An Online Decision Aid to Help Patients and Caregivers Decide on Treatments for Sickle Cell Disease

This project has results

Abstracts

Public Abstract

Download this abstract: English (pdf) | Español (pdf) | Audio Recording (mp3)

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
  • Decision aid for patients considering treatments for SCD
  • Usual care
Outcomes Knowledge, decisional conflict, decisional preparedness, decisional regret
Timeframe 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.

Journal Articles

Related Articles

Journal of the National Medical Association

A Qualitative Study of Chronic Pain and Self-Management in Adults with Sickle Cell Disease

PLoS One

From trust to skepticism: An in-depth analysis across age groups of adults with sickle cell disease on their perspectives regarding hydroxyurea

Biology of Blood and Marrow Transplantation

Experiences and Decision-Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients' and Caregivers' Perspectives

PLoS One

Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease

More on this Project  

This project's final research report is expected to be available by June 2019.

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.

Project Details

Principal Investigator
Lakshmanan Krishnamurti, MD
Project Status
Completed; PCORI Public and Professional Abstracts Posted
Project Title
Comparative Effectiveness of a Decision Aid for Therapeutic Options in Sickle Cell Disease
Board Approval Date
May 2013
Project End Date
September 2018
Organization
Emory University^
Year Awarded
2013
State
Georgia
Year Completed
2018
Project Type
Research Project
Health Conditions 
Genetic Disorders
Genetic Disorders - Other
Blood Disorders
Sickle Cell Disease
Rare Diseases
Rare Diseases - Other
Intervention Strategies : 
Drug Interventions
Other Health Services Interventions
Technology Interventions
Training and Education Interventions
Populations : 
Racial/Ethnic Minorities
Low Health Literacy/Numeracy
Children -- 18 and under
Individuals with Rare Disease
Funding Announcement
Assessment of Prevention, Diagnosis, and Treatment Options
Project Budget
$2,143,228
Study Registration Information
HSRP20143291
NCT02326597

^Lakshmanan Krishnamurti, MD, was affiliated with the University of Pittsburgh when this project was initially funded.

Page Last Updated: 
July 22, 2019