharathorn, V., Uberti, J. P., & Deol, A. (2019) · Leukemia & lymphoma/Leukemia and lymphoma · doi:10.1080/10428194.2019.1605070 · Cited by 19
“Treatment with hypomethylating agents (HMAs), like azacitidine and decitabine, can induce CR rate of 10–20%, and hematological improvement in 23–49% patients and improved overall survival (OS) in intermediate-2 and high risk MDS patients [14,15].”
Background5-Azacytidine modulates CpG methylation levels of EZH2 and NOTCH1 in myelodysplastic syndromes
Gawlitza, A. L., Speith, J., Rinke, J., Sajzew, R., Müller, E. K., Schäfer, V., Hochhaus, A., & Ernst, T. (2019) · Journal of Cancer Research and Clinical Oncology · doi:10.1007/s00432-019-03016-9 · Cited by 19
Lee, B., Kang, K., Jeon, M. J., Yu, E. S., Kim, D. S., Choi, H., Lee, S. R., Sung, H. J., Kim, B. S., Choi, C. W., et al. (2020) · Scientific Reports · doi:10.1038/s41598-019-56642-1 · Cited by 19
Kiesewetter, B., Cherny, N. I., Boissel, N., Cerisoli, F., Dafni, U. G., de Vries, E. G. E., Ghia, P., Gökbuget, N., González‐Calle, V., Huntly, B. J. P., et al. (2020) · ESMO Open · doi:10.1136/esmoopen-2019-000611 · Cited by 19
Evolving therapies for lower-risk myelodysplastic syndromes
Bewersdorf, J. P., & Zeidan, A. M. (2020) · Annals of Hematology · doi:10.1007/s00277-020-03963-1 · Cited by 19
Tanaka, U., Kajioka, S., Finoti, L. S., Palioto, D. B., Kinane, D. F., & Benakanakere, M. R. (2021) · Biomedicines · doi:10.3390/biomedicines9020199 · Cited by 19
“The most common treatment for MDS include hypomethylating agents such as azacytidine and decitabine [18].”
BackgroundCurrent Therapy of the Patients with MDS: Walking towards Personalized Therapy
Palacios‐Berraquero, M. L., & Piérola, A. A. (2021) · Journal of Clinical Medicine · doi:10.3390/jcm10102107 · Cited by 19
“In addition, in a randomized trial, the median OS was 9 months lower than with AZA, although the differences were not significant due to the small number of patients who were candidates for intensive chemotherapy [46].”
BackgroundPersonalized Medicine for TP53 Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia
Cluzeau, T., Loschi, M. L., Fenaux, P., Komrokji, R. S., & Sallman, D. A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms221810105 · Cited by 20
“5 months and the overall response rate (ORR) was 29%, including 17% of patients experiencing complete remission (CR) [18].”
BackgroundSelf-citationZeidan, A. M., Tsai, J., Karimi, M., Schmier, J. K., Jayade, S. P., Zormpas, E., Hassan, A., Ruiters, D., Anthony, C., Hill, K., et al. (2022) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2022.04.023 · Cited by 19
Moyo, T. K., Mendler, J. H., Itzykson, R., Kishtagari, A., Solary, É., Seegmiller, A. C., Gerds, A. T., Ayers, G. D., Dezern, A. E., Nazha, A., et al. (2022) · BMC Cancer · doi:10.1186/s12885-022-10073-w · Cited by 19
Self-citationGerke, M. B., Christodoulou, I., & Karantanos, T. (2023) · Cancers · doi:10.3390/cancers15153815 · Cited by 19
“The overall response rate (ORR) was 29% with AZA treatment, compared to 12% in conventional care regimes [39].”
BackgroundZha, J., Bi, S., Deng, M., Chen, K., Shi, P., Feng, L., He, J., Pu, X., Guo, C., Zhao, H., et al. (2021) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2021.174107 · Cited by 18
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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
