Postazacitidine clone size predicts long-term outcome of patients with myelodysplastic syndromes and related myeloid neoplasms

Key Points • Major clone sizes in posttreatment samples are strongly correlated with clinical response.• Inclusion of posttreatment clone size into the prognostic model allows better prognostication for MDS cases treated with azacitidine.

[1]  Y. Kamatani,et al.  Germline DDX41 mutations define a unique subtype of myeloid neoplasms. , 2022, Blood.

[2]  T. Naoe,et al.  Prospective comparison of 5- and 7-day administration of azacitidine for myelodysplastic syndromes: a JALSG MDS212 trial , 2022, International Journal of Hematology.

[3]  L. Milanesi,et al.  Classification and Personalized Prognostic Assessment on the Basis of Clinical and Genomic Features in Myelodysplastic Syndromes , 2021, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[4]  A. Viale,et al.  Implications of TP53 allelic state for genome stability, clinical presentation and outcomes in myelodysplastic syndromes , 2020, Nature Medicine.

[5]  M. Konopleva,et al.  Genomic context and TP53 allele frequency define clinical outcomes in TP53-mutated myelodysplastic syndromes. , 2020, Blood advances.

[6]  L. Mannone,et al.  Clonal selection in therapy‐related myelodysplastic syndromes and acute myeloid leukemia under azacitidine treatment , 2020, European journal of haematology.

[7]  M. Tormo,et al.  Impact of clinical features, cytogenetics, genetic mutations, and methylation dynamics of CDKN2B and DLC-1 promoters on treatment response to azacitidine , 2020, Annals of Hematology.

[8]  P. Kuang,et al.  Hypomethylating agents for patients with myelodysplastic syndromes prior to hematopoietic stem cell transplantation: a systematic review and meta-analysis , 2019, Annals of Hematology.

[9]  Elena K. Müller,et al.  5-Azacytidine modulates CpG methylation levels of EZH2 and NOTCH1 in myelodysplastic syndromes , 2019, Journal of Cancer Research and Clinical Oncology.

[10]  G. Garcia-Manero,et al.  Proposals for revised IWG 2018 hematological response criteria in patients with MDS included in clinical trials. , 2019, Blood.

[11]  S. Tsunoda,et al.  Age-related remodelling of oesophageal epithelia by mutated cancer drivers , 2019, Nature.

[12]  T. Haferlach,et al.  A Personalized Prediction Model to Risk Stratify Patients with Myelodysplastic Syndromes , 2018, Blood.

[13]  M. Voso,et al.  Somatic mutations as markers of outcome after azacitidine and allogeneic stem cell transplantation in higher-risk myelodysplastic syndromes , 2018, Leukemia.

[14]  L. Bullinger,et al.  Cytogenetics and gene mutations influence survival in older patients with acute myeloid leukemia treated with azacitidine or conventional care , 2018, Leukemia.

[15]  Li-li Wang,et al.  Predictors of clinical responses to hypomethylating agents in acute myeloid leukemia or myelodysplastic syndromes , 2018, Annals of Hematology.

[16]  T. Haferlach,et al.  Efficacy of azacitidine is independent of molecular and clinical characteristics - an analysis of 128 patients with myelodysplastic syndromes or acute myeloid leukemia and a review of the literature , 2018, Oncotarget.

[17]  E. Solary,et al.  Prognostic Role of Gene Mutations in Chronic Myelomonocytic Leukemia Patients Treated With Hypomethylating Agents , 2018, EBioMedicine.

[18]  W. Hiddemann,et al.  Persistence of pre-leukemic clones during first remission and risk of relapse in acute myeloid leukemia , 2017, Leukemia.

[19]  A. Lucia,et al.  Mutations in the DNA methylation pathway and number of driver mutations predict response to azacitidine in myelodysplastic syndromes , 2017, Oncotarget.

[20]  Susan R. Wilson,et al.  Integrative Genomics Identifies the Molecular Basis of Resistance to Azacitidine Therapy in Myelodysplastic Syndromes. , 2017, Cell reports.

[21]  H. Deeg,et al.  Mutational analysis in serial marrow samples during azacitidine treatment in patients with post-transplant relapse of acute myeloid leukemia or myelodysplastic syndromes , 2017, Haematologica.

[22]  S. Miyano,et al.  Genetic abnormalities in myelodysplasia and secondary acute myeloid leukemia: impact on outcome of stem cell transplantation. , 2017, Blood.

[23]  S. Miyano,et al.  Clonal evolution in myelodysplastic syndromes , 2017, Nature Communications.

[24]  G. Garcia-Manero,et al.  Decitabine in TP53-Mutated AML. , 2017, The New England journal of medicine.

[25]  D. Neuberg,et al.  Prognostic Mutations in Myelodysplastic Syndrome after Stem‐Cell Transplantation , 2017, The New England journal of medicine.

[26]  L. Bullinger,et al.  Evaluating the impact of genetic and epigenetic aberrations on survival and response in acute myeloid leukemia patients receiving epigenetic therapy , 2017, Annals of Hematology.

[27]  Christopher A. Miller,et al.  TP53 and Decitabine in Acute Myeloid Leukemia and Myelodysplastic Syndromes. , 2016, The New England journal of medicine.

[28]  A. Trumpp,et al.  Mutational hierarchies in myelodysplastic syndromes dynamically adapt and evolve upon therapy response and failure. , 2016, Blood.

[29]  Y. Chung,et al.  Somatic mutations predict outcomes of hypomethylating therapy in patients with myelodysplastic syndrome , 2016, Oncotarget.

[30]  B. Quesnel,et al.  Molecular prognostic factors in acute myeloid leukemia receiving first-line therapy with azacitidine , 2016, Leukemia.

[31]  S. Nahas,et al.  Impact of TP53 mutation variant allele frequency on phenotype and outcomes in myelodysplastic syndromes , 2016, Leukemia.

[32]  M. Stratton,et al.  Mutation allele burden remains unchanged in chronic myelomonocytic leukaemia responding to hypomethylating agents , 2016, Nature Communications.

[33]  M. Konopleva,et al.  Clinical implications of TP53 mutations in myelodysplastic syndromes treated with hypomethylating agents , 2016, Oncotarget.

[34]  S. Papageorgiou,et al.  Treatment with 5-Azacytidine improves clinical outcome in high-risk MDS patients in the ‘real life’ setting: A single center observational study , 2016, Hematology.

[35]  Christopher A. Miller,et al.  Dynamic Changes in the Clonal Structure of MDS and AML in Response to Epigenetic Therapy , 2016, Leukemia.

[36]  G. Mufti,et al.  Mutations in histone modulators are associated with prolonged survival during azacitidine therapy , 2015, Oncotarget.

[37]  M. McCarthy,et al.  Age-related clonal hematopoiesis associated with adverse outcomes. , 2014, The New England journal of medicine.

[38]  B. Quesnel,et al.  Molecular Prognostic Factors in Acute Myeloid Leukemia (AML) Patients Receiving First Line Therapy with Azacytidine (AZA) , 2014 .

[39]  S. Gabriel,et al.  Clonal hematopoiesis and blood-cancer risk inferred from blood DNA sequence. , 2014, The New England journal of medicine.

[40]  D. Neuberg,et al.  TET2 mutations predict response to hypomethylating agents in myelodysplastic syndrome patients. , 2014, Blood.

[41]  D. Neuberg,et al.  Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation. , 2014, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[42]  C. Preudhomme,et al.  Prognostic value of TP53 gene mutations in myelodysplastic syndromes and acute myeloid leukemia treated with azacitidine. , 2014, Leukemia research.

[43]  M. Konopleva,et al.  Lack of association of IDH1, IDH2 and DNMT3A mutations with outcome in older patients with acute myeloid leukemia treated with hypomethylating agents , 2014, Leukemia & lymphoma.

[44]  S. Parmar,et al.  Expression of PD-L1, PD-L2, PD-1 and CTLA4 in myelodysplastic syndromes is enhanced by treatment with hypomethylating agents , 2013, Leukemia.

[45]  M. Breccia,et al.  Revised IPSS (IPSS-R) stratification and outcome of MDS patients treated with azacitidine , 2013, Annals of Hematology.

[46]  Ken Chen,et al.  Clonal architecture of secondary acute myeloid leukemia. , 2012, The New England journal of medicine.

[47]  P. Fenaux,et al.  Predicting the outcome of patients with higher-risk myelodysplastic syndrome treated with hypomethylating agents , 2012, Leukemia & lymphoma.

[48]  M. Vignetti,et al.  Role of BCL2L10 methylation and TET2 mutations in higher risk myelodysplastic syndromes treated with 5-Azacytidine , 2011, Leukemia.

[49]  M. Esteller,et al.  DNMT3B gene amplification predicts resistance to DNA demethylating drugs , 2011, Genes, chromosomes & cancer.

[50]  B. Quesnel,et al.  Impact of TET2 mutations on response rate to azacitidine in myelodysplastic syndromes and low blast count acute myeloid leukemias , 2011, Leukemia.

[51]  A. Stamatoullas,et al.  Prognostic factors for response and overall survival in 282 patients with higher-risk myelodysplastic syndromes treated with azacitidine. , 2011, Blood.

[52]  Chris A. Nasrallah,et al.  Early epigenetic changes and DNA damage do not predict clinical response in an overlapping schedule of 5-azacytidine and entinostat in patients with myeloid malignancies. , 2009, Blood.

[53]  Valeria Santini,et al.  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. , 2009, The Lancet. Oncology.

[54]  B. Cheson,et al.  Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. , 2006, Blood.

[55]  L. Donehower,et al.  PPM1D dephosphorylates Chk1 and p53 and abrogates cell cycle checkpoints. , 2005, Genes & development.

[56]  Peter A. Jones,et al.  Demethylation of a hypermethylated P15/INK4B gene in patients with myelodysplastic syndrome by 5-Aza-2'-deoxycytidine (decitabine) treatment. , 2002, Blood.

[57]  J. Holland,et al.  Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. , 2002, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[58]  Y. Nagata.  [Molecular international prognostic scoring system for myelodysplastic syndromes]. , 2023, [Rinsho ketsueki] The Japanese journal of clinical hematology.

[59]  S. Geng,et al.  Co-occurrence of RUNX1 and ASXL1 mutations underlie poor response and outcome for MDS patients treated with HMAs. , 2019, American journal of translational research.

[60]  A. Jankowska,et al.  Impact of molecular mutations on treatment response to DNMT inhibitors in myelodysplasia and related neoplasms , 2014, Leukemia.

[61]  P. Font,et al.  Inter-observer variance with the diagnosis of myelodysplastic syndromes (MDS) following the 2008 WHO classification , 2012, Annals of Hematology.

[62]  R. Arceci.  Impact of TET2 mutations on response rate to azacitidine in myelodysplastic syndromes and low blast count acute myeloid leukemias , 2011 .