Researchers at King’s College Hospital have been awarded a grant for a study aimed at understanding why some aplastic anaemia patients later develop a type of leukaemia.
The grant, from the Aplastic Anaemia Trust, will be used to pay for genetic sequencing on bone marrow samples from 200 patients from across Europe, to look for new abnormalities in genes that may be an early signal for acute leukaemia or an early type of pre-leukaemia called myelodysplastic syndrome (MDS).
Professor Judith Marsh, a haematology consultant at King’s College Hospital, will lead the research with Professor Ghulam Mufti.
King’s is the leading national centre for aplastic anaemia, a rare disease of the bone marrow stem cells in which not enough blood cells are produced. At ten years after treatment, around 15% of patients with aplastic anaemia may go on to develop MDS or acute leukaemia.
Professor Marsh said: “Aplastic anaemia patients have a lack of stem cells in the bone marrow, which means a lack of white blood cells, which are needed to protect against infections, a lack of red blood cells, which are needed to carry oxygen around the body, and a lack of platelets, which are needed to stop bleeding. It’s a very serious disease with a high risk of severe infection and serious bleeding, although many patients will get better with treatment. However, we want to understand the genetic reasons why some patients with aplastic anaemia later develop MDS.”“This research will help us understand more about these new mutations within the DNA of bone marrow stem cells and how they develop in patients receiving drug treatment for aplastic anaemia,” she said. “We are using the latest DNA technology, which will provide us with unique information that will help us pick up on earlier signs that a patient may go on to later develop MDS. Earlier detection will allow us to make better treatment decisions at an early stage of their illness, which will mean better outcomes for patients.”
For information about the Aplastic Anaemia Trust, which supports aplastic anaemia patients and raising money for research, go to: www.theaat.org.uk.