This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.
What is the research about?
Each year, about 1 in 2,500 babies in the United States are born with sickle cell disease, or SCD. SCD is a genetic health problem that can make it hard for blood to flow through the body. It can cause pain, organ damage, and early death. A medicine called hydroxyurea can improve SCD. Patients who take this medicine have less pain, fewer complications, and live longer. But this medicine can have side effects, such as nausea, headache, and hair loss. It may also pose long-term risks, such as cancer or birth defects. Many parents find these side effects and risks concerning and don’t start their child on hydroxyurea. Those who do often question whether they made the right decision.
National guidelines recommend that doctors use shared decision making to discuss SCD treatment with parents. Shared decision making is a process in which patients work with their doctors to select tests and treatments. But many doctors don’t know the best ways to talk about SCD treatment options with parents.
In this study, the research team is comparing two ways to help doctors and parents of children with SCD discuss treatment options.
Who can this research help?
Doctors can use results from this study when considering how to help parents decide on treatment for children with SCD.
What is the research team doing?
The research team is enrolling 260 parents of children with SCD at nine hematology clinics across the United States. The team is assigning sites by chance to one of two ways to help doctors discuss treatment options with parents of children with SCD.
At one group of sites, parents receive a set of four decision aids that help doctors and parents talk about SCD treatment options. Decision aids help people choose between two or more healthcare options based on what is most important to them. The research team is training doctors on how to use them when talking with parents. The decision aids include
- A brochure for parents to review before their appointment
- A treatment card for parents and doctors to use during their discussion about treatment
- An after-visit booklet for parents to take home
- Videos that show parents talking about how they decided about SCD treatment for their child
At the other sites, doctors offer care as usual. They also use the American Society of Hematology pocket guide as a reference when discussing treatment options with parents of children with SCD.
At the study start and again four to six months later, the research team is asking parents how involved they were in the treatment decision and how conflicted they felt about the decision they made. In addition, the team is looking at electronic medical records to see how many doctors offer patients hydroxyurea. The team is comparing results between sites that got the pocket guide and those that got the decision aids.
Parents of children with SCD, doctors, nurses, community organizers, health insurers, social workers, and adults with SCD are helping design and plan the study. They are also helping monitor study progress and risks.
Research methods at a glance
Shared Decision Making
Training and Education Interventions
Low Health Literacy/Numeracy
Children -- 18 and under
Individuals with Rare Disease