Chapter Four
Designing The Project
Question, Communicate, Certify, and Share
Centered on a patient-focused research query, we engage stakeholders at every project stage, ensuring clear study procedures and interventions aligned with patient needs. We prioritize meaningful outcomes for patients, families, and stakeholders and plan to disseminate study insights to engage participants and other involved parties.
Project Design
Research Question
Communication
Meaningful Outcomes
What is Patient-Centered Outcomes Research?
How to Select Meaningful Outcomes
in plans for Starzl patient-centered outcomes research and in making treatment decisions with families?
- Focus on the goals of the patient and family – Work to align transplant team goals with family goals.
- Acknowledge and value the perspective and priorities of all stakeholders.
- Patients and caregivers – and their support system
- All healthcare providers – including allied health, mental health, social work, child life
- Consider the whole patient – not just the liver transplant.
- Explain your opinion – and actively listen to the opinions of others.
- Review the available evidence for treatment decisions – and available experience of participants.
- Be open to sharing evidence and experience.
- Both are valuable, but be transparent about which is which.
- Plan time for discussion and consensus-building.
- Be open to compromise and change.
How does the study design keep the work patient-centered?
Research that is truly centered on the child and family needs to…
- Make participation easy and convenient: virtual visits, avoid extra appointments, local labs, user-friendly platform, work around parents’ schedules
- Address the priorities of patients and families
- Recognize the contributions of participants: compensation, acknowledgement/appreciation, sharing of results
- Involve meaningful communication, with trusted team members, about all aspects of the study:
- What is the goal of doing this study? How will it help us and other families
- What are the short and long-term risks to my child?
Chapter Four
4A: Research Question
Focus on a patient-centered research question
Patient-centered outcomes research addresses vital questions important to patients and caregivers, focusing on expectations based on personal factors, exploring options and their impacts, improving outcomes, and optimizing decisions in healthcare for children within care systems.
Questions That Matter to Patients and Caregivers
Patient-centered outcomes research aims to ask – and answer – questions that matter to patients and caregivers
- “Given personal characteristics, conditions, and preferences, what should I expect will happen to me?”
- “What are my options, and what are the potential benefits and harms of those options?”
- “How can clinicians and the care-delivery systems they work in help us make the best decisions about my child’s health and healthcare?”
- “What can I do to improve the outcomes that are most important to me?”
#1 Reason That Parents/Children Decide to Enroll in a Research Study
Make the PCOR Process Workable
- Focus it on patient and family priorities. Be clear about how the project will improve the health and lives of patients and families.
- Continue iterative input
- Create a team/community around the work
- Make it multi-disciplinary – Include social work, nursing, coordinators and the multidisciplinary team as a whole
- Minimize the burden
- Limited time commitment, Non arduous data collection
- Appropriate protected time for participation in research – for all team members
- Making it accessible–easy to use, not time consuming, offering incentives (whether it be monetary or contributing to overall larger goals related to our practice
- Focus on reducing burden on patients (e.g., minimal visits/asks, reimbursement for travel).
- Embedded into normal workflow, be as least disruptive as possible
- Keep technology easy-to-use, across time and devices
- Develop a pediatric specific PCOR training to focus on pediatric specific components of research design.
Chapter Four
4B: Communication
Effectively communicate with stakeholders
Communicate efficiently with stakeholders to engage and empower all team members throughout the project, implement patient- and family-centric recruitment and consent plans, and delineate study procedures for patient comprehension. Design intervention and monitoring strategies aligned with patient and family needs.
Empower Everyone to Speak Up and Share
- Make time and space to listen. Schedule time, and sit down. Have the right people in the room.
- Recognize that each transplant journey is different – consider alternative ideas and special situations.
- Create opportunities for shared decision-making.
- End every conversation with an opportunity to share concerns or ask questions – and information about how to do this later if needed.
- Be respectful of everyone’s time. Stay on schedule.
- Elicit opinions and concerns, using open-ended questions. Listen actively to answers.
Considering a New Treatment for Kids
- Create a comfortable space for the child and family – together and individually.
- Check-in on mood and comfort – Acknowledge feelings and worries.
- Make space for questions.
- Include them in decision-making – make sure that they have the information they need to help make decisions.
- Remind them that they are the experts on their own experiences. Ask for their preferences.
- Acknowledge the child as an individual.
How Does The Study Design Keep The Work Patient-Centered?
Research that is truly centered on the child and family needs to…
- Make participation easy and convenient: virtual visits, avoid extra appointments, local labs, user-friendly platform, work around parents’ schedules
- Address the priorities of patients and families
- Recognize the contributions of participants: compensation, acknowledgement/appreciation, sharing of results
- Involve meaningful communication, with trusted team members, about all aspects of the study:
- What is the goal of doing this study? How will it help us and other families
- What are the short and long-term risks to my child?
How Can Providers Empower Patient Partners?
Chapter Four
4C: Meaningful Outcomes
How to selecting meaningful outcomes
Study OUTCOMES are what actually happens to a person during and after a research study. Outcomes can look at both the benefits and harms of a treatment. Comparing the outcomes of people that receive one treatment versus the other helps us understand which treatment works better – and for which people.
We want to focus on patient-centered outcomes. These are outcomes that are:
- Important to a patient’s survival, function, or feelings
- As identified or affirmed by patients themselves OR
- Judged to be in the patient’s best interest by providers and caregivers.
Hopes and Dreams of Children Living with Liver Transplants
- To be healthy
- To minimize medication burden
- To live a “normal” life
- To not be limited by health or the transplant
- To pursue happiness
- To be resilient, mentally and physically
- To gain independence
What MakesYou Most Proud of Your Transplant Recipient?
- Resilience – bouncing back from anything that comes his way
- How brave and resilient he is – using his experience to remind himself that “I can do this – I have been through worse”
- Handling all the medical things like such a champion
- Meeting milestones:
- Dancing in her ballerina costume –proud of reading in school
- Full of life and embracing all that there is to offer in middle school
- Transition to young adulthood – taken over her own meds
- Now 23 and getting her master’s degree in data science
- Very comfortable with advocating for what he wants and does not want
- She is active now – she had bad ascites, and now she can do anything – she’s just rambunctious.
- She’s thriving – “just a normal kid”
Milestones
- Growth – achieving age-appropriate height and weight
- Gaining skills – intellectual, physical, social
- Having the same energy as peers
- Participating in activities that make them happy
- Attending school
- Making friends and participating in social activities
- Playing sports without limitations
- Graduating from school
- Travelling, for school or other actvities
- Living at college or on their own, outside of parents’ home
- Health literacy
- Taking ownership of their medications, health and healthcare
- Ability to advocate for themselves
- Parent: Long-term, to be able to advocate for herself and her ongoing medical needs
- Provider: I would hope my patients have the best health literacy possible so they are excellent self-advocates
- Supporting and advocating for others – either in the transplant community (e.g. support groups, transplant camp), in the family, at school or in other settings
Detailed Information for Researchers
When considering a new treatment for kids with liver transplants, what do we need to know?
- What is the purpose of this treatment? How will it be helpful?
- Why does my child need this treatment?
- What is the goal of this treatment?
- Will this be an ongoing problem that my child will have throughout their life?
- How likely is this treatment to work? What are the chances it won’t work?
- How will we monitor my child’s response to the treatment?
- How often will they need to have labs or other monitoring?
- What are the risks of this treatment? What are the possible negative outcomes?
- What side effects do I need to look out for? Are there “expected” or very common side effects?
- How does this treatment impact other medications or medical conditions that my child has?
- What are the potential impacts of this treatment on my child’s other organs? On their overall health and functioning?
- What are the long-term side effects? What are the risks for my child in the future of receiving this treatment now?
- What will the burden of this treatment be on my child and my family?
- How long is the course of treatment?
- How many times a day is the medicine taken? Is it compounded? How is it stored?
- What is the recovery time from the procedure or treatment? (How long will my child have to wait before eating? How long will we have to be in the hospital?)
- How will this treatment (or the monitoring after it) impact our daily lives at home?
- Will insurance cover this treatment?
- Who will be involved in giving the treatment – and in monitoring the effects of the treatment?
- Is this treatment the only option? Why are we choosing this one?
- What are the next steps after this treatment? What happens if this treatment does not work?
Best & Worst Aspects of Life with a Liver Transplant
