No abstract

Thirty years ago, the first major federal legislation concerning sickle cell disease treatment was passed, resulting in the development of comprehensive sickle cell centers. We are now at another watershed moment in the treatment of this illness with the passage in October 2004 of the Sickle Cell Treatment Act, designed to substantially expand specialized sickle cell treatment programs. This legislation offers a remarkable opportunity to significantly improve health outcomes for individuals with sickle cell disease if it is implemented with a specific focus on the distinct but related issues of equity and quality. Despite major advances in sickle cell disease treatment that have occurred over the past 3 decades, important gaps exist both in the equity of government and private philanthropic support for research and in the uniform provision of high quality clinical care. This article assesses the current gaps in funding support and in the implementation of improvements in clinical care in order to suggest strategies for making optimal use of the opportunity that the new legislation presents to improve the health of all individuals affected by this 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.