Hydroxyurea

Dr Rana’s Drugs for Sickle Cell Disease Series Hydroxyurea It was last week that I saw Sidra, an 11-year-old patient with sickle cell disease. Sidra’s face was beaming with smiles as she walked into our office with her mother. Her mother showed me her grades, which were all As. Sidra is in the fifth grade and at the top of her class. She was born with sickle cell disease and I met mother and father soon after she was born. I was to provide education regarding sickle cell disease. Her mother works for a non-profit organization and her father is a teacher in the public-school system. The diagnosis was extremely hard for mother and she blamed herself. Sidra’s parents had talked about prenatal diagnosis. Every time we discussed sickle cell disease and its complications, her mother would start sobbing and tears would roll down her face—she felt that she had done this to Sidra. We started talking about treatment at around 6 months of age. At 9 months, after several discussions, Sidra’s parents agreed to start Hydroxyurea. Mother would empty the capsule into a small cup of juice and give it to Sidra. We saw Sidra every 2–3 months for blood work. At this visit, there were several third and fourth year medical students with me as we talked with Sidra’s family and examined her. Sidra has never had a painful crisis or acute chest syndrome. She is a bubbly, happy child no different than any other child in her class. Our medical students who had only seen adult patients admitted to the wards for pain and other complications were in awe and amazed. We joked about her dance class and gymnastics. Sidra is not sure what she wants to do when she grows up, but going to medical school is a definite possibility. Sidra’s story is not unique. Most of the children we started on Hydroxyurea and who take it regularly are not much different from Sidra. This has been a big success for all of us who care for children with sickle cell disease. I saw my first child with sickle cell disease in 1977 at a hospital in Brooklyn, New York. This child had meningitis, and despite the best of care, died within a day. I was at the University of Rochester for 2 years as a fellow in Pediatric Hematology. As a trainee, I saw a few patients with sickle cell disease who had developed stroke. My research in Rochester focused on immune deficiency in sickle cell disease. At that time there was really no treatment for sickle cell disease. At that time, I knew I wanted to take care of children with sickle cell disease and find better treatments for it. I joined the Howard University faculty in 1980. I was lucky to work with mentors like Dr Oswaldo Castro and others who were engaged in research for sickle cell disease. I saw many children with sickle cell disease and severe complications. I remember Damien, a young teenager who suffered from severe pain crises every month. He would get admitted to the hospital, receive Demerol injections for a week, and then go home. He ended up missing almost 2 weeks of school with every crisis. In the 1990s when Dr. Castro and others completed the study of Hydroxyurea in adults, I offered this treatment to Damien and his family. He started taking the medication. I saw him in clinic for renewal of the medication a couple more times and then did not hear from him. He had not been admitted, so I got his phone number from the records department and called him. He told me that he was doing well and had not had any pain in 2 years. He told me that he had been calling my office to get the medication renewed. I asked him why he had not come to the clinic. He said, “Why should I come to the clinic when I have not been sick?” A big milestone in my professional journey was the opportunity to develop and participate in the Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) along with other researchers. BABY HUG studied Hydroxyurea in children as young as 9 months of age with sickle cell disease. The study took a long time to get started and the first publication was not until 12 years later. The study clearly showed that Hydroxyurea was safe in young children and it decreased the risk of pain crises and other complications. Other studies of Hydroxyurea in children and adults have shown that it decreases the chances of developing stroke and other complications. It also improves the chances of living longer with sickle cell disease. The chance of dying from sickle cell disease complications is less than half when people are taking it versus those who don’t take it. So, what is the problem? Most people with sickle cell disease are not taking Hydroxyurea. Misinformation abounds. Many physicians do not prescribe it or do not provide adequate information. To dispel a few myths:

1. Hydroxyurea does not cause cancer in people with sickle cell disease.
2. Hydroxyurea does not cause birth defects if women take it during pregnancy.
3. Hydroxyurea decreases the chances of pain crises, acute chest syndrome, priapism, needing blood transfusion, and death tremendously.
4. Hydroxyurea is safe.
5. In order to get maximum benefit, you must take it every day, 7 days a week, 365 days per year.
6. People who do not take it are not going to benefit from it.
7. All insurance companies pay for Hydroxyurea.

Please feel free to email me if there are questions. Sohail R. Rana, MD Director of Community Outreach and Education Center for Sickle Cell Disease Howard University Email: [email protected]