Oral Chemotherapy Drug Shows Long-Term Benefits For Children With Sickle Cell Disease (Image Credits: iStock)
A new study has found that an oral drug called hydroxyurea can safely and effectively treat children with sickle cell disease. The research was published on April 17 in the medical journal Blood Advances. The study showed that children who regularly took hydroxyurea had fewer emergency room visits and spent less time in the hospital than those who didn’t take the drug.
Dr Paul George, lead researcher and a pediatric blood disease specialist at Emory University in Atlanta, said, “Hydroxyurea is the most effective medicine we have for sickle cell disease, and this study shows it keeps helping children over time.”
Sickle cell disease is a serious blood disorder that affects how red blood cells work. Instead of being round and smooth, the red blood cells in people with this disease become sickle-shaped and sticky. These cells can block blood flow, causing pain, infections, and damage to organs. In the US, about 100,000 people live with sickle cell disease. It mostly affects Black and Hispanic children.
Hydroxyurea is a once-daily pill that helps red blood cells stay healthy. It lowers the number of painful episodes and reduces the need for blood transfusions. The medicine can also improve anaemia and prevent clots in the lungs.
The US government recommends giving hydroxyurea to children with severe sickle cell disease starting at 9 to 12 months old. Although hydroxyurea was first used as a chemotherapy drug, it has been used to treat sickle cell disease for many years. Still, some families worry about the long-term safety, especially in children.
Dr Wilbur Lam, another researcher on the study, said, “Hydroxyurea has always raised some safety concerns because of its history as a chemotherapy drug. But this study should help families feel more confident that it’s safe and truly helps kids with sickle cell.”
The study followed more than 2,100 children with sickle cell disease who were treated at least three times between 2010 and 2021 at Children’s Hospital of Atlanta. Around 58 per cent of the children took hydroxyurea, with many staying on the drug for about five years.
The results showed that children who took hydroxyurea regularly had better health outcomes. They visited the hospital less often and were admitted for shorter stays, even after researchers looked at how serious their condition was and how closely they followed their treatment.
Dr George added, “The benefits of hydroxyurea were clear, but we also saw that children who didn’t take it regularly didn’t get the same improvements. That shows how important it is to stick with the medication.”
This study shows that hydroxyurea is a safe and helpful treatment for children with sickle cell disease. It can make a big difference in their health and quality of life, especially when taken as prescribed.
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