Treatment of Sickle Cell Disease: There’s More Than Just Pain Medications

Sickle cell disease (SCD) affects nearly 100,000 Americans currently. This chronic debilitating disease of the red blood cells, can cause a multitude of complications – pain, stroke, kidney disease, etc. For many years the only thing available to patients was the treatment of pain episodes with medications. Unfortunately, this only treats a symptom and not the disease itself. However, now we have hydroxyurea as a treatment option.

Hydroxyurea (sometimes referred to as HU) is a medication taken by mouth that can help children and adults with sickle cell disease. Currently, it is the only U.S. Food and Drug Administration (FDA) approved medication to treat SCD in adults. Multiple studies have shown that it is safe and effective in children as well. Hydroxyurea helps red blood cells stay round and flexible instead of the sickle or banana shape seen in SCD. These round red blood cells are able to flow more easily through the blood vessels, reducing SCD complications. Specifically, hydroxyurea has been shown to reduce the number of pain crises, reduce the number of acute chest syndrome (lung complication) events, reduce the need for blood transfusions and reduce number of hospitalizations. Additionally, most patients become less anemic while taking hydroxyurea. Hydroxyurea was first used as a treatment for cancer, but we give a much lower dose to children with SCD. At these reduced doses, we do not see the usual side effects seen with cancer treatment (loss of hair, vomiting or loss of appetite). Many people have been concerned about the risk of developing cancer while taking hydroxyurea. An increased risk of cancer has not been seen in children or adults with SCD taking hydroxyurea. Hydroxyurea is very safe when monitored appropriately. It can reduce the number of neutrophils (a white blood cell that fights infection) and platelets (a blood cell that stops bleeding). If this is seen, the dose of hydroxyurea can be reduced and treatment can continue.

In 2014 the National Heart, Lung and Blood Institute (NHLBI, part of the National Institutes of Health) released guidelines for the management of SCD. In these guidelines, it is recommended that all children with severe SCD over the age of 9 months should be offered hydroxyurea therapy regardless if they have had SCD complications. The goal now is to prevent as many complications as possible. Here at Texas Children's Hospital we have more than 300 children with SCD receiving hydroxyurea therapy. Although hydroxyurea is not a cure, it has made a significant impact on this chronic condition. Today, children with SCD are living longer, healthier lives because of hydroxyurea. For more information on sickle cell disease, please view our SCD Handbook. To learn more about Texas Children’s Sickle Cell Program, please visit here.