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Updated: Nov 4, 2025
A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Engaging Caregivers and Providers of Children With Sickle Cell Anemia in Shared Decision Making for Hydroxyurea:
Anna M Hood1, Heather Strong2, Cara Nwankwo3
1Developmental Neurosciences, Institute of Child Health, University College London, London, United Kingdom.
Insights
This study compares a standard clinician pocket guide to a new hydroxyurea decision toolkit for parents of children with sickle cell anemia. The goal is to improve shared decision-making and treatment uptake for this serious genetic blood disorder.
Area of Science:
- Pediatric Hematology
- Genetics
- Public Health
Background:
- Sickle cell anemia (SCA) is a severe genetic blood disorder in children, associated with significant health risks and high healthcare utilization.
- Hydroxyurea is the primary disease-modifying therapy for SCA, recommended for children as young as 9 months.
- Suboptimal uptake of hydroxyurea stems from caregiver information gaps and concerns, and a lack of clinician training in shared decision-making (SDM).
Purpose of the Study:
- To determine the most effective method for facilitating shared decision-making (SDM) discussions about hydroxyurea between parents and clinicians.
- To improve caregiver knowledge and reduce decisional uncertainty regarding hydroxyurea treatment for young children with SCA.
- To increase the uptake of hydroxyurea in children aged 0-5 years with SCA.
Main Methods:
- A comparative study design was used to evaluate two guideline dissemination methods: a standard clinician pocket guide versus a specialized hydroxyurea SDM toolkit (H-SDM toolkit).
- Primary outcomes included caregiver-reported decisional uncertainty and knowledge about hydroxyurea.
- Secondary outcomes assessed the prescription rates of hydroxyurea and subsequent health outcomes in children aged 0-5 years with SCA.
Main Results:
- The study enrolled 120 caregiver participants between November 2017 and February 2021.
- Ethical approval was obtained from the Cincinnati Children's Hospital Medical Center Ethics Committee in November 2017.
- Data collection and analysis are ongoing to determine the effectiveness of the H-SDM toolkit compared to usual care.
Conclusions:
- The study aims to enhance the quality of care for children with SCA by improving SDM.
- Multicomponent dissemination strategies, developed with stakeholder input, are crucial for addressing barriers to high-quality SCA care.
- Informed and shared decision-making empowers caregivers to choose the best health options for their children with SCA.
Background:
Sickle cell anemia (SCA) is a genetic blood disorder that puts children at a risk of serious medical complications, early morbidity and mortality, and high health care utilization. Until recently, hydroxyurea was the only disease-modifying treatment for this life-threatening disease and has remained the only option for children younger than 5 years. Evidence-based guidelines recommend using a shared decision-making (SDM) approach for offering hydroxyurea to children with SCA (HbSS or HbS/β0 thalassemia) aged as early as 9 months. However, the 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 tools, especially those that provide medical evidence and consider caregivers' preferences and values, to facilitate a shared discussion with caregivers.
Objective:
The aim of this study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that considers medical evidence and parent values and preferences).
Methods:
We designed our study to compare the effectiveness of two methods for disseminating hydroxyurea guidelines to facilitate SDM: a clinician pocket guide (ie, usual care) and a clinician hydroxyurea SDM 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) who were offered and prescribed hydroxyurea and the resultant health outcomes.
Results:
The Ethics Committee of the Cincinnati Children's Hospital Medical Center approved this study in November 2017. As of February 2021, we have enrolled 120 caregiver participants.
Conclusions:
The long-term objective of this study 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 can make informed and shared decisions about their health.
Trial Registration:
ClinicalTrials.gov NCT03442114; https://clinicaltrials.gov/ct2/show/NCT03442114.
International Registered Report Identifier (Irrid):
DERR1-10.2196/27650.
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