Nonalcoholic steatohepatitis (NASH) with liver fibrosis is an increasingly important
cause of liver-related morbidity and mortality. A diagnosis of NASH can only be made
using liver biopsy. Liver histology also forms the endpoint for the expedited licensing
strategies that have been approved by regulators to allow patients with NASH access
to treatment before the impact of these on clinical outcomes is known. Validation
of these histological surrogate endpoints is critical for the ongoing development
of new therapies for NASH. The use of liver biopsy to define both trial entry and
endpoints raises questions about the use of treatments for NASH in practice when the
effectiveness of treatment will likely depend, at least in part, on the use of histology
for patient selection in the real world.
Keywords
NASH - NAFLD - surrogate - validity
