Abstract
Background: Guidelines recommend that hydroxyurea be offered to children with sickle cell disease (SCD) as early as 9 months of age using shared decision-making. To help clinicians implement shared decision-making with parents, we developed the Hydroxyurea Shared Decision-Making (H-SDM) toolkit. We evaluated its effectiveness on parent decisional uncertainty, perceptions of shared decision-making, hydroxyurea knowledge, and the likelihood of being offered and prescribed hydroxyurea. Procedure: Sites began in the usual care condition (clinician pocket guide) before crossing over to the H-SDM toolkit condition between 2018 and 2022. Caregivers of children with SCD (birth to 5 years) eligible for hydroxyurea completed assessments at baseline, immediately after discussing hydroxyurea with their clinician, and 3–7 months later. Results: Participants included 176 caregivers (93.2% female, 89% Black); most toolkit participants were enrolled during the pandemic (n = 81). There were no statistically significant differences between conditions on parent decisional uncertainty, perceptions of shared decision-making, or hydroxyurea knowledge (p-values >0.05). However, there was a clinically important difference in certainty, with higher decisional uncertainty in the usual care group. A greater proportion of participants enrolled during usual care were offered (80.7%) and prescribed hydroxyurea (48.2%), compared to 58.7% offered and 39.7% prescribed during the toolkit condition (p-values ≤0.01). Conclusions: Findings suggest the toolkit may help parents feel more confident in deciding about hydroxyurea. Given the significant impacts of the COVID-19 pandemic on study implementation, the impact on hydroxyurea uptake requires additional exploration. Ultimately, the H-SDM toolkit may be most beneficial for clinics that do not routinely use a shared decision-making process for those considering hydroxyurea.
| Original language | English (US) |
|---|---|
| Article number | e31639 |
| Journal | Pediatric Blood and Cancer |
| Volume | 72 |
| Issue number | 5 |
| DOIs | |
| State | Published - May 2025 |
Funding
We sincerely thank the patients living with SCD and their caregivers who participated in this study, members of the Stakeholder Advisory Council, medical and graduate students including Joanna Rebitski and Emmanuel Gray, and clinical research coordinators and data analysts Jennifer Allen, Catharine Whitacre, and Stacey Gomes. We would also like to acknowledge James Peugh, PhD, William Brinkman, MD, Amber M. Yates, MD, Connie M. Piccone, MD, Lynne Neumayr, MD, Emily R. Meier, MD, Sohail Rana, MD, Emmanuel J. Volanakis, MD, Maria Britto, MD, and Russell E. Ware, MD for their contributions to the study. The research reported in this paper was funded through a Patient-Centered Outcomes Research Institute (PCORI) Award (CDR-1609-36055). The statements presented in this paper are solely the responsibility of the authors and do not necessarily represent the views of PCORI, its Board of Governors, or the Methodology Committee. We sincerely thank the patients living with SCD and their caregivers who participated in this study, members of the Stakeholder Advisory Council, medical and graduate students including Joanna Rebitski and Emmanuel Gray, and clinical research coordinators and data analysts Jennifer Allen, Catharine Whitacre, and Stacey Gomes. We would also like to acknowledge James Peugh, PhD, William Brinkman, MD, Amber M. Yates, MD, Connie M. Piccone, MD, Lynne Neumayr, MD, Emily R. Meier, MD, Sohail Rana, MD, Emmanuel J. Volanakis, MD, Maria Britto, MD, and Russell E. Ware, MD for their contributions to the study. The research reported in this paper was funded through a Patient\u2010Centered Outcomes Research Institute (PCORI) Award (CDR\u20101609\u201036055). The statements presented in this paper are solely the responsibility of the authors and do not necessarily represent the views of PCORI, its Board of Governors, or the Methodology Committee.
Keywords
- hydroxyurea
- pediatric
- shared decision-making
- sickle cell
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Hematology
- Oncology
