PGI26 - A Review of Patient-Reported Outcomes in Nonalcoholic Steatohepatitis (NASH)

Abstract

Nonalcoholic steatohepatitis (NASH) is characterized by fat build up in the liver and results in inflammation and liver cell damage. NASH is rapidly becoming one of the top causes of cirrhosis, hepatocellular carcinoma and indication for liver transplantation. Patient-reported outcome (PRO) measures are an important component to assessing disease impact and therapy response in patients with NASH. The objective of this research is to identify commonly used PROs in NASH, evaluate their development, and assess their utility in various types of studies. A literature review using PubMed and a search on clinicaltrials.gov was conducted to identify PROs used in NASH studies. These PROs were evaluated against the FDA’s PRO Guidance criteria with regard to: (1) content validity, particularly patient input in concept elicitation, item generation, and cognitive debriefing; and (2) psychometric testing, particularly construct validity, internal consistency reliability, test-retest reliability, and responsiveness. Each criteria was assessed and given a rating. Finally, an attempt was made to make recommendations on which PROs should be used in different types of studies. Only one NASH-specific PRO publication currently exists: the NASH-CHECK. The NASH-CHECK has only gone through qualitative validation and needs further psychometric analyses. Other PROs used in NASH are generic liver-related measures such as the Chronic Liver Disease Questionnaire, the EORTC QLQ-HCC18 (EORTC Quality of Life Questionnaire - Hepatocellular Carcinoma / Primary Liver Cancer Module), and the LDQOL | Liver Disease Quality of Life Questionnaire. Generic VAS scales have also been used to measure condition- specific symptoms such as itching. Other PRO measures have been appropriately validated but not in specifically in the NASH population. Most PROs used in NASH are borrowed from other diseases or are general PROs and do not address the specific symptoms or concerns of NASH patients. As such, there is need for further NASH-specific PRO development.