Thalassaemias are among the most common inherited disorders in the world and can result in severe anaemia3

Create infographic with copy below

The term “thalassaemia” generally refers to a family of disorders secondary to combinations of over 200 known mutations in the
β-globin gene (beta thalassaemia) or to a smaller number of mutations in the α-globin gene (alfa thalassaemia).17

Beta thalassaemia major is a clinical diagnosis referring to a patient who has a severe form of the disease and requires chronic transfusions early in life.3

Create infographic with copy below

Chronic transfusion allows to survive in patients with beta thalassaemia major (Cooley's Anaemia- Mediterranean Anaemia). Because the synthesis of beta chains is almost completely inhibited in thalassaemia major, a severe anaemia begins at about 3 to 6 months of age.18

Detoxification of excess iron is the most important function of chelation therapy. Thalassaemia treatment with iron chelation should start early to prevent iron overload.3

As iron continues to build up in the body, complications happen more often.

About 70%

of deaths in thalassaemia major patients are due to heart complications and disease.6

Despite improvements in care, iron overload in the heart is the leading cause of death among people with thalassaemia major6

% of people with transfusion dependent thalassaemia chart % of people with transfusion dependent thalassaemia chart % of people with transfusion dependent thalassaemia chart

Adapted from Borgna-Pignatti C. et al. Haematologica 2004. Table 1, Causes of death for the entire population of patients and for those born after 1970; p. 1189

Iron overload affects your kidneys and liver

Careful monitoring of liver and kidney function is important to prevent further damage to the organs. You can ask to your doctor for kidneys or liver complications and how to take care of their health.3

Iron chelation needs to be adjusted regularly or even switched depending on iron level, weight, and tolerance to side effects3

There are a number of parameters that need to be monitored during the iron chelation therapy, such as:4,15,19
Serum ferritin Liver iron concentration: Cardiac MRI T2*:
ferritin plays an important role in the storage of intracellular iron. Serum ferritin is a useful monitoring tool for iron overload in thalassaemia major15 one of the liver functions is to store iron for when the body needs it. Liver is also a primary target for iron toxicity and its functions need to be monitored regularly19 when the storage capacity of ferritin is exceeded, iron is deposited in the myocardium with the risk of cardiac failure. Cardiovascular magnetic resonance imaging is a useful noninvasive tool for evaluating the amount of iron in the heart4,19

Lowering serum ferritin and liver iron concentration are not enough. Reducing cardiac iron is the priority in thalassaemia.6
Appropriate chelation treatment should maintain iron levels within recommended ranges.19

In case of need to report an adverse drug reaction, please refer to your physician, asking him to fill in and submit the relevant case report to the concerned Health Authority, according to the Pharmacovigilance requirements in force in your country. Nevertheless, please be kindly reminded that each patient can directly report any such cases directly to the national reporting system.