Background and Aims: Nonalcoholic steatohepatitis (NASH) is a
progressive form of fatty liver disease characterized by excessive liver
fat accumulation, inflammation, cell injury and fibrosis. The aim was
to explore the impact of NASH from the patients’ perspective and to
determine the availability of suitable patient-reported outcome
(PRO) scales for NASH adults.
Methods: Searches conducted using PubMed (from 2006 to 2015)
and grey literature identified key articles and patient-reported
narratives occurring in social media/patient support forums. Areas
of NASH-impact identified by the process were summarised and
reviewed by medical experts and patient support group representatives
(US/UK).
Results: Published studies on patient-perceived NASH impact are
scarce. Early stage NASH is often considered asymptomatic but
chronic fatigue, malaise and right upper-quadrant abdominal
discomfort related to hepatomegaly are reported; symptom impact
increases with progressing fibrosis. Psychiatric assessments reveal
significantly increased major depression disorder (MDD) and generalised
anxiety disorder (GAD) for confirmed NASH patients
compared to matched controls (MDD: p = 0.018; GAD p = 0.005).
Confirmed NASH patients also have worse health-related quality of
life (HRQOL) compared to fatty liver disease patients with no NASH
on the Short Form–36 (SF-36) (physical health component scores:
44.5 NASH, 47.1 no-NASH p = 0.018; vitality scores: 44.4 NASH, 46.6
no-NASH, p = 0.043; bodily pain: 47.7 NASH, 50.0 no-NASH, p = 0.043;
general health: 41.8 NASH, 44.2 no-NASH, p = 0.023); however, the
SF-36 is a generic instrument not validated for NASH and is unlikely
to address all issues relevant to NASH patients. No NASH-specific PRO
instrument was identified by the current search. The social media
review identified patient narratives presented on support group
websites which reinforce the observation that NASH profoundly
impacts many aspects of patients’ lives, including; ability to conduct
day-to-day activities, personal and social relationships, and HRQOL.
Clinical experts and patient representatives agreed that the review
findings matched their views of the patient experience but noted that
further qualitative work was needed with NASH patients.
Conclusions: While NASH clearly impacts patients’ HRQOL, no PRO
instrument has been developed based on patient insights. Clinical
experts and patient representatives confirmed the need to develop a
NASH-specific PRO measure based on patient and expert insights and
FDA guidance.
