Efforts to ensure SCD teens take hydroxyurea may boost life quality
Analysis: Intervention includes extra support from community health workers
An intervention designed to improve adherence to hydroxyurea — by providing extra support from community health workers (CHWs) and daily text reminders — may enhance quality of life for adolescents with sickle cell disease (SCD), though the benefits may fade without continued support.
The findings came from an analysis of secondary outcomes of the HABIT efficacy trial (NCT03462511), which initially tested whether the intervention could help teens build a daily medication habit.
“HABIT participants in the intervention group … reported that they enjoyed working with CHWs and learned new information about both hydroxyurea and SCD,” researchers wrote. “Further research directed to improve the sustainability of interventions is needed to improve hydroxyurea adherence and [quality of life] to reduce health disparities for youth with SCD.”
The study, “HABIT Efficacy Trial Intervention Improves Elements of General and Disease-Specific Quality of Life in Youth With Sickle Cell Disease,” was published in Pediatric Blood & Cancer and funded by the National Institute of Nursing Research, part of the National Institutes of Health.
‘Adolescents often struggle to take hydroxyurea consistently’
SCD is caused by mutations that result in the production of a defective form of adult hemoglobin, the protein that enables red blood cells to carry oxygen through the body. The faulty protein makes red blood cells stiff and sickle-shaped, and they become prone to die early and block small blood vessels. This leads to anemia — low red blood cell counts and/or low hemoglobin levels — as well as painful vaso-occlusive crises and other SCD symptoms.
In the U.S., SCD affects about 100,000 people, 40% of whom are children, according to the researchers.
Hydroxyurea, sold under the brand names Siklos or Xromi, is an oral medication approved to reduce the frequency of pain crises and the need for blood transfusions in people with the most severe form of SCD. Guidelines recommend offering it to children starting as early as 9 months old. Still, many adolescents struggle to take hydroxyurea consistently.
“When an adolescent begins treatment with hydroxyurea, there are many benefits,” Arlene Smaldone, PhD, a professor at Columbia School of Nursing who co-led the study, said in a university press release. “There’s less organ damage, their pain is reduced, and they have less hospitalizations. Treatment may also improve their quality of life by improving their physical and mental health and their ability to keep up with friends, school, and other obligations. However, because the disease can be physically and mentally draining, adolescents often struggle to take hydroxyurea consistently.”
Health-related quality of life scores showed improvement at 9 months
To explore whether improving adherence could also enhance quality of life, researchers in the U.S. analyzed secondary outcomes from the HABIT trial. The study recruited 50 English- and Spanish-speaking adolescents, ages 10 to 18, and their caregivers across sites in New York City and Philadelphia from Sept. 2018 through Dec. 2020.
Of these, 24 were randomly assigned to the intervention group and 26 to usual care. Those in the intervention group met with a CHW five times over three months to review educational handouts with their caregiver, attend one clinic visit, and receive guidance on hydroxyurea use. They also received daily text reminders for the following three months to reinforce their treatment routine.
Both groups received standard clinical care and educational materials about SCD and hydroxyurea throughout the trial, but the control group did not receive CHW support or text reminders.
Data on generic and disease-specific health-related quality of life (HRQoL) were collected through questionnaires at the start of the trial and again at 4, 9, and 12 months, with 45 participants completing the trial.
The teens had a mean age of 13.3 years, with no major demographic or lab differences between the two groups. In the year before the trial, 60% had visited the emergency department and 48% had been hospitalized for SCD-related care. Most caregivers were employed full-time (64%), and half were single parents.
CHWs can be a cost-effective adjunct to care, especially among low-resourced, underserved communities.
At the start, mean generic HRQoL scores were 71.2 out of 100, with about 30% of participants in the intermediate or impaired range. On the disease-specific HRQoL, the average overall score was 62.7, with 22% of participants in the “good” range. There were no significant HRQoL differences between groups at study entry.
At nine months, HRQoL scores improved in both groups relative to their initial scores.
Compared with the control group, however, teens in the intervention group showed significantly higher and clinically meaningful improvements in generic HRQoL total scores, along with higher scores in emotional and social functioning — reflecting better mood and stronger ability to engage with friends and activities. They also reported greater improvement in the disease-specific treatment subscale, reflecting more confidence in managing hydroxyurea.
By 12 months, both groups continued to show improvements compared with their initial scores. Still, differences between them were no longer significant, suggesting that while effective in the short term, the intervention’s effects were not sustained, according to the researchers.
“CHWs can be a cost-effective adjunct to care, especially among low-resourced, underserved communities,” the researchers wrote. “Future research is needed to test the effect, sustainability, and cost-effectiveness of CHW interventions for people living with SCD.”
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