Related Experiment Video
Updated: Jun 3, 2026
Comet Assay to Quantify DNA Damage in FLT3 Mutant-expressing 32D Cells after Exposure to Type I and Type II FLT3 Inhibitors
Published on: October 17, 2025
Azacitidine has limited activity in 'real life' patients with MDS and AML: a single centre experience
Murat Ozbalak1, Mustafa Cetiner, Huseyin Bekoz
1Division of Hematology, Department of Internal Medicine, Istanbul University Cerrahpasa Medical Faculty, Istanbul, Turkey.
Abstract:
Myelodysplastic syndrome (MDS) represents a heterogeneous group of potentially malignant diseases of bone-marrow stem cells. Acute myelogenous leukaemia (AML) is an inevitable outcome for many patients with MDS. Azacitidine has been reported to result in comparably higher response rates and improved survival than other treatment strategies. In this retrospective study, we report the results on 25 'real life' patients with MDS, CMML or AML treated with azacitidine between 2005 and 2009. All patients fulfilled the World Health Organization criteria for MDS and AML. No eligibility criteria other than diagnosis were considered. Complete response (CR) rate was observed in three of the 25 'real life' patients (12%) with a median duration of CR of 5 months (4-6 months). Seven patients (28%) had mono- or bi-lineage haematologic improvement and 15 patients (60%) showed neither morphologic nor haematologic response. Among 17 non-AML patients, the median time from onset of Aza-C treatment to AML transformation was 10 months (4-15 months). Overall death rate was 72%. All of the eight AML patients died. The death rate under Aza-C among non-AML patients was 59%. Unlike the results of the clinical trials, our data show that Aza-C has a limited activity in 'real-life' patients with MDS and AML. It is obvious that Aza-C can induce complete or partial responses in a considerable number of MDS patients but responses are usually not durable as we observed in our patients.
Insights
This study found that azacitidine showed limited effectiveness in
Area of Science:
- Hematology
- Oncology
- Stem Cell Biology
Background:
- Myelodysplastic syndrome (MDS) is a group of bone marrow diseases.
- Acute myelogenous leukemia (AML) often develops from MDS.
- Azacitidine is a treatment option for MDS and AML.
Purpose of the Study:
- To evaluate the effectiveness of azacitidine in real-world MDS and AML patients.
- To compare real-world outcomes with clinical trial results.
- To assess response rates, duration of response, and survival in patients treated with azacitidine.
Main Methods:
- Retrospective study of 25 patients with MDS, CMML, or AML treated with azacitidine (2005-2009).
- Patients met World Health Organization criteria for MDS and AML.
- No eligibility criteria other than diagnosis were applied.
Main Results:
- Complete response (CR) rate was 12% (3/25 patients), with a median CR duration of 5 months.
- Hematologic improvement (mono- or bi-lineage) was seen in 28% (7/25) of patients.
- 60% (15/25) of patients showed no morphologic or hematologic response.
- Median time to AML transformation in non-AML patients was 10 months.
- Overall mortality rate was 72%, with all 8 AML patients dying.
- Mortality rate among non-AML patients treated with azacitidine was 59%.
Conclusions:
- Azacitidine demonstrated limited activity and durability in real-world MDS and AML patients.
- Observed responses were generally not sustained, contrasting with clinical trial findings.
- Further research is needed to optimize azacitidine use and explore alternative strategies for these patient populations.