Hydroxyurea treatment toolkit aims to help SCD patients’ parents
Goal is shared decision-making between doctors, parents of young children
A toolkit may facilitate the active involvement of parents of young children with sickle cell disease (SCD) in shared decision-making regarding hydroxyurea treatment for their children, a study suggests.
However, the odds of these young patients being offered and prescribed hydroxyurea were higher among those who received usual care — which includes a clinician pocket guide with a summary of guidelines — than among those receiving a toolkit that includes materials for clinicians and caregivers, the data showed.
“Ultimately, the … toolkit may be most beneficial for clinics that do not routinely use a shared decision-making process for those considering hydroxyurea,” the researchers wrote, suggesting it might help “families experiencing high decisional uncertainty around” such treatment.
The study, “Engaging Parents of Children With Sickle Cell Disease in Shared Decision-Making for Hydroxyurea: The ENGAGE-HU Study,” was published in the journal Pediatric Blood & Cancer.
Investigating ways to best decide on use of hydroxyurea treatment
SCD is caused by mutations in the HBB gene that lead to the production of an abnormal version of hemoglobin known as hemoglobin S, which causes red blood cells to acquire a sickle-like shape. Hemoglobin is the protein responsible for carrying oxygen in red blood cells.
Sickled cells are more easily destroyed, and may clump inside blood vessels, blocking blood flow and triggering vaso-occlusive crises, known as VOCs.
Hydroxyurea is an oral medication used to reduce VOC frequency and the need for blood transfusions in people with SCD. According to the researchers, guidelines recommend that hydroxyurea be given to children as young as 9 months of age through shared decision-making between clinicians and caregivers — a collaborative process, as its name implies, in which parents actively participate in healthcare decisions.
To help in this process, researchers developed the Hydroxyurea Shared Decision-Making, or H-SDM, toolkit. It contains materials to improve clinicians’ communication skills and ability to implement shared decision-making, as well as parents’ decision aids, such as brochures and video narratives.
Pocket guides are widely used and typically rated as useful at the point of care by clinicians, but they do not address key barriers to the implementation of shared decision-making.
The efficacy of the toolkit was assessed in the observational ENGAGE-HU study (NCT03442114), conducted at 11 sites in the U.S. The study compared the H-SDM toolkit to a usual care method using the American Society of Hematology’s hydroxyurea clinician pocket guide. That strategy the guidelines into “who is eligible, what to do before starting, when hydroxyurea is started, and how to monitor it over time,” the investigators wrote.
According to the team, “pocket guides are widely used and typically rated as useful at the point of care by clinicians, but they do not address key barriers to the implementation of shared decision-making.”
In the study, sites began with the usual care condition before transitioning to the H-SDM toolkit in the years between 2018 and 2022. The team noted that the timing of the change and how long it took to recruit patients was not consistent across the sites.
COVID-19 pandemic found to have great impact on patient care
A total of 146 caregivers of young children with SCD, from birth to 5 years, were included in the study. Most caregivers were women (93.2%), and identified as the mother of the child (90.4%). A total of 89% of the caregivers were Black or African American individuals. Overall, 83 participants received usual care, while 63 received the toolkit.
Regarding the children, slightly more than three-quarters were in the age group from birth to 2 years, and more than 90% were Black or African American youth. Slightly more of the children were girls than boys. Most (94.5%) had HbSS, which occurs when both HBB gene copies are mutated to produce hemoglobin S.
The results showed no statistical differences in parent-reported scores of the Decisional Conflict Scale (DCS), which measures uncertainty when feeling uninformed about options, being unclear about personal values, or unsupported when having to make a choice.
“However, there was a clinically important difference between groups, in that the observed mean DCS total score for parents enrolled during the usual care period was 3.1 points higher,” the investigators wrote. In this tool, higher scores mean more uncertainty.
No significant differences were seen in caregivers’ perception of their active involvement in shared decision-making, or their knowledge of hydroxyurea.
A significantly higher proportion of participants enrolled during usual care were offered (80.7%) and prescribed hydroxyurea (48.2%) than those offered (58.7%) and prescribed (39.7%) the treatment using the toolkit.
“Contrary to hypotheses, the odds of being offered or prescribed hydroxyurea were significantly higher for parents enrolled during usual care,” the investigators wrote.
The researchers noted that most participants in usual care were enrolled before the COVID-19 pandemic and the majority of those in the toolkit group were recruited during the pandemic. As such, these results may limit definitive conclusions.
“With the onset of the pandemic, sites experienced significantly lower clinical volumes, practice changes (e.g., telehealth visits, less frequent visits), and high staff turnover, resulting in study implementation challenges,” the scientists wrote.
Given that, further research is needed, per the team.
“Additional information is needed to understand whether differences in hydroxyurea offers and prescriptions … are an artifact of the time of enrollment and/or other unknown pandemic-related factors,” the researchers wrote.
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