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| |  Deferasirox Monotherapy Improves Cardiac Iron Burden in Patients With Beta-Thalassaemia: Presented at ASH By Betty S. Riggs NEW ORLEANS -- December 10, 2009 -- Treatment with deferasirox for 18 months improves cardiac iron burden in heavily transfused patients with beta-thalassaemia, according to a study presented here on December 7 at the American Society of Hematology (ASH) 51st Annual Meeting and Exposition. John Wood, MD, Children’s Hospital of Los Angeles, Los Angeles, California, and colleagues evaluated the effect of deferasirox on cardiac iron content in 28 patients aged older than 10 years with beta-thalassaemia receiving chronic transfusion therapy (8/year) with a lifetime minimum of 100 packed red blood cell transfusions. Deferasirox was administered at 30 mg/kg/day at baseline, and the dose could be increased to 40 mg/kg/day in patients with serum ferritin (SF) >=1,000 mcg/L above baseline at 2 consecutive visits or with <25% improvement in cardiac T2* compared with baseline values, if liver iron concentration (LIC) >=3 mg Fe/g dry weight. Following core study completion (18 months), patients could continue treatment for an additional 6 months if their 18-month cardiac T2* was <20 ms and if they demonstrated >=25% improvement in cardiac T2* or LIC from baseline. SF was assessed monthly and LIC, cardiac T2*, and left ventricular ejection fraction were assessed by magnetic resonance imaging every 6 months. Serum creatinine, biochemical, and haematological status were also monitored. At the time of analysis, 22 patients had 18-month evaluations and 9 had 24-month evaluations. There were 6 discontinuations and 2 of these patients died after discontinuing. At 18 months, 10 out of 22 patients were receiving 40 mg/kg/day. There were improvements in T2* in 13 of 27 patients. Responders had significantly lower hepatic (P < .001), ferritin (P = .03), and cardiac (P = .05) iron burdens than the 14 nonresponders. Responding patients had progressively improved cardiac and liver iron burden throughout the study (2.4% and 3% per month, respectively), demonstrating reciprocal changes in cardiac T2* and LIC. Nonresponsive patients did not have lower LIC at any time point, and cardiac T2* steadily worsened (decreasing 0.84% per month). There was statistically significant improvement in T2* (P = .01) and LIC (P = .002) in the first 6 months; however, at 18 months the results were not significant. Baseline LIC was a powerful predictor of response, but initial T2* did not predict therapeutic response. At 24 months, 7 of 9 patients were on 40 mg/kg/day and 8 of 9 (89%) had increased cardiac T2*, with a mean improvement of 2.7% per month. The most common drug-related adverse events (AEs) were nausea (n = 7), diarrhoea (n = 5), and rash (n = 5). There was 1 serious AE, involving hospitalisation for abdominal pain and vomiting; this patient completed the study. Funding for this study was provided by Novartis Pharmaceuticals Corp. [Presentation title: Initial Liver Iron Predicts Cardiac Chelation Efficacy of Deferasirox (Exjade) Monotherapy in Chronically Transfused Beta-Thalassemia (Beta-Thal) Patients: 18- and 24-Month Data. Abstract 4069]
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