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Project Summary Project Information Key Dates
Dissemination Project (Limited Competition D&I PFA)
In progress; Recruitment not applicable

Supporting Families' Treatment Decisions with Evidence on Treatment of Urea Cycle Disorders

Project Summary

PCORI implementation projects promote the use of findings from PCORI-funded studies in real-world healthcare and other settings. These projects build toward broad use of evidence to inform healthcare decisions.

This PCORI-funded dissemination project is providing new evidence on the risks and benefits of treatment options for urea cycle disorders, or UCDs, to support families’ treatment decisions.
UCDs are rare inherited disorders in which the body can’t get rid of ammonia, a substance made when the body breaks down protein in food. Ammonia builds up in the bloodstream, which can lead to brain damage, disability, or death. Parents and their clinicians consider many factors when making decisions about treatment for their child’s UCD.

What is the goal of this dissemination project?

UCDs can be treated with medical management. This treatment involves medicine and a diet that helps the body get rid of ammonia. UCDs can also be treated with a liver transplant, which prevents the buildup of ammonia but comes with surgery risks. A PCORI-funded research study found no difference in survival, brain function, and quality of life between patients who had a liver transplant and those on medical management.

For patients who had a liver transplant, the study saw a trend toward higher family quality of life and better mental ability. This trend was strongest among patients who had transplants sooner.

This project is helping to make families of children with UCDs and their clinicians aware of these research findings to support their decisions about UCD treatment.

What will this project do?

The project team is creating two sets of videos, one for families and one for clinicians. The videos present evidence from the PCORI-funded study and related research. The team will then work with project advisors, patient organizations, and doctors’ professional organizations to reach families of patients with UCDs and their care teams.

During the project, the videos can be viewed on a mobile app. The mobile app allows viewers to provide feedback and ask questions. Based on the questions received, the project team is creating follow-up videos that address patient and family concerns. After the project, all videos will be posted publicly. Families and care teams will be able to access the videos without using an app.

What is the expected impact of this project?

Because UCDs are very rare, the project team can reach most families of, and care teams working with, patients with UCDs in the United States. The team expects to reach about 40 percent of patients with UCDs who qualify for a liver transplant and at least one person on each patient’s care team. In all, 50—100 UCD clinicians will receive the resources from this project.

The evaluation will confirm that the project is working as intended to bring these new findings to families of patients with UCDs and their clinicians to support their decisions. The evaluation will assess increases in knowledge, awareness, and motivation to use the evidence in decision making.

More about this dissemination project:

Stakeholders Involved in This Project

  • Urea Cycle Disorders Consortium
  • National Urea Cycle Disorders Foundation
  • National Organization for Rare Disorders
  • Society of Pediatric Liver Transplantation
  • Society for Inherited Metabolic Disorders
  • American College of Medical Genetics and Genomics
  • American Academy of Pediatrics (Washington, DC Chapter)
  • An advisory committee that includes medical professionals who care for patients with UCD and members of patient advocacy organizations

Dissemination Strategies

  • Refining messages for families and clinicians that capture and summarize evidence from the PCORI-funded study and other relevant research
  • Incorporating messages into educational videos that can be viewed on a mobile app
  • Working with key stakeholder organizations to distribute links to the app and videos among their membership
  • Developing a menu of different dissemination channels (e.g., email, newsletters, social media) that organizations can select from to reach their membership
  • Partnering with organizations and vendors that have successfully developed materials and tools that support dissemination and reaching UCD patients
  • Making the videos available to the public for free after the project is over

Evaluation Outcomes

To document dissemination:

  • Number of families and clinicians who view the videos
  • Success of various dissemination channels in reaching the target audiences

To assess healthcare and health outcomes:

  • Awareness of evidence on UCD treatment
  • Knowledge of evidence on UCD treatment
  • Motivation to apply the evidence in UCD treatment decision making

Project Information

Nicholas Ah Mew, MD
Children's National Health System
$300,000
Current Evidence on Liver Transplant versus Medical Management in Urea Cycle Disorders: Opening a Dialogue with Patients, Caregivers, and Providers

Key Dates

February 2023
2021

Initial PCORI-Funded Research Study

This dissemination project focuses on putting findings into practice from this completed PCORI-funded research study: Comparing Treatment Options for Urea Cycle Disorders

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Last updated: October 18, 2023