Pro: 'The usefulness of biomarkers in glomerular diseases'. The problem: moving from syndrome to mechanism--individual patient variability in disease presentation, course and response to therapy

Abstract

The diagnosis and treatment decisions in glomerular disease are principally based on renal pathology and nonspecific clinical laboratory measurements such as serum creatinine and urine protein. Using these classification approaches, patients have marked variability in rate of progression and response to therapy, exposing a significant number of patients to toxicity without benefit. Additionally, clinical trials are at risk of not being able to detect an efficacious therapy in relevant subgroups as patients with shared clinical-pathologic diagnoses have heterogeneous underlying pathobiology. To change this treatment paradigm, biomarkers that reflect the molecular mechanisms underlying the clinical-pathologic diagnoses are needed. Recent progress to identify such biomarkers has been aided by advances in molecular profiling, large-scale data generation and multi-scalar data integration, including prospectively collected clinical data. This article reviews the evolving success stories in glomerular disease biomarkers across the genotype-phenotype continuum and highlights opportunities to transition to precision medicine in glomerular disease.

Keywords: molecular taxonomy; nephrotic syndrome; precision medicine; translational medicine.

FIGURE 1:

Towards precision medicine of renal disease. Data capturing all aspects of the disease are combined with biomedical research to create a knowledge network, available to the research community to define disease in functional terms (Reprinted from Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease, with permission from the National Academy of Sciences).

FIGURE 2:

Integration of multiple layers of data across the genotype-phenotype continuum to identify glomerular disease biomarkers.