Project Summary

WAGR syndrome is a rare genetic condition that affects multiple body systems. There are fewer than 500 known cases of this disorder. “WAGR” is an acronym that stands for Wilms tumor, Aniridia, Genitourinary abnormalities, and Range of developmental delays. A wide variety of other conditions are also associated with the syndrome. While some of these conditions are being studied, there is a lack of knowledge and research related to Wilms tumor in WAGR syndrome. Wilms tumor is a rare pediatric kidney cancer. This tumor occurs in about 50 percent of patients with WAGR syndrome.

Concerns of parents of children and adults with WAGR syndrome include lack of knowledge on which to base Wilms tumor treatment decisions and lack of patient-centered research design. Researchers believe that significant progress can be made in clinical studies, but they lack avenues for parent stakeholder input. These stakeholders need to meet to address current knowledge and practices, to identify and resolve gaps between experiences and concerns of parents of children with WAGR syndrome and assumptions and priorities of Wilms tumor researchers.

The International WAGR Syndrome Association (IWSA) will hold a conference at the University of Michigan, Ann Arbor. This conference will engage parents and researchers in planning clinical studies; developing a patient-centered model for this research; developing consensus on research questions; and creating educational and awareness materials for use by all stakeholders.

The conference committee will invite 30 key stakeholders. The conference will begin with PCOR and CER training given by a PCORI Ambassador and former PCORI reviewer. Parents of patients with WAGR syndrome will give presentations about their experiences with Wilms tumor and share questions and concerns regarding research and clinical studies. Clinicians and WAGR syndrome-related foundations will make similar presentations. Researchers will discuss current knowledge about Wilms tumor in WAGR syndrome and explore unanswered questions. Breakout sessions with stakeholder groups and roundtable discussions will enable the groups to make progress toward the conference’s goals. A videographer will document activities and individual interviews for educational materials.

Projected outcomes and long-term objectives include: engagement of all stakeholders in the creation of patient-centered research projects with patient-reported outcomes; demonstration of the value of including parents in clinical study planning; creation of stakeholder communities trained in and committed to PCOR; and fostering partnerships to inspire stakeholder collaboration.

The IWSA is a patient advocacy organization with 225 member families, engaged via a variety of support and information-sharing activities. IWSA parents will serve as key personnel on the conference committee, as well as attend, present, and participate in the conference, including creation and dissemination of postconference materials. The stakeholder community also includes foundations promoting awareness of and stimulating research on WAGR syndrome; rare disease organizations; Wilms tumor clinical specialists and researchers; pediatric cancer patients, researchers, and clinicians; and organizations sharing pediatric cancer information.

Project collaborators include the IWSA, WAGR Warriors, Miranda’s Mission, CS Mott Children’s Hospital (Michigan), Children’s National Medical Center (Washington, DC), University of Michigan, and A Twist of Fate-ATS.

Project Information

Kelly Trout BSN, RN
International WAGR Syndrome Association

Key Dates

December 2018


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Last updated: April 12, 2024