Real-World Database Study Affirms Long-Term Benefit of Hydroxyurea for Anemia in Pediatric SCD

Heme Today spoke with Paul George, MD, PhD, of Emory University School of Medicine in Atlanta, Georgia, and the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta. He is the first author of a database study, published in Blood Advances, to evaluate the long-term efficacy of hydroxyurea for sickle cell anemia in pediatric patients.

Dr. George explained that the in-practice experience of clinicians who manage sickle cell disease (SCD) in pediatric patients, as well as the results from decades of randomized clinical trials, affirms that hydroxyurea is very effective at preventing clinical complications of SCD for at least the first few years of treatment. Clinicians’ experience has been that this effectiveness continues over the long term, but there is a lack of clinical trial data to confirm this sense, and trials have tended to only monitor for 1 to 3 years.

“The issue with that is that hydroxyurea is currently recommended as a lifelong medication for kids with a severe form of sickle cell disease. So, of course, it would be important to know for this lifelong medication how the impact of the medication progresses over time,” Dr. George elaborated.

The study conducted by Dr. George and colleagues addressed this need for data about hydroxyurea’s efficacy over the long term in pediatric SCD. It examined information from the Aflac Cancer and Blood Disorders Center’s clinical database of real-world longitudinal data from pediatric patients with SCD. This included data from more than 2,000 pediatric patients on hydroxyurea treatment, some for as long as 10 years.

“Importantly, what we were seeing is that that benefit in keeping kids out of the emergency room, keeping kids out of the hospital, persists after years of use for hydroxyurea. That was our big overall takeaway from the study,” Dr. George specified.

Dr. George noted that clinicians’ experience using hydroxyurea to prevent anemia in pediatric SCD has been that the drug’s preventive effect seems to wane the longer a patient is on the therapy. However, the study, which examined patients’ hemoglobin values among its secondary endpoints, pinpointed that it was patients who were not adherent to their hydroxyurea regimen who were lacking hematologic benefit after years of use. Dr. George views this finding as reinforcing that patients and their managing clinicians must not become complacent in care.

“If there’s a kid who’s been on hydroxyurea for 4 years, 5 years, 6 years, and especially if they’re entering adolescence, just make sure you continue at visits to stress the importance of taking this daily medication every day because that’s where you’re going to see the biggest benefit,” Dr. George clarified.

Although his team’s study used real-world data, Dr. George acknowledged as a limitation that the electronic medical records they consulted included few patient-reported outcomes.

“We know for many diseases, but especially sickle cell disease, a lot of the complications of the disease are not seen in the hospital. They’re not seen in the clinic. They’re actually what the kid or young adult is experiencing at home. Incorporating that sort of information into routine clinical collection I think could be helpful moving forward,” Dr. George concluded.