PCORI funds dissemination projects to promote the understanding and awareness of findings from PCORI-funded studies. This project focuses on disseminating findings from the completed PCORI-funded research project: Comparing Treatment Options for Urea Cycle Disorders

1. What were the results from the original PCORI-funded research study?

The research team did not find statistically significant differences in survival, mental ability, or quality of life between patients with urea cycle disorders (UCDs) who had liver transplants and those managed with diet and medications. However, the team saw a trend toward higher quality of life in those who had liver transplant, and saw a consistent trend in better mental ability, especially in patients who had liver transplant sooner.

In interviews, the research team learned that families and doctors did not just consider the severity of the UCD, but also considered many other personal, social, and medical factors when deciding on treatment. The importance of these factors changed over time as children grew older and their needs changed. Most families who chose liver transplant did so because they reached a tipping point where they felt that they could no longer manage their child’s UCD with diet and medications.

2. Why is this research finding important?

The research questions studied in the original PCORI-funded project were posed by the families of patients with UCD. They want to understand what treatments would lead to better outcomes for their loved ones, because there is very little published about this topic. Our comparison of outcomes in UCD patients who had liver transplant or medical management included more patients than previous studies. Also, unlike prior studies, our study also accounted for many other factors that could influence survival, mental ability, or quality of life, so that we could get the fairest comparison of medical management versus liver transplant. Even though we could not prove with certainty that survival is worse but mental ability and quality of life is better with liver transplant, we think it is important for families to understand the trends observed. We also think families will want to know this information, because there is so little known about this topic.

3. What is the goal of this project?

The aims of the project are:

• Aim 1: To empower patients and families in their decision making about UCD treatment by applying new and existing evidence on the risks and benefits of medical management versus liver transplant.
• Aim 2: To support patient-provider treatment counseling interactions in pediatric primary and sub-specialty metabolic and transplant care settings through sharing new and existing evidence on UCD patient priorities as well as the relative risks and benefits of medical management versus liver transplant in UCD.

4. What is the project team doing?

The team will make an educational video for patients and families, and another video for healthcare providers, to explain the results of the study and the results of other relevant research. These videos will be viewed on an app that will be developed for this project, which will be available for download to mobile phone, tablet or computer. The app will also have a Frequently Asked Questions section that will include an explanation about why we are doing this project as well as links to other helpful resources.

After watching the video, viewers will also be asked to submit any follow-up questions they might have. The project team will review the questions and will release 2 follow-up Q&A videos that will answer these questions. These follow-up videos will also be available through the app.

We believe our videos will help patients, families, and doctors better understand what is currently known about outcomes in UCD with liver transplant and with diet and medications. Members of the project team and their advisors will share the link to download the app to as many patients, families, metabolic specialists, liver transplant doctors, and pediatricians as they can. Because UCDs are very rare, and because we will reach out to families and the healthcare workers taking care of UCD patients, we expect to reach at least one person involved in the care plan of every UCD patient for whom liver transplant is being considered.

5. How is the team evaluating this project?

The research team will keep track of the number of people that watch these videos through the app to evaluate our success in reaching the patients, families, metabolic specialists, liver transplant doctors, and pediatricians who may benefit most from this new knowledge. Before people can watch the videos, they will be asked to answer a few questions about their knowledge of medical treatments and liver transplant in UCD and how they intend to use this knowledge when making treatment decisions for UCD. They will be asked similar questions after watching the video so that we can understand the impact our educational video had on viewers.

6. How is the team involving patients and others in making sure the findings reach people who can use them?

The project team includes members of the Urea Cycle Disorders Consortium (a team of healthcare workers that focuses on research and treatment of UCD) and the National Urea Cycle Disorders foundation (the national advocacy group for patients with UCD). Additionally, our project will have an advisory committee made up of members of different organizations of medical professionals that care for UCD patients, research consortia, and patient advocacy organizations. Each of these advisors has provided a letter of support that states that they will provide feedback at different stages of our work and will promote and share a link to download the app to patients with UCD and the healthcare providers who take care of patients with UCD. They might share this link with others by email, newsletters, social media, or other online posts. They may also tell people about this project on any phone conferences or add information about our project on their websites.

7. How will this project help ensure future uptake and use of findings from patient-centered outcomes research?

Once we have collected enough data about viewers’ response to our videos, we will publicly post all videos and other materials openly online so that new viewers can see the videos without having to download an app. After this project is over, the project team and its advisors will still promote the videos. We may also continue to work together to share results of other projects that patients and families are interested in and continue making Q&A videos. The app we make may also be used to share other results with patients, families, and healthcare workers.