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KMID : 0869620110280030262
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Journal of Korean Society of Hospital Pharmacists
2011 Volume.28 No. 3 p.262 ~ p.273
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Evaluation of the efficacy and safety of deferasirox in pediatric patients who changed iron chelating agent from deferoxamine to deferasirox
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Kim Sung-Hwan
Choi Mi-Hye
Jung Sun-Hoi
Son In-Ja
Lee Hye-Sook
Ahn Hyo-Seop
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Abstract
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Deferoxamine has for decades been the standard for iron chelation therapy, but requires administration as a slow continuous subcutaneous or intravenous infusions five to seven times per week, potentially leading to poor compliance, reduced effectiveness. Deferasirox was developed in response to the need for an oral iron chelating agent. Its convenient, once-daily, oral administration schedule offers the potential to improve compliance. However, long-term efficacy and safety remain to be established. This study evaluated the efficacy and safety of deferasirox in pediatric patients who were switched off deferoxamine iron chelating therapy and introduced to deferasirox. It was conducted by method of retrospective chart review. Patients were divided into two groups. Group A(n=22) was constituted of patients who were converted from deferoxamine to deferasirox for their iron chelating therapy. Group B(n=24) was constituted of patients starting iron chelating therapy directly on deferasirox and had never changed. We evaluated the efficacy and the safety of deferasirox through intra-group and inter-group comparisons. The patients in group A, those who were administered deferoxamine, showed a trend of decrease in ferritin levels one month after administration(-222±117.16 ng/ml, p=0.845). In contrast, when the same patients changedtheir chelating agent to deferasirox, there was a tendency for ferritin levels to increase(+40.67±965.5 ng/ml, p=0.384). However, the ferritin level drop was not statistically different between the drugs (p=0.422). The use of deferasirox in group B decreased ferritin levels more than any other group, but was not statistically significant(-701.5±1687.5 ng/ml, p=0.053). Adverse events were more frequently observed with deferasirox than with deferoxamine in group A(59.1% vs 27.3%). The most common adverse events with deferasirox were gastrointestinal problems and LFT abnormality. On the other hand, skin problems were the most frequent adverse reactions to deferoxamine. Deferasirox showed similar efficacy to deferoxamine in terms of decreasing ferritin levels in pediatric patients. It might be suggested to initiate deferasirox as early as possible due to its efficacy profile and convenient administration regimen. A higher adverse event rate, however, may necessitate for careful monitoring of the patient’s clinical status while using deferasirox.
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KEYWORD
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iron chelation, deferasirox, deferoxamine, ferritin, pediatrics
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