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

Challenge

Pediatric NAFLD was undercharacterized in prospective real-world settings, with most available data from tertiary referral centers that lacked the diversity and community site representation needed to understand the full spectrum of pediatric disease in usual clinical practice.

Solution

The TARGET-NASH pediatric cohort enrolled children and adolescents at both academic and community pediatric sites, with centrally abstracted medical record data characterizing demographics, disease severity, comorbidities, and medication use in this understudied population.

Impact

Providing the first large, multi-site prospective characterization of pediatric NAFLD in real-world US practice establishes the evidence base for pediatric trial design, informs enrollment criteria for pediatric NAFLD programs, and demonstrates that TARGET-NASH's infrastructure is capable of supporting pediatric drug development.