title: [Outcomes of refractory or relapsed DNMT3A + cytogenetically normal acute myeloid leukemia patients followed the therapy including decitabine combined with CAG or CAG-like regimen].
abstract: OBJECTIVE
  To study clinical characteristics of refractory or relapsed DNMT3A⁺ cytogenetically normal acute myeloid leukemia（CN-AML）patients, and to explore the overall response rate（ORR）and side effects of these patients followed the therapy including decitabine with CAG or CAGlike regimen.
  
  
  METHODS
  In this study we retrospectively analyzed 53 refractory or relapsed CN- AML patients receiving the therapy including decitabine combined with CAG and CAG- like regimen in our center from April 2011 to October 2014. The clinical characteristics and ORR were further analyzed. Based on gene mutations, these patients could be divided into 2 groups: DNMT3A⁺ AML patients（n=24）and DNMT3A- AML patients（n=29）.
  
  
  RESULTS
  The median age of DNMT3A⁺AML patients was 46 years old, higher white blood cells and bone marrow blasts were observed in DNMT3A+ AML group. The ORR and complete response（CR）rate of DNMT3A+ group were 62.50% and 54.17%, respectively. No differences were observed in ORR and CR rates（P>0.05）between these two groups. DNMT3A⁺/FLT3-ITD⁺ CN-AML patients（n=14）had higher ORR and CR rates than DNMT3A-/FLT3-ITD⁺CN- AML patients（n=15）（P= 0.040 and 0.042, respectively）. The one- year overall survival （OS） of DNMT3A⁺ AML group and DNMT3A- AML group were 59.58% , 54.09% , no differences were observed （P=0.438）. 25 patients received further therapy of allo-HSCT, the one-year OS of DNMT3A⁺ CN-AML was 87.50% and one-year disease free survival（DFS）was 72.73%, while the one- year OS was 61.54% and one- year DFS was 58.02% in DNMT3A⁻ group. No differences were observed between 2 groups （P=0.456, 0.217）.
  
  
  CONCLUSIONS
  Decitabine combined with CAG or CAG-like regimen was an effective and safe treatment for refractory or relapsed CN- AML patients. Compared to DNMT3A⁻/FLT3- ITD⁺ CN- AML patients, DNMT3A⁺/ FLT3-ITD⁺ CN-AML patients had higher ORR and CR rates. Decitabine bridged hematopoietic stem cells transplant could likely improve the survival of refractory or relapsed CN-AML patients.
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Yanjun
authors.initials: Y
authors.lastname: Sun
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Yang
authors.initials: Y
authors.lastname: Xu
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Depei
authors.initials: D
authors.lastname: Wu
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Hongjie
authors.initials: H
authors.lastname: Shen
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Zhen
authors.initials: Z
authors.lastname: Yang
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Huiying
authors.initials: H
authors.lastname: Qiu
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Suning
authors.initials: S
authors.lastname: Chen
authors.affiliation: Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou 215006, China.
authors.forename: Aining
authors.initials: A
authors.lastname: Sun
mesh_terms: D015250:Aclarubicin / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001374:Azacitidine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D003561:Cytarabine / Q000627:therapeutic use; D004248:DNA (Cytosine-5-)-Methyltransferases / Q000235:genetics*; D000090205:DNA Methyltransferase 3A; D000077209:Decitabine; D018572:Disease-Free Survival; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000628:therapy*; D009154:Mutation; D012074:Remission Induction; D012189:Retrospective Studies
chemical_list: C000717967:DNMT3A protein, human; D003561:Cytarabine; D016179:Granulocyte Colony-Stimulating Factor; D015250:Aclarubicin; D000077209:Decitabine; D004248:DNA (Cytosine-5-)-Methyltransferases; D000090205:DNA Methyltransferase 3A; D001374:Azacitidine