Acute myeloid leukaemia: new treatment targets molecular targeting
The disease has an incidence of about 3-4 cases per 100,000 people per year: in Italy there are 2,000 new cases every year with an average age of 68 years
by Adriano Venditti*
Key points
Acute myeloid leukaemia (AML) is an aggressive form of blood cancer characterised by rapid progression and not infrequent recurrence. It develops in myeloid cells, immature cells in the bone marrow that normally become white blood cells, red blood cells or platelets. In people with AML, these cells are overproduced and do not turn into mature blood cells, leading to anaemia (fatigue, paleness, difficulty breathing and tachycardia), a susceptibility to infection and haemorrhaging, particularly of the skin and mucous membranes.
Approximately 3,000 new cases are registered each year in Italy
AML has an incidence of about 3-5 cases per 100,000 people per year; in Italy there are about 3,000 new cases each year. However, the risk of developing the disease varies with age and in most cases it affects older people, with an average age at diagnosis of 68 years.
Five-year survival from diagnosis of AML is less than 20 per cent and varies depending on individual factors such as the patient's age, general health, and response to treatment.
There are different types of AML that we are now able to distinguish and identify thanks to rather sophisticated molecular biology technologies that can give us a very precise picture of the genetic characteristics of leukaemic cells. FLT3 mutations are among the most common. Approximately 80% of FLT3 mutations are FLT3-ITD (internal tandem duplication) mutations that promote neoplastic cancer growth and contribute to a particularly poor prognosis, including an increased risk of recurrence, a poor response rate to salvage therapy and a shorter survival expectancy compared to AML patients without mutation.
How the treatment scenario is changing
Today, the therapeutic scenario of AML is changing significantly. In particular, the possibility of identifying the genetic and molecular characteristics of this disease has made it possible to develop targeted therapies, to understand the mechanisms of AML and the prognosis of patients. In recent years, new drugs have arrived that are able to interact with the genetic alterations of leukaemic cells or with certain metabolic processes. These drugs can be used alone or in combination with chemotherapy.
