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Azacitidine is a drug given to be people with MDS but some people have to stop this treatment due to side effects. Dr Sternberg wants to find out whether combining azacitidine with another drug can stop this from happening.

The context

Myelodysplastic syndrome (MDS) is a serious disorder of blood cell production that causes fatigue, infection and bleeding. Certain types of MDS ('high risk' MDS) can change in character and become cancerous - resulting in acute myeloid leukaemia (AML). Azacitidine is a type of chemotherapy drug that doctors use to treat people with AML and MDS. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML and MDS, important genes that regulate cell growth and division can be ‘switched-off’ when chemical tags stick to DNA inside cells and this can cause cells to grow out of control. Azacitidine is a drug that tries to reverse this by removing the chemical tags, switching on the gene again.

The project

A recent trial has shown that azacitidine improves survival in people with high risk MDS. But many people in this trial had to have their azacitidine reduced or stopped because of side effects. Researchers want to see a drug called eltrombopag helps reverses side effects, such as bleeding, during azacitidine treatment. Although eltrombopag has been used for other disorders, doctors do not know what the correct dose eltrombopag should be for people with high risk MDS patients being treated with azacitidine.

The aims

  • Find out the highest, safest dose of eltrombopag to give with azacitidine
  • Find out what the side effects are of having these two drugs together are
  • See if the treatment helps people with MDS that has come back after a stem cell transplant