University of Glasgow researchers, led by Dr Karen Keeshan, have uncovered the “distinct biology” of paediatric acute myeloid leukaemia (AML), including a genetic signature separate from that of the adult disease.
During the study, published last week in the journal Nature Communications, the team developed a model of AML in mice, which showed that the age of the cell that becomes diseased is a key factor in the disease that follows. This included unique blood cell features in paediatric AML and distinct changes to the bone marrow environment.
Analysing the mouse and human AML samples, they also revealed a gene signature unique to paediatric AML, which included genes involved in immune cell interactions.
Professor Brenda Gibson, of the Royal Hospital for Children, Glasgow, said: “Despite giving children with AML the most intensive chemotherapy, a third of children still relapse; and when children with AML relapse, it is often fatal. In addition to a significant relapse rate, chemotherapy comes with a mortality rate from the treatment alone and also carries the risk of serious side effects which may affect children for the rest of their life.
“Dr Keeshan’s work is important because, by identifying differences in the biology of child and adult AML, we can hopefully identify targeted treatments that may not just improve outcomes, but importantly may be associated with fewer side effects than intensive chemotherapy. As a result the survivors may have a better quality of life, which is very important for children with AML.”
Dr Keeshan said: “Historically children with AML have received treatment based on adult practice, and we need better treatments specifically for children with AML.
“Our work has identified a distinct paediatric gene profile and paediatric gene targets; and by identifying targetable features of the disease in children, we can pursue new and better strategies to treat paediatric AML.”
Source: Chaudhury, S., O'Connor, C., Cañete, A., Bittencourt-Silvestre, J., Sarrou, E., Prendergast, Á., Choi, J., Johnston, P., Wells, C.A., Gibson, B., Keeshan, K. (2018) “Age-specific biological and molecular profiling distinguishes paediatric from adult acute myeloid leukaemias”, Nature Communications, available at doi: 10.1038/s41467-018-07584-1
If you are interested in Paediatric Haematology you may wish to join our Paediatric Specialist Interest Group, and attend our meetings. Paediatric Symposium is on 14th February and we have Paediatric Sessions at our ASM on 1st April.