Deferasirox (DFX) is an oral iron chelator with an established dose-dependent efficacy in transfusion-related iron overload. Whereas emerging long-term data confirm the safety of the drug, with transient moderate elevation of serum creatinine level, several authors have reported renal tubular dysfunction. The aim of this study was to evaluate tubular and glomerular function before and after the initiation of DFX therapy in a pediatric patient population.
Ten children (4 girls, mean age 12.4 ± 3.9 years) enrolled in a routine blood transfusion program were treated with 24.8 ± 9.6 mg/kg per day of DFX, and renal function was assessed before and 17.2 ± 8.9 months after the initiation of DFX therapy.
Prior to treatment with DFX, all patients had a normal glomerular function rate (GFR) (125 ± 15 ml/min per 1.73 m(2)) and normal tubular function. Following the initiation of DFX therapy, the GFR decreased by approximately 20 % with one patient with a GFR of <80 mL/min per 1.73 m(2) and seven patients with a GFR of <100 mL/min per 1.73 m(2). Two patients experienced a generalized proximal tubular dysfunction whereas nine patients presented at least one sign of proximal tubular dysfunction.
Renal toxicity is a frequent adverse event of DFX treatment, presenting as both glomerular and proximal dysfunction. A routine renal assessment is therefore required to prevent chronic kidney disease that may result from prolonged tubular injury.
[Show abstract][Hide abstract] ABSTRACT: The outlook for patients with TM and other transfusion dependent anemias has improved substantially with the availability of three iron chelating drugs and the use of T2* MRI to detect cardiac siderosis before cardiac symptoms develop. Combined therapy with DFO and DFP has proved particularly effective at treating a previously fatal iron induced cardiomyopathy. In the UK, infection rather than iron induced cardiomyopathy is now the main cause of mortality in TM (23). It seems likely that patient preference and compliance will result in the increased use of the oral chelators and corresponding reduced use of subcutaneous DFO. Randomised trials of oral chelators against DFO may become more difficult to perform because of patient preference. This will be particularly so if a third orally active iron chelator becomes clinically available. With each drug alone, however, a proportion of patients, perhaps 20%, will be inadequately chelated because of lack of efficacy or because the drug dosage has to be reduced or stopped because of side-effects. Switching chelators and combination therapy of the oral chelators is likely to increase in use so a randomized trial of the two oral chelators DFP and DFX in combination against alternative chelation regimens is urgently needed.
[Show abstract][Hide abstract] ABSTRACT: Deferasirox (DFRA) is a novel oral chelator agent for treatment of iron overload. Although well established in the treatment of β-thalassemia major (β-TM), it has not yet been fully investigated in patients with sickle cell disease. The aim of this report is to present the preliminary results of a pilot study assessing the effect of 4 years of DFRA treatment in six patients with sickle cell disease who are in need of recurrent transfusions. Our results show a significant reduction of ferritin levels and improvement of liver hemosiderosis, assessed by means of magnetic resonance imaging T2* (MRI T2*). None of the patients presented any serious adverse effects and the treatment was well tolerated. These results are in accordance with previous studies about the use of DFRA in sickle cell disease.
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