Safety and efficacy of deferasirox in multitransfused Indian children with β-thalassaemia major.

Abstract

Background: Iron chelation is an important component of management of transfusion-dependent patients with thalassaemia major. Deferasirox is a relatively new oral iron chelator and experience of its use in children is limited.

Aim: To report experience with deferasirox in north Indian children with β-thalassaemia major.

Methods: This prospective study included 40 patients with transfusion-dependent β-thalassaemia major. The patients were receiving deferiprone alone (37 patients) or deferiprone and desferrioxamine combination (three patients) before commencing deferasirox. Patients were clinically monitored every month. Information on side-effects including gastro-intestinal symptoms, skin rash or discoloration, jaundice and complaints regarding vision and hearing were obtained from patient records. Laboratory investigations included complete blood count and renal and liver function tests estimated at baseline and then every month. Serum ferritin level was estimated at baseline and then every 3 months. The initial dose of deferasirox was 20 mg/kg/body weight and was increased to 25 mg/kg if serum ferritin remained unchanged or increased 3 months after deferasirox therapy.

Results: Therapy with deferasirox in 40 children was well tolerated. Gastro-intestinal symptoms were the most common side-effects. Nausea, vomiting and abdominal pain were observed in 25%, 20% and 15% patients, respectively. Skin rashes were seen in 5% cases. We observed greyish-brown pigmentation of the skin in four (10%) children which has not been described before. A non-progressive rise in serum creatinine was observed in 16 (40%) patients. In the majority, however, serum creatinine remained within the normal range. Leucopenia, neutropenia and thrombocytopenia were not observed. None of the side-effects necessitated cessation of the drug therapy. Serum ferritin levels fell in 24 of 32 patients (75%) who received deferasirox for over 1 year from a mean (SD) 6323·37 (2756·5) μg/L to 5458·91 (2301·2) μg/L (p<0·05).

Conclusions: Therapy with deferasirox is safe in paediatric patients with thalassaemia major. However, they should be carefully monitored for side-effects.