Finding a new and better way to treat acute promyelocytic leukaemia (APL)

Acute promyelocytic leukaemia (APL) is a rare and aggressive type of blood cancer that affects a specific type of white blood cell called promyelocytes. In the UK, 200–300 people are diagnosed with APL each year. Treatment often involves taking tablets that help the leukaemia cells grow into normal, healthy blood cells and infusions which are given through a drip into a vein to help kill the leukaemia cells and stop them from growing. Although the majority of people can be cured, treatment is very intense and time consuming, often involving many hospital visits for many months to have blood tests, heart checks, and for long two-hour appointments to receive an infusion. There is a need for better treatments that place less burden on people and their families and can allow them to lead a more normal life.

In this project, Professor Knapper and his team are testing a new treatment that involves taking as a tablet at home instead of travelling to hospital for appointments to receive infusions. They believe this tablet treatment will work just as well and be an easier treatment option with less side effects. Researchers will study people who are in remission from APL after initial hospital treatment. Half of them will have to travel to hospital to receive infusions and the other half will take this new tablet treatment at home for six months. The researchers will compare both treatments to see how well the leukaemia is controlled. They will monitor side effects and assess overall quality of life and treatment costs for the NHS.

If the new home tablet treatment is as effective as the hospital treatment, this could transform treatment for people with APL. It could provide a new way of treating the disease where people with APL do not have to frequently travel to hospital for appointments, experience fewer side effects, and are able to return to their normal lives more quickly. This would provide a much better and more convenient way for managing and treating the disease as families would face less financial and emotional stress, whilst saving the NHS money.