Nonalcoholic Fatty Liver Disease in the U.S.: Clinical Characteristics of Patients Enrolled in TARGET-NASH.

Challenge

Drug development for NAFLD required a comprehensive real-world baseline characterization of enrolled patients across the disease spectrum—from simple steatosis to cirrhosis—at academic and community sites, to establish a reference population and validate the cohort's representativeness for drug development purposes.

Solution

The first consecutive patients enrolled in TARGET-NASH were characterized using standardized case definitions across NAFL, NASH, and NAFLD cirrhosis in both adults and pediatric patients, providing a comprehensive cross-sectional description of the cohort's baseline demographics, disease severity, and comorbidities.

Impact

Establishing the TARGET-NASH baseline population profile validated the registry's representativeness for drug development and demonstrated that it included patient populations—particularly those with cirrhosis and cardiovascular disease—typically excluded from clinical trials, supporting the registry's utility for naturalistic comparator data.