Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease

Background: Hydroxyurea (HU) is highly effective treatment for sickle cell disease (SCD). While pediatric use of HU is accepted clinical practice, barriers to use may impede its potential benefit. Procedure: A survey of parents of children ages 5-17 years with SCD was performed across five institutions to assess factors associated with HU use. Results: Of the 173 parent responses, 65 (38%) had children currently taking HU. Among parents of children not taking HU, the most commonly cited reasons were that their hematology provider had not offered it, their child was not sufficiently symptomatic and concerns about potential side effects. Even parents of HU users reported widespread concern about effectiveness, long-term safety, and off-label use. In bivariate analyses, children's ages, parental demographics such as education level, or travel time to their hematology provider were not correlated with HU use. Bivariate analysis and multivariate logistic regression revealed three significant factors associated with current HU use: better parental knowledge about its major therapeutic effects (P<0.001), sickle genotype (P=0.005), and institution of clinical care (P=0.04). Conclusions: Pervasive concerns about HU safety exist, even among parents of current users. Varying knowledge among parents appears to be independent of their demographics, and is associated with HU use. Inter-institutional variability in parental knowledge and drug uptake highlights potentially potent site-specific influences on likelihood of HU use. Overall, these survey data underscore the need for strategies to bolster parental understanding about benefits of HU and address concerns about its safety.