Health and Me

Ishita Roy

Chemo Drug Could Be Effective For Kids With Sickle Cell Disease

chemo drug could benefit kids with sickle cell disease

A recent study published in <em>Blood Advanced </em>has reaffirmed the long-term safety effectiveness of hydroxyurea. This is an oral <span>chemotherapy</span> drug, which is used to manage sickle disease (SCD) in children. The findings have given a new hope and reassurance to families and caregivers who are trying to wade the waters of this chronic condition. 

<h4>What Is <span rel="noindex">Sickle Cell Disease</span>?</h4>

As per the National Heart, Lung, and Blood Institute, sickle cell disease, also known as sickle cell anemia, is a group of inherited disorders that affect hemoglobin. Hemoglobin is the major protein that carries oxygen in the red blood cells. Usually, red blood cells are disc-shaped and flexible, so they can easily move around the blood vessels. However, in this condition, the red blood cells are misshaped, like a crescent, or "sickle", which is where the disease gets its name from. This happens due to a gene mutation that affects hemoglobin molecule. When red blood cells sickle, they do not bend or move easily and thus can block blood flow to the rest of the body. 

People with sickle cell disease could experience onset of strong episodes of pain, also called pain crises. 

<h4>How Will Hydroxyurea Change The Conditions Of Those With Sickle Cell Disease?</h4>

Hydroxyurea, originally developed as a chemotherapy drug, is now a cornerstone in the treatment of SCD. It is taken orally once a day and works by helping red blood cells maintain a more normal, flexible, and round shape. This reduces the likelihood of blockages in the blood vessels.

According to St. Jude Children’s Research Hospital, the drug also helps reduce pain crises, lowers the need for blood transfusions, improves anemia, and cuts down the risk of potentially fatal complications like blood clots in the lungs. Given these benefits, federal guidelines recommend offering hydroxyurea to children with severe SCD starting as early as 9 to 12 months of age.

The study was conducted by the researchers at Emory University School of Medicine. The study analyzed 2,147 children with sickle cell disease and tracked them between 2010 to 2021. These kids were being treated at the Children's Hospital of Atlanta. Among them, 58% had been prescribed hydroxyurea. The average treatment duration was five years, with some children age 8 or older on continuous therapy throughout the study period.

The study found that children who took hydroxyurea had fewer emergency room visits. They also spent fewer days in the hospital as compared to those who did not take the drug. Furthermore, the benefits remained even when researchers adjusted for disease severity and medication adherence. 

Dr. Paul George, lead author of the study, emphasized, “Our results reinforce that hydroxyurea...continues to have really important benefits over time for pediatric patients.”

Despite its proven effectiveness, hydroxyurea has faced skepticism due to its origins as a chemotherapy drug. “There have always been some lingering fears about its safety and efficacy, especially for children,” explained Dr. Wilbur Lam, co-author of the study.

However, the long-term data from this study helps to dispel these concerns. Lam added, “This study can provide some reassurance...that this therapy...continues to be a safe option with a true benefit outside of a controlled setting.”

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A recent study published in Blood Advanced has reaffirmed the long-term safety effectiveness of hydroxyurea. This is an oral chemotherapy drug, which is used to manage sickle disease (SCD) in children. The findings have given a new hope and reassurance to families and caregivers who are trying to wade the waters of this chronic condition. What Is Sickle Cell Disease?As per the National Heart, Lung, and Blood Institute, sickle cell disease, also known as sickle cell anemia, is a group of inherited disorders that affect hemoglobin. Hemoglobin is the major protein that carries oxygen in the red blood cells. Usually, red blood cells are disc-shaped and flexible, so they can easily move around the blood vessels. However, in this condition, the red blood cells are misshaped, like a crescent, or "sickle", which is where the disease gets its name from. This happens due to a gene mutation that affects hemoglobin molecule. When red blood cells sickle, they do not bend or move easily and thus can block blood flow to the rest of the body. People with sickle cell disease could experience onset of strong episodes of pain, also called pain crises. How Will Hydroxyurea Change The Conditions Of Those With Sickle Cell Disease?Hydroxyurea, originally developed as a chemotherapy drug, is now a cornerstone in the treatment of SCD. It is taken orally once a day and works by helping red blood cells maintain a more normal, flexible, and round shape. This reduces the likelihood of blockages in the blood vessels.According to St. Jude Children’s Research Hospital, the drug also helps reduce pain crises, lowers the need for blood transfusions, improves anemia, and cuts down the risk of potentially fatal complications like blood clots in the lungs. Given these benefits, federal guidelines recommend offering hydroxyurea to children with severe SCD starting as early as 9 to 12 months of age.What Did The Study Find?The study was conducted by the researchers at Emory University School of Medicine. The study analyzed 2,147 children with sickle cell disease and tracked them between 2010 to 2021. These kids were being treated at the Children's Hospital of Atlanta. Among them, 58% had been prescribed hydroxyurea. The average treatment duration was five years, with some children age 8 or older on continuous therapy throughout the study period.What Were The Key Findings?The study found that children who took hydroxyurea had fewer emergency room visits. They also spent fewer days in the hospital as compared to those who did not take the drug. Furthermore, the benefits remained even when researchers adjusted for disease severity and medication adherence. Dr. Paul George, lead author of the study, emphasized, “Our results reinforce that hydroxyurea...continues to have really important benefits over time for pediatric patients.”Addressing Safety ConcernsDespite its proven effectiveness, hydroxyurea has faced skepticism due to its origins as a chemotherapy drug. “There have always been some lingering fears about its safety and efficacy, especially for children,” explained Dr. Wilbur Lam, co-author of the study.However, the long-term data from this study helps to dispel these concerns. Lam added, “This study can provide some reassurance...that this therapy...continues to be a safe option with a true benefit outside of a controlled setting.”