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Chemo Drug Brings New Hope For Children

Jason Phillippe by Jason Phillippe
April 21, 2025
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Chemo Drug Brings New Hope For Children

Photo by National Cancer Institute on Unsplash

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The fight against sickle cell is getting better.

A recent study underscores the long-term safety and effectiveness of hydroxyurea, an oral chemotherapy medication, in managing sickle cell disease (SCD) in children. Published in Blood Advances, the study reveals that children who took hydroxyurea experienced fewer emergency room visits and hospital admissions compared to those who did not use the medication. These results strengthen the evidence that hydroxyurea is the most effective and accessible treatment available for pediatric SCD.

Sickle cell disease is a hereditary blood disorder that causes red blood cells to become misshapen and rigid. These abnormal cells can obstruct blood flow, leading to pain, infections, and organ damage. Hydroxyurea helps by enhancing the shape and flexibility of red blood cells, preventing these complications. The medication also reduces the frequency of painful episodes and the need for blood transfusions while improving anemia and circulation.

Although hydroxyurea was originally used as a chemotherapy drug, concerns about its safety have been raised. However, this study—conducted with over 2,100 children treated at a children’s hospital in Atlanta from 2010 to 2021—provides robust evidence supporting its safety. Approximately 58% of participants used hydroxyurea for an average of five years, and the findings consistently showed long-term benefits, particularly for those who adhered to the treatment regimen.

The study also highlighted that improvements in anemia, such as better hemoglobin levels, were only evident in children who followed the treatment closely. This emphasizes the importance of consistent medication adherence to achieve the full benefits. While hydroxyurea is recommended for children with severe SCD starting at around 9 to 12 months of age, treatment plans should be individualized based on the severity of the disease and the child’s specific health needs.

In conclusion, the study provides strong evidence that hydroxyurea is a safe and effective treatment for managing pediatric sickle cell disease, even outside clinical trial settings. It supports continued efforts to increase its use and encourage adherence to improve long-term health outcomes for children living with this challenging condition.

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