Deferasirox Reduces Iron Burden in Patients With Beta-Thalassaemia: Presented at ASH
By Betty S. Riggs
NEW ORLEANS -- December 9, 2009 -- Long-term deferasirox treatment for up to 5 years significantly decreased the iron burden in patients aged 2 years and older with beta-thalassaemia, according to a study presented here on December 7 at the American Society of Hematology (ASH) 51st Annual Meeting and Exposition.
Maria Domenica Cappellini, MD, University of Milan and Fondazione IRCCS Ospedale Maggiore Policlinico Mangiagalli e Regina Elena, Milan, Italy, and colleagues evaluated the efficacy and safety of deferasirox over 5 years in patients who had previously participated in a large, 1-year, phase 3, randomised, clinical trial comparing deferasirox and deferoxamine. Patients who completed the 1-year study were permitted to enter a 4-year extension study.
Those patients who had received deferasirox in the 1-year study continued this treatment in the extension (n = 296), whereas those who had received deferoxamine crossed over to deferasirox (crossover cohort; n = 259). Dose adjustments were allowed based on serum ferritin (SF) levels and safety markers. The primary efficacy endpoint was the change in liver iron concentration (LIC) as assessed by liver biopsy or superconducting quantum interference device.
Patients who received at least 1 dose of study drug were included in the analysis. A total of 181 patients (61.1%) in the deferasirox cohort and 190 patients (73.4%) in the crossover cohort completed the extension.
At start of deferasirox, mean LIC was 14.0 +- 9.8 and 10.4 +- 7.6 mg Fe/g dry weight (dw), and median SF was 2,211 and 1,758 ng/mL in deferasirox and crossover cohorts, respectively.
There were no significant differences between groups in transfusion requirements.
In patients who received at least 5 years of deferasirox and at least 4 years in the crossover group, mean absolute change in LIC was -5.3 +- 10.1 mg Fe/g dw (P < .001) and -2.4 +- 7.6 mg Fe/g dw (P < .001), respectively. In these groups, median absolute change in SF was -775 ng/mL (range -10,164 to 2,572; P < .001) and -371 ng/mL (range -4,498 to 2,636; P < .001), respectively.
The most common reasons for discontinuation were consent withdrawal (n = 62) and adverse events (AEs; n = 43). The most common AEs leading to discontinuation were increased alanine aminotransferase, increased transaminases, and glycosuria.
During the extension study, there were 3 deaths in the deferasirox cohort and 2 in the crossover cohort. None of the deaths were considered to be related to study drug.
The most common drug-related AEs in the deferasirox and crossover cohorts were increased blood creatinine, nausea, vomiting, diarrhoea, rash, abdominal pain, and upper abdominal pain. The frequency of drug-related AEs decreased from year to year.
Funding for this study was provided by Novartis Pharmaceuticals Corporation.
[Presentation title: Efficacy and Safety of Deferasirox (Exjade) in Patients With Beta-Thalassemia Major Treated for Up to 5 Years. Abstract 4063]
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