In Robin sequence (RS), infants are born with a small lower jaw, causing their tongues to be pushed back. This causes them to struggle with breathing and eating because their tongue blocks their airway. They also frequently have cleft palates, causing additional feeding problems. These breathing and feeding problems have a profound impact on the child and caregiver. Despite this, RS parents have not been engaged in patient-centered outcomes research (PCOR). Additionally, RS researchers lack knowledge of PCOR and comparative effectiveness research (CER) methods and skills. The lack of stakeholder engagement among RS families and absence of PCOR and CER knowledge among RS researchers are major barriers to improving outcomes for children with this rare disease.
The goal of this project is to build an RS community better able to participate in PCOR and CER. We propose to establish the Stakeholder Alliance for Children with Robin Sequence (StARS) composed of RS parents, health systems personnel, clinicians, and researchers. We will build on our established consortium and engage key stakeholders in RS treatment and research.
Stakeholders will be engaged at a local level and then through a series of educational and engagement webinars and annual meetings. These activities will provide education regarding PCOR and work to identify outcomes desired by RS families, PCOR priorities, and barriers to engaging in PCOR.
The network we propose to build will include patient stakeholders trained in PCOR and CER and prepared to be full research partners and medical stakeholders trained in PCOR and armed with the skills to communicate with nonscientist stakeholders. This network will be well positioned to undertake future PCOR projects in the field of RS treatment. Our long-term goal is to design and implement studies and trials that answer RS parent-/caregiver-derived questions to achieve better patient outcomes.
Stakeholders will include RS parents/caregivers, health systems personnel, clinicians, and researchers. The complexity of RS children’s care requires multiple clinician types (each with unique perspectives) to be engaged and included in the StARS network.
Meaningful participation is central to our engagement plan. RS parents and other stakeholders will be involved at each step of the project, starting with establishment of the StARS network; deciding the mission and governance; refining the communication plan; and identifying patient-centered outcomes, PCOR priorities, and barriers. Stakeholders will identify a CER question to investigate. Engagement will occur at least monthly throughout the project.
StARS collaborators include clinicians, researchers, and patient caregivers at eight project sites Children’s Hospital of Atlanta, Children’s Hospital Colorado, Floating Hospital for Children in Boston, Children’s Mercy Hospital (Kansas), Iowa Children’s Hospital, Primary Children’s Hospital (Utah), Seattle Children’s Hospital, and UC Davis Children’s Hospital. In addition to these local sites, we are collaborating with the Pierre Robin Sequence Foundation advocacy group, as well as several social media RS advocacy groups.