Identification of microRNA biomarkers predictive of clinical outcomes in Aplastic Anaemia and Myelodysplastic Syndrome

2016 – 2019 (Grant-in-Aid) Identification of microRNA biomarkers predictive of clinical outcomes in Aplastic Anaemia and Myelodysplastic Syndrome, Dr Lynette Chee, Melbourne Health.

DNA is the genetic material which provides the information that determines human characteristics, like the colour of our eyes and hair. RNA is another genetic material related to DNA and also influences how cells behave. A special type of RNA, called microRNAs, can change how cells behave. These microRNAs may be able to predict how likely patients will improve from treatment in blood cancers such as myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML), or even the disease progression of Aplastic Anaemia to MDS/AML. This progression from Aplastic Anaemia to MDS and AML is called clonal progression and has a poor chance of recovery.

In Dr Chee’s exploratory cohort, she discovered that Aplastic Anaemia patients who had progressed to MDS/AML had a similar microRNA expression profile to patients who develop MDS without prior Aplastic Anaemia. In addition, specific microRNAs at diagnosis and post-treatment were associated with disease progression and treatment response. These findings will now be validated in a larger cohort of patients.