Ponatinib for chronic myeloid leukaemia in paediatric patients

Chronic myeloid leukaemia (CML) is a rare type of cancer, especially in children. CML is characterised by the uncontrolled production of white blood cells called myeloid cells; this means cells divide but do not mature. These immature dividing cells, known as blast cells, fill up the bone marrow and stop it making healthy blood cells. This puts the child at increased risk of infection. The overproduction of white blood cells also interferes with the production of healthy red blood cells and platelets, leading to symptoms such as anaemia and bruising. Children with chronic phase CML may have no symptoms but blast cells are present in the blood and bone marrow. The chronic phase can last several years before transforming to the second, accelerated phase. Currently, CML in children cannot be cured by any type of chemotherapy, although some medications can eliminate easily detectable signs of leukaemia from the blood, prolonging the chronic phase of the disease.