Results Summary
What was the research about?
Sickle cell disease, or SCD, is a genetic disorder that can make it hard for blood to flow through the body. SCD affects both adults and children, and its symptoms include pain and fatigue. In the United States, most people with SCD are African American. SCD is also common in people who are Hispanic. Although there is no cure for SCD, treatments can relieve symptoms. Choosing the right treatment can be hard because the treatments have different benefits and harms.
In this study, the research team made an online decision aid to help people choose among three SCD treatments for themselves or their children. Decision aids help people choose between healthcare options based on what’s most important to them. The decision aid had text, audio recordings, and video of patients, caregivers, and experts talking about living with SCD. It told patients about benefits and harms of SCD treatments. The team wanted to learn if using the decision aid made it easier for people to choose a treatment.
What were the results?
Of the 120 patients and caregivers, 76 did not complete the study. As a result, the research team can’t be sure if the decision aid was helpful.
For those who stayed in the study
- Six months after using the decision aid, patients and caregivers felt more ready to make treatment choices compared with those who didn’t use the decision aid.
- Three months after using the decision aid, patients and caregivers felt more confident in deciding on a treatment compared with those who didn’t use it. But there was no difference between the two groups after six months.
Using the decision aid didn’t change
- How well patients and caregivers knew the benefits and harms of treatments
- Which treatment a patient or caregiver would pick
- How much patients or caregivers regretted their treatment choice
In interviews with the research team, patients and caregivers said that the decision aid was both useful and easy to use.
Who was in the study?
The study included 120 adults with SCD or who took care of a child with SCD. Of these patients and caregivers, 75 percent were African American, and 8 percent were Hispanic. The average age was 34, 76 percent were women, and 80 percent had some college education.
What did the research team do?
The research team assigned adults by chance to have or not have access to the online decision aid. Everyone in the study received care from their doctors as usual.
The research team asked everyone to fill out online surveys at the start of the study and again three and six months later. In addition, the team interviewed patients and caregivers. The team asked if the decision aid was useful and if it helped
- Teach patients and caregivers about SCD
- Patients and caregivers clarify what was important to them about treatment for SCD
- Prepare patients and caregivers to make a treatment choice
Patients, caregivers, doctors, community advocates, and policy makers with expertise in SCD helped create and test the decision aid.
What were the limits of the study?
Only 44 of the 120 people who started the study stayed in it until the end. This wasn’t enough people for the research team to be sure if the decision aid worked.
Future research could find out why patients and caregivers may or may not want to complete this type of study. In addition, future studies could include people with different backgrounds.
How can people use the results?
This decision aid will require further testing before it can be widely used. But patients with SCD and caregivers of children with SCD may find it useful to help them learn about available treatments.
Professional Abstract
Objective
To evaluate the effectiveness of a web-based decision aid to help patients and caregivers choose among treatment options for sickle cell disease (SCD)
Study Design
| Design Elements | Description |
|---|---|
| Design | Randomized controlled trial |
| Population | 120 adult patients with SCD and caregivers of children with SCD |
| Interventions/ Comparators |
|
| Outcomes | Knowledge, decisional conflict, decisional preparedness, decisional regret |
| 6-month follow-up for study outcomes |
This randomized controlled trial sought to compare the effectiveness of a web-based decision aid for patients and caregivers versus usual care for making treatment decisions for SCD. Decision aids help people make informed choices among healthcare options that are consistent with their values. Researchers developed and tested a web-based decision aid that
- Informed users about harms and benefits of three treatments for SCD—hydroxyurea, chronic blood transfusions, and bone marrow transplantation
- Included a web-based library of written materials, audio, and video clips featuring patients, caregivers, and experts who discussed living with SCD
- Helped users decide on a treatment based on their individual values and preferences
Researchers randomized adults, who either had SCD or cared for a child with SCD, to use the decision aid in addition to usual care from their current healthcare providers or to receive usual care alone.
The study included 120 patients and caregivers. Of these, 75% were African American and 8% were Hispanic. The average age was 34, 76% were female, and 80% had at least some college education.
At baseline and three and six months later, researchers administered online surveys to patients and caregivers to assess outcomes. In addition, researchers used qualitative interviews to evaluate acceptability and usability of the decision aid and its usefulness in educating patients and caregivers about SCD, clarifying values and preferences related to treatment, and preparing for decision making.
Patients, caregivers, healthcare providers, community advocates, and policy makers helped develop and test the patient decision aid.
Results
Of the 120 participants, 76 did not complete the study. Among those who completed follow-up, researchers observed greater improvements in preparedness for decision making from baseline to six months in the intervention group compared with the usual care group (p=0.0009). From baseline to three months, the intervention group had greater improvements in decisional self-efficacy compared with the usual care group (p=0.05) but not at six months. Researchers found no significant differences between the two groups in decisional conflict, treatment knowledge, or choice predisposition.
Limitations
Because of study attrition, the results were not conclusive.
Conclusions and Relevance
Although it was not possible to draw conclusions about the impact of the decision aid, interviews suggest that participants found the decision aid to be acceptable and useful.
Future Research Needs
Future research could identify and address concerns among patients with SCD and their caregivers that may affect their willingness to complete these types of studies. Future studies could include diverse participants to ensure broad applicability of results.
Final Research Report
View this project's final research report.
More About This Research
Peer-Review Summary
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented, and the researchers made changes or provided responses. Those comments and responses included the following:
- Reviewers noted the high rate of nonresponse on the follow-up surveys. Reviewers recommended that the researchers reduce the emphasis on quantitative findings. The researchers revised their report to point out that they could not draw conclusions based on the follow-up surveys because of the amount of missing outcome data.
- Reviewers lauded the qualitative work done as part of this study. Given the problems with missing outcome data, they encouraged the researchers to focus on the qualitative results. The researchers reorganized the report to provide more emphasis on the qualitative methods and results.
- Reviewers expressed concern about some inconsistencies in what the researchers considered as primary outcomes of the randomized control trial. In some places, the researchers seemed to indicate that acceptability of the intervention was a major outcome of the trial. However, the reviewers pointed out that acceptability of the intervention could not be a trial outcome since it could only be measured in the intervention group. The researchers revised their report to clarify that acceptability was a major outcome for Aim 1 on the development of the intervention.
Conflict of Interest Disclosures
View the COI disclosure form.
Journal Citations
Results of This Project
Related Journal Citations
Peer-Review Summary
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented, and the researchers made changes or provided responses. Those comments and responses included the following:
- Reviewers noted the high rate of nonresponse on the follow-up surveys. Reviewers recommended that the researchers reduce the emphasis on quantitative findings. The researchers revised their report to point out that they could not draw conclusions based on the follow-up surveys because of the amount of missing outcome data.
- Reviewers lauded the qualitative work done as part of this study. Given the problems with missing outcome data, they encouraged the researchers to focus on the qualitative results. The researchers reorganized the report to provide more emphasis on the qualitative methods and results.
- Reviewers expressed concern about some inconsistencies in what the researchers considered as primary outcomes of the randomized control trial. In some places, the researchers seemed to indicate that acceptability of the intervention was a major outcome of the trial. However, the reviewers pointed out that acceptability of the intervention could not be a trial outcome since it could only be measured in the intervention group. The researchers revised their report to clarify that acceptability was a major outcome for Aim 1 on the development of the intervention.
Conflict of Interest Disclosures
Project Information
Key Dates
Study Registration Information
^Lakshmanan Krishnamurti, MD, was affiliated with the University of Pittsburgh when this project was initially funded.