Sickle cell disease is a genetic disorder. An increasing number of people show secondary iron overload due to repeated red blood cell transfusions. Since the human body has no means of actively getting rid of excessive iron, drug treatment (iron chelators) is needed. Several years ago, a new oral iron chelator, deferasirox, was introduced. However, it is not known whether deferasirox offers advantages compared to deferoxamine or deferiprone with regard to effectiveness and safety.
One randomised study with 203 participants comparing deferasirox to deferoxamine was identified. The study lasted for 12 months. Iron removal measured by the surrogate marker serum ferritin was similar for both drugs. Both drugs worked equally well in reducing liver iron concentration, which was measured by a biomagnetometer (SQUID). No data on patient-important outcomes such as death or end-organ damage were collected.
The safety of deferasirox was acceptable; the main side effects were increases of creatine, abdominal pain and diarrhoea. Rare side effects (less than 5%) such as eye or hearing problems were not evaluated. Patient satisfaction and compliance with therapy was significantly better with deferasirox.
Only one study could be included in this review. This study suggests that deferasirox works as well as deferoxamine and has an acceptable safety record, but there are not enough data looking at long-term safety. We should wait for the results of the ongoing trial before deciding whether deferasirox could be added as an alternative to the first-line option of deferoxamine. Currently, its use seems to be warranted as second-line therapy for people with sickle cell disease who cannot tolerate or comply with deferoxamine. Future studies need to examine the long-term effectiveness and safety of deferasirox as well as the cost-effectiveness; in addition, patient-important outcomes should be addressed.