oral deferasirox effective iron chelator for transfusional iron overload?

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Inpharma 1387 - 17 May 2003 Oral deferasirox * may be an effective iron chelator in patients with thalassaemia and transfusional iron overload, reports a multinational group of researchers. Their study involved 24 patients aged 16 years with thalassaemia and transfusional iron overload who had been treated with deferoxamine 20 mg/kg/day for 4 weeks. The patients were randomised to receive deferasirox [ICL 670] 10 mg/kg/day (n = 5), 20 mg/kg/day (6) or 40 mg/kg/day (7), or placebo, for 12 days. Deferasirox administration significantly increased the unsaturated iron-binding capacity in serum, relative to placebo. Furthermore, net iron excretion was dose-dependently increased by deferasirox. Most patients treated with deferasirox 20 mg/kg/day appeared to achieve, or be very close to achieving, iron balance. In five out of six of these patients, net iron excretion was similar to the amount of iron they would be likely to receive from their transfusion. Notably, deferasirox was well tolerated in this study. * Novartis; phase III for the treatment of iron overload Nisbet-Brown E, et al. Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet 361: 1597-1602, 10 May 2003 800925880 1 Inpharma 17 May 2003 No. 1387 1173-8324/10/1387-0001/$14.95 Adis © 2010 Springer International Publishing AG. All rights reserved

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Inpharma 1387 - 17 May 2003

■ Oral deferasirox* may be an effective iron chelatorin patients with thalassaemia and transfusional ironoverload, reports a multinational group of researchers.Their study involved 24 patients aged ≥ 16 years withthalassaemia and transfusional iron overload who hadbeen treated with deferoxamine 20 mg/kg/day for ≥ 4weeks. The patients were randomised to receivedeferasirox [ICL 670] 10 mg/kg/day (n = 5), 20mg/kg/day (6) or 40 mg/kg/day (7), or placebo, for 12days. Deferasirox administration significantly increasedthe unsaturated iron-binding capacity in serum,relative to placebo. Furthermore, net iron excretionwas dose-dependently increased by deferasirox. Mostpatients treated with deferasirox 20 mg/kg/dayappeared to achieve, or be very close to achieving,iron balance. In five out of six of these patients, netiron excretion was similar to the amount of iron theywould be likely to receive from their transfusion.Notably, deferasirox was well tolerated in this study.* Novartis; phase III for the treatment of iron overload

Nisbet-Brown E, et al. Effectiveness and safety of ICL670 in iron-loadedpatients with thalassaemia: a randomised, double-blind, placebo-controlled,dose-escalation trial. Lancet 361: 1597-1602, 10 May 2003 800925880

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Inpharma 17 May 2003 No. 13871173-8324/10/1387-0001/$14.95 Adis © 2010 Springer International Publishing AG. All rights reserved