Accounting for the Placebo Effect and Optimizing Outcomes in Clinical Trials of Nonalcoholic Steatohepatitis (NASH)
- Resource Type
- Review Paper
- Authors
- Noureddin, Nabil; Han, Ma Ai Thanda; Alkhouri, Naim; Noureddin, Mazen
- Source
- Current Hepatology Reports. 19(1):63-69
- Subject
- Nonalcoholic fatty liver disease
Nonalcoholic steatohepatitis
Nonalcoholic fatty liver disease activity score
Placebo effect
Magnetic resonance imaging–estimated proton density fat fraction
Magnetic resonance spectroscopy
- Language
- English
- ISSN
- 2195-9595
Purpose of Review: Understand the placebo effect and optimize the design of nonalcoholic fatty liver disease (NAFLD) clinical trials.Recent Findings: The placebo effect on histologic outcomes and liver fat on MRI-based imaging has been assessed in a systematic review and meta-analysis, and three phase 3 studies have been reported. The placebo effect on improvement in the main histologic outcome was present in ~ 25% of the patients. The placebo effect on components of the NAFLD activity index in trials using the magnetic resonance imaging proton density fat fraction and magnetic resonance spectroscopy as outcomes also was reported. In this review, we discuss factors that led to placebo effects in the reported studies and suggest measures to ameliorate the effects in future NASH trials.Summary: The significant placebo response in NASH clinical trials is evidence that investigators should consider this effect in estimating sample size and designing future clinical trials.