Taking Care of Youth With Sickle Cell Disease – Taking it to the Next Level

In a recently released article in Pediatrics, Dr. Brousseau and colleagues (10.1542/peds.2018-3285) examine key questions about hydroyurea use among children with sickle cell anemia and other sickle cell genotypes. It has been clearly shown in controlled trials at academic centers that use of hydroxyurea decreases episodes of pain (vaso-occlusive crises) and rates of hospitalizations and Emergency Department visits for children and adults with sickle cell disease.  This data is impressive and solid enough that since 2014, the National Heart Lung and Blood Institute (NHLBI) has recommended offering hydroxyurea to all children 9 months of age or older who have sickle cell anemia regardless of severity. What’s not yet known is whether these potential benefits and guidelines have translated to the community and beyond single academic sites.

To answer this question, the authors used a multi-state Medicaid claims data base called the Truven/IBM-Watson Health MarketScan Medicaid data base, and examined claims over the period of 2009-2015 for youth ages 1-19 years old with sickle cell disease. Since 80% of children with sickle cell disease receive Medicaid insurance, this should capture most eligible individuals; over 5,000 individuals were included for each year. The approach to the analysis is well explained and easy to follow, and the main outcome was patient receipt of hydroxyurea, as measured by a filled pharmacy prescription claim. Percent of days on hydroxyurea was calculated, with “coverage” defined as 66% of days on medication.

I’m not giving away much to share that the good news was limited to an increase in utilization over time, and the bad news was that just 1 in 4 youth with sickle cell anemia (28.2% in 2015) received any one month of hydroxyurea, and even fewer achieved the relatively low bar set for continuous coverage. While the study results are highly disappointing in terms of the main study question, the opportunities for improving care are enormous.  What is a reasonable plan moving forward? Clearly those who provide direct care to youth with sickle cell diseases are in a good position to respond. But this seems a perfect question for research methodologies that include patients, not just as participants, but as researchers. What are the barriers to hydroxyurea use from the patient and family perspective? What would make hydroxyurea use acceptable and even desirable among those with sickle cell disease? Community based participatory research (CBPR) is a relevant model, in which individuals from the community partner with academic researchers.¹ In CBPR community and neighborhood partners work hand in hand with scientists to conduct the research – this includes developing relevant research questions and using participatory methods that don’t lose sight of the family’s and child’s perspectives and needs, such as focus groups and semi-structured interviews led by peers. We can do a lot better for youth with sickle cell disease, and the challenge Brousseau and colleagues’ article sets out for us is a first step.

Reference

1. Belone L, Lucero JE, Duran B, et al. Community-Based Participatory Research Conceptual Model: Community Partner Consultation and Face Validity. Qual Health Res. 2016;26(1):117–135. doi:10.1177/1049732314557084