Project Summary
Background: Pediatric renovascular hypertension (pRVH), high blood pressure resulting from renal artery and aortic narrowing, risks significant morbidity and mortality. Timely detection and treatment are critical to prevent kidney injury, cardiopulmonary complications, and stroke. Low disease frequency limits existing data. The optimal management approach for these children remains ill-defined, and there remains significant variation in practice considering indications for and timing of revascularization/repair. The expansion of novel surgical and endovascular (catheter-based) techniques has dramatically changed the landscape of patient care. As multidisciplinary centers of excellence are few, patients and families are often challenged to balance vastly differing sets of recommendations. The decision-making process in this complex landscape is lacking the voice of the patient and family. Larger-scale research that prioritizes outcomes important to patients and their families is urgently needed to advance understanding of this disease, and define best practices in the care of these affected children.
Proposed Solution to the Problem: The University of Michigan’s pRVH Center features a multidisciplinary team of providers, nurses, and social workers alongside a growing patient and family support group. In 2019, the inaugural International pRVH Symposium was hosted (www.umichpedirvhsymposium.org). The program encompassed best practices in patient care and recent discoveries. A post-symposium survey revealed 95 percent supported expanding collaborative research. The PCORI Capacity Building Engagement Award will develop infrastructure and partnership through a formalized pRVH PCOR Collaborative that will facilitate PCOR training, support, and networking among patients with pRVH and their families and critical stakeholders. This critical foundation will fuel large-scale research, as prioritized and directed by a comprehensive group, that will decrease the number of years of life lost for these young patients, optimize sustained quality of life, and identify lessons perhaps applicable to other rare disorders impacting children.
Objectives: The main outcome of this work is to assemble a broad collaborative of patients with and families affected by pRVH and stakeholders that are well informed, who will participate in identifying pRVH-related knowledge gaps that would benefit from PCOR and CER, and will guide the research prioritization.
SPECIFIC AIM 1: Create a pRVH PCOR Collaborative that includes an advisory board, a critical stakeholder group, and a broader virtual research network (VRN).
SPECIFIC AIM 2: Prioritize PCOR and comparative effectiveness research (CER) research priorities that consider critical diagnostic and treatment decisions).
SPECIFIC AIM 3: Conduct a pRVH PCOR Conference in 2022 that will disseminate the collaborative’s interim work, share PCOR programming, and bring together stakeholders to formalize prioritized research plans.
Activities:
- Advisory and critical stakeholder groups will be convened, expanding on the 2019 inaugural pRVH Symposium planning committee, invited guests, and attendees. The project team will create a pRVH PCOR Collaborative website with content to empower stakeholders to be full participants in their partnership.
- A patient and family VRN will be created. Survey and focus groups will capture VRN perceptions and experience around the diagnosis and management of pRVH. A literature review from the 2019 pRVH Symposium, coupled with survey and focus group data, will guide the creation of conceptual models and fact sheets for CER question development, which stakeholders will review and ultimately prioritize.
- A 2022 pRVH PCOR Conference will augment a scientific session with PCOR programming that describes successful patient-researcher collaborations and important skills, knowledge, and experience for both researchers and patients participating in PCOR. Stakeholders will finalize a work plan for sustainable and prioritized research conduct.
Projected Outcomes and Outputs: This project will assemble a large cohort of patients with pRVH and affected families that are well informed about pRVH and how to participate in PCOR and CER trials. The VRN will facilitate future patient activation for pragmatic clinical trials, which are very much needed for this rare disease process.
The team anticipates the following outputs for multimodal dissemination from the pRVH PCOR Collaborative:
- The results from the initial survey that assesses patient/caregiver interest and knowledge gaps from the patient and family perspective
- The results from the literature review and the CER fact sheets
- The results of the survey of the collaborative’s VRN vote on research question prioritization
Patient and Stakeholder Engagement Plan: The pRVH PCOR Collaborative will actively solicit and evaluate ways to make stakeholder engagement more effective throughout the project. Many patient families of affected children already serve to support others. The team will engage these parent “champions” to start, while also leveraging and strengthening existing relationships within pRVH Centers of Excellence and patient advocacy organizations. Stakeholder partners will:
- Prioritize research questions with survey tool design, fact sheet design and review, and regular virtual meetings/workshops
- Disseminate study findings efficiently
- Help with implementation of results, all while incorporating patient perspectives throughout this research continuum
Project Collaborators: Magical Mila Foundation, Williams Syndrome Association, Neurofibromatosis Network.