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Research Identifies Need for Improved Understanding of Sickle Cell Disease in Adulthood
Date:12/8/2007

ent was more effective than the "gold standard" treatment of deferiprone alone, with milder and reversible side effects, such as reversible leukocytopenia (low number of white blood cells) and hypertransaminasemia, which was defined as the increase of serum alanine transaminase levels up to two times in comparison to normal values. In addition, after one year of treatment, the deferiprone-deferoxamine group showed greater efficacy in terms of serum ferritin level reduction than the deferoxiamine-only arm of a previous randomized clinical trial in which a comparable cohort of patients were studied.

-- Deferasirox, an oral iron chelator, works as well as deferoxamine, which requires subcutaneous injection, among sickle cell patients; possible implications for improved patient compliance, reducing risk of organ damage [Abstract #3395]

Elliott Vichinsky, MD, Children's Hospital and Research Center, Oakland, CA

This four-year extension trial among 185 patients with sickle cell disease is a follow-up study to a one-year trial that showed that deferasirox and deferoxamine were equally effective at reducing liver iron concentration. In this four-year extension study, all patients stayed on deferasirox (n=132) or were switched to the therapy (n=53). The trial showed that deferasirox demonstrated dose-dependent efficacy in patients with sickle cell disease, with a manageable tolerability profile and no new adverse events reported over a median of 2.1 years of treatment. Because deferoxamine must be delivered via a subcutaneous injection, patients must typically visit their doctor's office or local clinic. Deferasirox is taken orally and therefore may support improved patient compliance, a key factor in avoiding complications of iron overload, including kidney damage.

In addition to sickle cell disease and thalassemia, press briefings will take place at the annual meeting focusing on leukemias, hematologic malignancies, blood clotting and blee
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SOURCE American Society of Hematology
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