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

Abstract

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.

Adherence to Hydroxyurea Therapy in Patients with Sickle Cell Disease at King Khalid University Hospital in Riyadh

“…The use of hydroxyurea in children with SCD is significantly associated with: (1) sickle genotype, (2) better parental knowledge about the major therapeutic effects of the drug, and (3) institution of this therapy by hematologists and provision of the drug to symptomatic patients [5].…”

“…Hydroxyurea is the most commonly used targeted therapy that increases Hb F production in patients with SCD [2,4,5]. Indications for using hydroxyurea in patients with SCD include: (1) frequent painful crises (2) recurrent chest syndrome or other severe venoocclusive events, (3) severe, symptomatic and chronic anemia, and (4) reduction of pain, dactylitis, acute chest syndrome and anemia in children and adolescents [6,7].…”

Quality of Life, Clinical Effectiveness, and Satisfaction in Pediatric and Young Adult Patients with Sickle Cell Disease Receiving Hydroxyurea Therapy

“…These findings are not surprising in the context of studies on poor compliance of patients with SCD to HU therapy. [6][7][8]12,13,15,24,25,[27][28][29] With the increasing number of clinical studies that support HU use in the patients with SCD, a properly feasible strategy will likely be needed to effective use of HU in clinical practice.…”

“…After taking signed written informed consent from the patients and/or their parents, the following data were obtained through face-to-face survey methods: demographic and clinical characteristics of pediatric and young adult patients (using Demographic Data Collection Form and Case Report Form), health status of pediatric patients (using Child Health Questionnaire-Parent Form 50; CHQ-PF50), quality of life of young adult patients (using Life Quality Survey Short Form-36; SF-36), and effectiveness and acceptance of HU therapy in pediatric and young adult patients (using Case Report Form and HU Satisfaction Survey). 6,[8][9][10][11][12][13][14][15] S St ta at ti is st ti ic ca al l a an na al ly ys si is s: : Qualitative variables were expressed as number and percent. Quantitative variables regarding health status of pediatric patients and compliance to HU therapy in pediatric and young adult patients were described in the form of mean±standard deviation (SD), median, and range.…”

Hydroxyurea Is Associated With Lower Costs of Care of Young Children With Sickle Cell Anemia

“…2,3 Promoting the appropriate use of hydroxyurea is a promising way to improve health outcomes among patients with sickle cell disease, but barriers to its widespread use need to be addressed. 4,5 The BABY HUG study was an institutional review board-approved National Institutes of Health-funded multicenter randomized double-blinded trial of hydroxyurea in very young children with sickle cell anemia (HbSS or HbSb°t halassemia), 9 to 18 months old at enrollment, who were not selected for clinical severity. 6 Subjects received oral hydroxyurea at a dose of 20 mg/ kg/day or placebo for 2 years.…”