JMIR Publications

ABSTRACT

Introduction: Sickle cell anemia (SCA) is a genetic blood disorder that places children at risk for serious medical complications, early morbidity and mortality, and high healthcare utilization. Until recently, hydroxyurea was the only disease-modifying treatment for this life-threatening disease and has remained the only option for children less than five years of age. Evidence-based guidelines recommend using a shared decision-making approach to offer hydroxyurea to children with SCA (HbSS or HbS/β0thalassemia) as early as nine months of age. However, uptake remains suboptimal, likely because caregivers lack information about hydroxyurea and have concerns about its safety and potential long-term side effects. Moreover, clinicians do not routinely receive training or the tools to facilitate a shared discussion with caregivers, especially one that provides medical evidence and considers caregivers’ preferences and values. Methods and Analysis: We designed our study to compare the effectiveness of two methods for disseminating hydroxyurea guidelines to facilitate shared decision-making: 1) a clinician pocket guide (i.e., usual care), and 2) a clinician hydroxyurea shared decision-making toolkit (H-SDM toolkit). Our primary outcomes are caregiver reports of decisional uncertainty and knowledge of hydroxyurea. The study also assesses the number of children (aged 0 – 5 years) offered and prescribed hydroxyurea and resultant health outcomes. The long-term objective of this research is to improve the quality of care for children with SCA. Using multicomponent dissemination methods developed in partnership with key stakeholders and designed to address barriers to high-quality care, caregivers of patients with SCA have the opportunity to make informed and shared decisions about their health. Trial registration: ClinicalTrials.gov: NCT03442114. Registered February 28th, 2018.