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Key Study Findings: Is hydroxyurea treatment associated with lower medical costs for young children with sickle cell anemia?

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The journal Pediatrics has published the first study to look at the costs and health outcomes (cost-effectiveness) of hydroxyurea (HU) treatment in young children with sickle cell disease (SCD). A previous scientific article from the BABY HUG study found that 1 year old children with sickle cell anemia randomly selected to receive HU had fewer hospital stays between ages 1 and 3 years than children who did not receive HU. In this new study, the cost of providing the HU treatment and follow-up doctor visits is weighed against the reduced number of hospital stays and the associated cost-offset (money saved). BABY HUG investigators and CDC researchers found that HU not only provides good value for the money (is cost-effective) but also reduces total health care costs (is cost-saving). You can read the article’s abstract here.

Main Findings from this Study

  • The total cost of health care for young children 1-3 years of age with sickle cell anemia who received HU treatment was reduced compared to those who did not receive this type of treatment.
    • Children who received HU treatment had nearly a third (30%) fewer hospital stays between ages 1 and 3 years.
    • Average Medicaid reimbursements for hospital stays for US children with sickle cell anemia in 2009 were used to calculate the costs of hospital care.
      • Total hospital costs during 2 years were estimated to be $1.8 million for the children who received HU treatment and $2.5 million for the children who did not receive this type of treatment. The use of HU treatment in this study could have saved Medicaid $700,000.
      • Per-child annual hospital costs were $9,450 for those who received HU treatment versus $13,716 for those who did not; 31% lower for the HU group.
    • HU treatment requires additional follow-up doctor visits and lab tests to look for side effects of HU. The added cost of HU medication and outpatient care was relatively low compared with the reduction in hospital costs.
      • The cost of HU medication for 2 years was $1,368.
      • Total annual outpatient cost for 2 years was $1,622 for the children who received HU treatment and $246 for those who did not receive this type of treatment.
    • Total costs were reduced by 21% for children who received HU treatment compared to those who did not receive this type of treatment.
      • Total annual medical cost was $11,072 ($9,450 + 1,622) for children on HU and $13,962 ($13,716 + 246) for those who did not receive this type of treatment.
  • Medicaid programs and other public health insurers can expect reductions in healthcare costs as more young children with sickle cell anemia receive HU.
    • 74% of children enrolled in BABY HUG were enrolled in Medicaid or state health plans.

What is Sickle Cell Disease (SCD)?

A group of inherited red blood cell disorders in which the red blood cells become hard and sticky and look like a C-shaped farm tool called a "sickle." The sickle cells die early, which causes a constant shortage of red blood cells. Also, when they travel through small blood vessels, they get stuck and clog the blood flow. This causes pain and other serious problems such as infection, acute chest syndrome and stroke.

About this Study

Researchers compiled information from the BABY HUG study and health insurance claims data to determine the total healthcare costs associated with HU treatment in young children with sickle cell anemia.

CDC Activities

The Centers for Disease Control and Prevention (CDC), National Center on Birth Defects and Developmental Disabilities (NCBDDD), Division of Blood Disorders (DBD) considers SCD a major public health concern and is committed to monitoring the numbers of people affected by this condition, raising awareness, and promoting health education. Highlighted below are select activities that showcase CDC’s work on this important topic:

  • Analyzing information that was collected from seven states that participated in the Registry and Surveillance for Hemoglobinopathies (RuSH) project. This two year project identified unique ways to gather the information needed to learn more about people with SCD from multiple data sources.
  • Carrying out the Public Health Research Epidemiology and Surveillance for Hemoglobinopathies (PHRESH) project in California, Georgia and Mississippi, which will strengthen and improve upon the information gathering strategies developed in RuSH to help better understand SCD and the healthcare needs of those with SCD.
  • Funding three medical centers and a state public health department to determine the safety and risk of health problems for those who receive frequent blood transfusions, including people with SCD.
  • Learning what information is needed to increase the public’s and healthcare providers’ awareness and knowledge about SCD and how best to share the information.
  • Hosting an ongoing monthly public health webinar series to raise awareness among healthcare professionals, public health staff, patients, and others.

What is Sickle Cell Disease (SCD)?

A group of inherited red blood cell disorders in which the red blood cells become hard and sticky and look like a C-shaped farm tool called a "sickle." The sickle cells die early, which causes a constant shortage of red blood cells. Also, when they travel through small blood vessels, they get stuck and clog the blood flow. This causes pain and other serious problems such as infection, acute chest syndrome and stroke.

More Information

To learn more about sickle cell disease, please visit https://www.cdc.gov/ncbddd/sicklecell/.

To learn more about the RuSH and PHRESH projects, please visit https://www.cdc.gov/ncbddd/hemoglobinopathies/index.html

Paper Reference

Wang WC, Oyeku SO, Luo Z, Boulet SL, Miller ST, Fish B, Thompson BW, Grosse SD. Hydroxyurea is associated with lower costs of care of young children with sickle cell anemia. Pediatrics. 2013 [epub September 2, 2013]

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