Background Iron chelation is an important component of management of transfusion-dependent patients with thalassaemia major. Deferasirox is a relatively new oral iron chelator (US FDA approved in 2005) with the limited experience in children.

Aims To present our experience with deferasirox in patients with thalassaemia major (TM) in the context of: effects on serum ferritin level in chronically transfused patients with thalassaemia major, side effects and patients tolerance to the drug and effects on serum creatinin and liver transaminases.

Methods Four patients with TM with mean age of 3,1 years (range 2,5–3,5) were included in the study. Mean follow up was 37 months (range 29–42). The disease was diagnosed in early childhood (during the first year of life) with the following signs and symptoms: extreme pallor, jaundice, failure to thrive, poor feeding, irritability, decreased activity and hepatosplenomegaly. Regular blood transfusions were applied to treat chronic hemolytic anaemia.

Results The mean serum ferritin (SF) at diagnosis was 471,3 ± 284,4 (range 155–706), and at the start of the treatment with deferasirox 6281 ± 9183,9 (range 767–20000). The number of blood transfusions before the treatment was around 28,3 ± 15,5 units (range 17–46), or about 679,3 ± 608,14 (range 220–1369) ml/kg body weight. Deferasirox was given seven days a week at a dose of 20 mg/kg body weight. The primary outcome variable was SF level at the start and at the end of the study. Echocardiography was made in all patients and it was normal. MRI-T2* could not be performed because there was no specific software. The level of SF at the end of the study period was 1862 ± 1312,15 (range 637–3710). Patients were monitored for hepatic and renal toxicity, visual or auditory changes and development of new symptoms. Adverse events were very mild gastrointestinal symptoms in 1 patient and no adverse events in the remaining 3 patients. Elevation of serum creatinin or hepatic transaminases was not observed in any subject. One patient interrupted the therapy as there was a marked fall in SF < 500 ng/L at the end of the therapy. The treatment was well tolerated; suspension of therapy was not required owing to toxicity.

Summary/conclusion The results suggest that deferasirox is effective in lowering iron burden, it is well tolerated and has a low potential for toxicity. Long term therapy will be needed to asses the benefits on iron balance and organ damage in chronically transfused patients with thalassaemia major.