Innovative Medication Delivery Program Improves Clinical Outcomes for Pediatric Patients with Sickle Cell Disease

Hydroxyurea is the gold standard preventive medication for children, adolescents, and adults with sickle cell disease (SCD). Despite expert recommendations that infants with HbSS or HbS/beta 0 thalassemia begin medication at nine months, traditional pharmacies do not manufacture or administer hydroxyurea in a liquid formulation. As a result, many families pay high prices for monthly delivery from a specialty or compounding pharmacy or forgo the treatment altogether.  

To improve access in pediatric patients with SCD, the pediatric care team at the Boston Medical Center of Excellence in Sickle Cell Disease created a program in 2016 to centralize the formulation and delivery of liquid hydroxyurea to families without cost. Data from a recently published article in the journal Pediatric Blood and Cancer indicates that consistent home delivery of liquid hydroxyurea improves access, reducing acute events, emergency department visits, and hospitalizations. 

Addressing disparities in medication access 

BMC’s Liquid Hydroxyurea Delivery Program addresses key barriers to medication access and health equity. SCD is an inherited blood disorder that predominately impacts people of African descent. Structural racism—from historical redlining to underfunded research—continues to impede improvement in outcomes for people living with the disease. At BMC, the majority of pediatric patients with SCD live in low-income, urban neighborhoods, where access to compounding or specialized pharmacies, like CVS, are miles away. As a result, the ability to be treated with this critical medication hinges on specialized delivery services, many of which are economically out of reach.  

“Hydroxyurea is a chemotherapy, and there continues to be mistrust around its toxicity, but there is also this practical issue that families can’t access it. You can’t take a medication if you don’t have it.”

Amy Sobota, MD, MPH, BMC pediatric hematologist

The urgency to close this equity gap is underscored in a recent study that found that the average life expectancy for publicly insured individuals with SCD is 52.6 years, well below the national average. The study also concluded that early and consistent access to hydroxyurea builds fetal hemoglobin in the body, protects healthy circulation, reduces mortality, and improves quality of life. 

“Hydroxyurea is vastly underutilized in people with sickle cell disease. There are a lot of factors that influence medication adherence. Hydroxyurea is a chemotherapy, and there continues to be mistrust around its toxicity, but there is also this practical issue that families can’t access it. You can’t take a medication if you don’t have it,” explains Amy Sobota, MD, MPH, BMC pediatric hematologist, study co-author.  

The prescription to patient pipeline 

BMC’s Center of Excellence in Sickle Cell Disease, the largest in New England, is uniquely poised to centralize liquid hydroxyurea’s prescription, formulation, and delivery. The hospital houses both a chemotherapy and a retail pharmacy, which work together to individualize liquid formulations of the drug and then deliver the prescriptions to households. Pediatric patients from ages six to nine months are eligible to receive the liquid form of the medication. 

Sobota and her colleagues conducted a retrospective chart review, analyzing data from 41 program participants from 2014 to 2020. The study showed patients had improved medication access after enrollment in the program, which led to a statistically significant decrease in hospitalizations and acute chest syndrome for participants one year after beginning the program. Although the study analyzes the data of a small cohort, findings indicate that prolonged adherence to hydroxyurea through direct delivery improves clinical outcomes.  

Sobota hopes BMC’s Liquid Hydroxyurea Delivery Program can serve as a model for other academic medical centers seeking to make the liquid formulation more readily available for their pediatric patients. Many institutions are equipped to compound the medication, and even offering the liquid formulation on-site would significantly improve patient access. In an era of historic treatment advancements for SCD, she argues implementation science promoting the uptake of evidence-based therapies can be impactful.  

“Our program is comprised of a lot of people’s hard work and dedication and the hospital’s financial support. It’s not rocket science. It is simply making the systems work for our patients, and it makes a huge difference,” she emphasizes.