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Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease
Suzette O Oyeku1, M Catherine Driscoll, Hillel W Cohen
1Department of Pediatrics, Division of General Pediatrics, Children's Hospital at Montefiore, Albert Einstein College of Medicine of Yeshiva University, Bronx, New York 10467, USA. soyeku@montefiore.org
Insights
Parental knowledge and clinical site significantly impact hydroxyurea (HU) use for sickle cell disease (SCD) in children. Addressing safety concerns and improving understanding of HU benefits are crucial for effective treatment.
Area of Science:
- Pediatric Hematology
- Sickle Cell Disease Management
- Pharmacological Treatments
Background:
- Hydroxyurea (HU) is a highly effective treatment for sickle cell disease (SCD).
- Despite accepted clinical practice, barriers hinder pediatric HU utilization.
- Understanding these barriers is key to maximizing HU's benefits in children.
Purpose of the Study:
- To survey parents of children (5-17 years) with SCD across five institutions.
- To identify factors associated with hydroxyurea (HU) use in pediatric SCD patients.
Main Methods:
- Survey administered to parents of children with SCD.
- Data collected from 173 parent responses across five clinical sites.
- Bivariate and multivariate logistic regression analyses performed.
Main Results:
- 38% of children were currently taking HU.
- Key factors associated with HU use included parental knowledge of HU's effects, sickle genotype, and clinical site.
- Common reasons for non-use included lack of provider recommendation, perceived lack of symptoms, and side effect concerns.
Conclusions:
- Significant parental concerns regarding HU safety persist, even among current users.
- Parental knowledge about HU, independent of demographics, is linked to its use.
- Inter-institutional variability suggests site-specific influences on HU uptake, highlighting the need for improved parental education and safety counseling.
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.
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