Patient Reported Outcomes Among Pediatric Patients with Nonalcoholic Fatty Liver Disease: TARGET-NASH

Pediatric NAFLD patients experience disease-specific psychological burdens—including fatigue, anxiety, and depression—but whether these patient-reported outcomes differed meaningfully by disease severity in a real-world pediatric cohort had not been studied.

The TARGET-NASH pediatric cohort was analyzed using validated PROMIS instruments to compare self-reported fatigue, anxiety, and depression T-scores across NAFL and NASH/cirrhosis subgroups, with stratification by ALT level.

Characterizing that children with more severe NAFLD report higher fatigue and depression scores provides the evidence base for including patient-reported outcomes as endpoints in pediatric NAFLD trials and supports regulators' expectation that therapies demonstrate benefit across clinically meaningful patient experience domains.

Pediatric NAFLD patient-reported outcome characterization for PRO endpoint development, Disease severity-PRO linkage in pediatric liver disease for regulatory strategy, Patient experience benchmarking in pediatric MASLD for drug development and advocacy