Researchers investigated the use of hydroxyurea, a drug approved to treat sickle cell anemia in adults, in young children in New York State who are receiving this treatment. The findings reveal that pediatric use of hydroxyurea is widespread and increasing, but that incomplete adherence may limit the treatment’s full disease-modifying effects.
The study, “Hydroxyurea Use in Young Children With Sickle Cell Anemia in New York State,” was published in the American Journal of Preventive Medicine.
Hydroxyurea (HU) was approved by the U.S. Food and Drug Administration (FDA) for adult patients with sickle cell anemia (SCA) in 1998. Although the drug’s precise mechanism of action is unknown, its efficacy is attributed to its ability to boost levels of fetal hemoglobin, which reduces the concentration of Hb S, the abnormal haemoglobin that causes sickle cell disease.
Clinical studies have shown that HU use in adolescents, children, and infants produces similar benefits as those observed in adults, without harmful effects on growth and development. In the study, researchers aimed to characterize the young SCA patient population receiving HU treatment in New York. The research focused on the extent of use, changes in treatment patterns over time, and by identifying regional or treatment center differences that may constitute barriers to use or cause different adoption rates.
Using Medicaid data, the researchers identified 273 children, born between 2006 and 2009. Results found that about 25% had filled at least one HU prescription by age 5, and about 40% had filled one by the study’s end. Adequate treatment adherence (80% coverage), however, was only seen in about one-third of the children initiated on HU therapy.
Researchers highlight that some regional and treatment center differences, unexplained but possibly due to factors such as parental acceptance or inadequate center resources, may limit the success of this therapy. For this reason, further studies are needed to address its unanswered questions. “Another important follow-up to this study will be to assess outcomes of the increased HU use for children, statewide, to determine if the established therapeutic benefits of HU treatment are realized in community health settings and if the higher initiation rates observed in some regions benefit more individuals,” the researchers concluded.