Background: For thousands of children with cirrhosis, liver failure, liver cancer, and genetic-based metabolic diseases, liver transplantation has transformed fatal conditions to treatable ones. The main gaps that now remain are integrating patient and family input and optimizing these children’s long post-transplant outcomes. At every point in this journey toward, through, and for a lifetime after transplant, myriad decisions must be made about medical care—always aiming to balance the risks and benefits that accompany transplant to achieve the best possible long-term outcomes. Ideally, families, children, and transplant providers are lifelong partners in these care decisions, working together from a patient’s earliest transplant experiences through adulthood.
Proposed Solution: PARTNER seeks to build on the established bonds between providers and patient partners, using the infrastructure and partnerships of the Starzl Network for Excellence in Pediatric Transplantation (SNEPT) and Transplant Families (TF) to expand from these personal relationships into organizational partnerships with shared priorities, knowledge, and a sustainable capacity to do collaborative patient-centered outcomes research (PCOR).
Objectives: The project’s long-term goal is sustainable, equitable health and wellness for pediatric transplant patients and their families. PARTNER aims to expand engagement and build sustainable expertise and supportive infrastructure for PCOR and comparative effectiveness (CER) research into SNEPT. Thus, PARTNER seeks to:
- Engage a diverse group of pediatric transplant families, providers, and other stakeholders as PCOR partners. The project team wants to foster productive, sustainable relationships and a shared understanding of patient- and community-engaged partnerships, key patient-centered outcomes (PCOs), and research priorities.
- Develop (a) knowledge and skills in SNEPT/TF participants and (b) infrastructure in SNEPT/TF to support collaborative development and conduct of future PCOR and CER.
- Co-create a PCOR roadmap, including projects to address priority areas, key patient-centered/reported outcomes, and infrastructure for PCOR (shared leadership, recruitment, data collection, study design).
Activities: The project will use a cyclical design to progressively develop PCOR skills within SNEPT/TF, while continuously eliciting input from the transplant community. The PCORI-funded ASPIRE (Accelerating Systematic Stakeholder, Patient, and Institution Research Engagement) group will mentor PARTNER during the design/lead activities and evaluate their engagement, adoption, and implementation. Each cycle has:
- Education Module: Adapted from ASPIRE and PCORI’s Research Fundamentals trainings and delivered as a 10- to 20-minute interactive lesson through digital health platform (Real Time Clinic, RTC).
- Survey (on RTC): To elicit feedback on how content should be applied in SNEPT/TF PCOR.
- Survey Analysis: Survey data will be summarized and reviewed and used to shape the focus group discussion guide the next cycle.
- Focus Group: For each module/survey, stakeholders will be asked to participate in a focus group for more in-depth discussion on the topic. Discussion among focus groups will be used to refine existing categories, identify sub-themes within categories, and map relationships between themes.
- Iterative Feedback: For each cycle, all pertinent findings will be summarized and disseminated to the broader participants for review and additional comment.Survey and focus group feedback will be used to draft and refine successive sections of the PARTNER PCOR Roadmap—a document that will outline principles and processes for PCOR within SNEPT. The PCOR Roadmap will include guidelines for shared leadership, productive partnerships, stakeholder roles and expectations, project design, and proposed study outcomes. It will also include initial project prioritization and research question development.
Projected Outcomes and Outputs: PARTNER will prepare the pediatric liver transplant community to extend SNEPT’s reach to PCOR/CER and to prioritize the patient-centered outcomes and interventions most meaningful and impactful for children who have received transplants and their families. The project aims to build engagement, sustainable expertise, and supportive infrastructure for PCOR/CER into SNEPT, with learning that can be disseminated to all TF’s partners. The goal of these efforts will be to ingrain critical skills, identify shared priorities, and develop feasible, sustainable plans for authentic PCOR in SNEPT.
Patient and Stakeholder Engagement Plan: Key stakeholders, including patients and family advocates, will be embedded and lead participation in every facet of PARTNER, including planning, design, implementation, interpretation, and dissemination. To maximize reach, the project will continue to use digital tools and virtual focus groups.
Project Collaborators: The Starzl Network for Excellence in Pediatric Transplantation (SNEPT), Transplant Families (TF), Accelerating Systematic Stakeholder, Patient, and Institution Research Engagement (ASPIRE), Real Time Clinic (RTC), Special Populations Health Equity Research and Education (SPHERE) Committee.