1 Betty and Guy Beatty Center for Integrated Research, Inova Health System, Falls Church, VA.
2 Department of Medicine, Center for Liver Diseases, Inova Fairfax Hospital, Falls Church, VA.
3 Center for Outcomes Research in Liver Diseases, Washington, DC.
BACKGROUND & AIMS:
Currently, standard of care (SOC) treatment for NASH is limited to lifestyle modifications. Drug regimens are being evaluated currently. We assessed the impact of a short term hypothetical treatment on clinical outcomes of NASH.
Markov models estimated differences in outcomes between SOC and 2 hypothetical NASH treatments (A and B). We modeled 10,000 50-year old biopsy-proven NASH patients over lifetime horizon. Health states included NASH with fibrosis (F1-F3), cirrhosis, hepatocellular carcinoma, liver transplant and mortality. Fibrosis Regression Factor (FRF) variable modeled the probability of 1-3 stage fibrosis improvement with treatment. Annual probability of treatment (ATP) ranged from 10-70%. Treatment success was defined as regression to fibrosis, while failure was defined as progression to stages beyond cirrhosis. In treatment-A, successful treatment was followed by a maintenance regimen which stopped disease progression. After a successful treatment-B, patients remained at risk of disease progression. Differences in outcomes were calculated between both treatments and SOC models. We conducted a probabilistic sensitivity analysis.
At 10% to 70% ATP, treatment-A averts 353 to 782 liver transplants and 1,277 to 2,381 liver-related deaths relative to SOC. Treatment-B averts 129 to 437 liver transplants and 386 to 1,043 liver related deaths. Sensitivity analysis shows our model is robust in estimating liver related mortality and LTs averted, but is sensitive when estimating QALYs gained.
With a small annual probability of treatment and FRF=1, a 2-year treatment followed by maintenance of histologic improvement for patients would be highly beneficial relative to short-term treatment alone.