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Updated: May 18, 2026
06:39
Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Epigenetic therapies in MDS and AML
Elizabeth A Griffiths1, Steven D Gore
1Roswell Park Cancer Institute, Buffalo, NY, USA. elizabeth.griffiths@roswellpark.org
Advances in Experimental Medicine and Biology
|September 8, 2012
Summary
Low-dose hypomethylating agents like azacitidine and decitabine offer improved quality of life for myelodysplastic syndrome (MDS) and secondary acute myeloid leukemia (AML) patients. These outpatient therapies provide significant clinical benefits despite low complete remission rates.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Myelodysplastic syndromes (MDS) and secondary acute myeloid leukemia (AML) historically had limited, toxic treatment options.
- Traditional chemotherapy for MDS and AML was often inpatient-based and associated with significant toxicity and limited efficacy.
Purpose of the Study:
- To evaluate the impact and role of low-dose hypomethylating agents in treating MDS and secondary AML.
- To highlight the benefits of novel outpatient therapies compared to older treatment regimens.
Main Methods:
- Utilized hypomethylating agents such as azacitidine and decitabine.
- Focused on outpatient administration and assessment of clinical outcomes and quality of life.
Main Results:
- Achieved complete remission (CR) rates of 15-20% with hypomethylating agents.
- Observed significant improvements in hemoglobin, platelet, and neutrophil counts in a larger patient percentage.
- Maintained good outpatient quality of life for patients receiving these therapies.
Conclusions:
- Low-dose hypomethylating agents represent a significant advancement in MDS and AML treatment, offering improved quality of life.
- Ongoing research explores combinations and sequences with other therapies to optimize outcomes for myeloid neoplasms.
- The precise mechanisms of action, including DNA hypomethylation, direct DNA damage, or immune modulation, are still under investigation but efficacy is undeniable.
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