At the 11th International Conference on Thalassaemia Hemoglobinopathies, and the 13th International Thalassaemia International Federation (TIF) Conference for Thalassaemia Patients and Parents being held in Singapore, Swiss drugmaker Novartis AG presented findings from different studies with their once-daily dissolvable tablet Exjade (generically deferasirox) as a treatment to improve quality of life and survival in patients with chronic anemia such as betathalassaemia, sickle cell disease (SCD) or myelodysplastic syndromes (MDS), and who through having to undergo repeated blood transfusions , suffer from iron build-up toxicity in their systems.

Approximately 78 percent of patients treated with Exjade had decreases in cardiac iron (measured by any change in T2, a magnetic resonance imaging measurement of myocardial iron) and 89 percent of patients had decreases in liver iron after six months of treatment (measured by decreases in liver iron concentration levels).

In addition, new long-term data from two other studies show that thalassaemia patients benefit from treatment with Exjade. At two years, in a study of 247 patients, Exjade significantly reduced iron toxicity in patients who had been previously treated with the alternative iron chelators desferrioxamine and/or deferiprone.

Results from a separate analysis of 680 heavily transfused thalassaemia patients support the efficacy and safety of Exjade seen over 3.5 years.

Exjade, administered as a dissolvable tablet in a drink, is the only once-daily oral drug that provides effective 24-hour iron chelation, granted an orphan drug status during development.
Regular blood transfusion therapy is essential for patients with thalassaemia, and may be necessary in diseases such as sickle cell disease (SCD) and myelodysplastic syndromes (MDS).

However, the consequence of blood transfusions is iron overload as the body has no physiological means to remove iron. This iron overload is life-threatening and causes significant tissue damage to organs such as the heart, liver and endocrine glands, and can ultimately lead to death.

Prior to the approval of Exjade, the most common way of performing iron chelation was with a treatment called desferrioxamine (DFO) and involved a painful nightly infusion by needle and pump that can last from eight to 12 hours every night for five to seven nights a week. As a result of the pain and inconvenience, many patients stopped or avoided iron chelation therapy, thus risking the toxic effects of iron overload.

It is estimated that as recently as 2000, around 50 percent of U.K. patients with beta-thalassaemia died before the age of 35, mainly because conventional iron chelation therapy is so burdensome.