Kidney Week

Abstract: SA-PO393

Conceptualizing the Future of Personalized Precision Medicine for Glomerular Disease

Session Information

• Glomerular Diseases: Clinical, Outcomes, Trials - III
  October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

• 1203 Glomerular Diseases: Clinical, Outcomes, and Trials

Authors

• Gipson, Debbie S., University of Michigan Mott Children's Hospital, Ann Arbor, Michigan, United States

Debbie S. Gipson,

• Trachtman, Howard, NYU Langone Health, New York, New York, United States

Howard Trachtman,

• Barisoni, L., U. Miami, Miller School of Medicine, Miami Beach, Florida, United States

L. Barisoni,

• Hodgin, Jeffrey B., The University of Michigan, Ann Arbor, Michigan, United States

Jeffrey B. Hodgin,

• Hamidi, Habib, University of Michigan, Ann Arbor, Michigan, United States

Habib Hamidi,

• Eddy, Sean, University of Michigan, Ann Arbor, Michigan, United States

Sean Eddy,

• Mariani, Laura H., University of Michigan, Ann Arbor, Michigan, United States

Laura H. Mariani,

• Sampson, Matt G., University of Michigan, Ann Arbor, Michigan, United States

Matt G. Sampson,

• Holzman, Lawrence B., University of Pennsylvania, Philadelphia, Pennsylvania, United States

Lawrence B. Holzman,

• Troost, Jonathan P., University of Michigan, Ann Arbor, Michigan, United States

Jonathan P. Troost,

• Sedor, John R., Cleveland Clinic, Cleveland, Ohio, United States

John R. Sedor,

• Kretzler, Matthias, U.Michigan, Ann Arbor, Michigan, United States

Matthias Kretzler,

Group or Team Name

• Nephrotic Syndrome Study Network

Background

Glomerular disease therapy selection and outcomes have been hampered by imprecise disease characterization. Scientific advances have been generated in research contexts but few have crossed the research - patient care divide.

Methods

We hypothesized that comprehensive clinical, demographic and molecular phenotypes could be integrated into a personalized, comprehensive disease profile that could be used to understand the disease within nephrotic syndrome subjects. Physician- scientists and project managers from NEPTUNE were invited to participate in the model development. A multicenter, interdisciplinary NEPTUNE Kidney Review Board (KRB) model was created. Testing of this model used archived cases from the NEPTUNE study cohort. Return of results to caregivers or subjects was prohibited during this model testing phase.

Results

Data domains including, clinical phenotype, pathology, genetics, renal gene expression, and proteomics, were evaluated for each case. KRB participants were educated about data domain content, analytic methods, results display and supporting evidence from published literature. Multicenter web-enabled conferences were used to a) present case-specific information, b) review subject-specific data for each domain and c) integrate the multi-scalar data to develop theoretical management and treatment strategies for the subject. The multicenter, NEPTUNE KRB pilot was launched in February 2018. This pilot phase allowed the team to practice baseline evaluation of each case using a systems biology approach in a blinded manner, assemble the information into a case specific knowledge environment. Following discussion, the outcome of the case based on longitudinal observation was disclosed.

Conclusion

Integration of clinical, pathologic and molecular data will enable personalized disease profiling and will be essential in matching the right therapies for the right patients. In order to accomplish this, a prepared community of clinicians and scientists are needed to effectively interpret the strengths and limitations of the evidence. Furthermore, addition of patient advisors and ethicists will strengthen this initiative. KRBs provide a rational and effective strategy to bring precision health to the glomerular disease community.

Funding

• NIDDK Support