Decitabine: An effective and safe treatment for myelodysplastic syndrome and acute myeloid leukemia

J Cancer Res Ther. 2019;15(7):1471-1476. doi: 10.4103/0973-1482.204849.


Objective: Decitabine is reported to be valuable in treating multiple malignant blood diseases. However, the application of decitabine in myelodysplastic syndromes (MDSs) and acute myeloid leukemia (AML) has not been fully examined. Thus, our study aimed to investigate the clinical efficacy and safety of decitabine in treating such patients.

Materials and methods: Clinical data of MDS or AML patients treated with decitabine were retrospectively analyzed. All the patients were regularly followed up, and the risk factors affecting clinical efficacy were also detected.

Results: A total of 36 patients (MDS, n = 27; AML, n = 9) were included in the study. The response rate of MDS patients was 55%, and there were three cases (15%) of complete remission (CR), three cases (15%) of marrow CR, and five cases (15%) of hematologic improvement. It was about three cycles to achieve the best efficiencies. Gender, age, percentage of blasts in bone marrow, International Prognostic Scoring System risk group, and cytogenetic factors were not associated with response rate. The median overall survival of MDS patients was 8 (1-44) months. Agranulocytosis (P = 0.037) and severe anemia (P = 0.044) were the independent factors for prognosis. The complete response rate of AML was 33.3%. From the investigation, infection was the most common complication in our cohort, especially lung infection with the incidence of 27.8%.

Conclusions: Our data demonstrated that decitabine was effective and relatively safe in treating MDS and AML. Patients with agranulocytosis and severe anemia were prone to have poor survival, which should be monitored in clinical practice.

Keywords: Acute myeloid leukemia; chemotherapy; clinical efficacy; decitabine; myelodysplastic syndromes; safety.

MeSH terms

  • Aged
  • Aged, 80 and over
  • Antimetabolites, Antineoplastic / administration & dosage
  • Antimetabolites, Antineoplastic / adverse effects
  • Antimetabolites, Antineoplastic / therapeutic use*
  • Decitabine / administration & dosage
  • Decitabine / adverse effects
  • Decitabine / therapeutic use*
  • Female
  • Follow-Up Studies
  • Humans
  • Leukemia, Myeloid, Acute / diagnosis
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / etiology
  • Leukemia, Myeloid, Acute / mortality
  • Male
  • Middle Aged
  • Myelodysplastic Syndromes / diagnosis
  • Myelodysplastic Syndromes / drug therapy*
  • Myelodysplastic Syndromes / etiology
  • Myelodysplastic Syndromes / mortality
  • Prognosis
  • Survival Analysis
  • Treatment Outcome


  • Antimetabolites, Antineoplastic
  • Decitabine