OBJECTIVES Hepatoblastoma is the most common liver tumor in children. More than 90% of cases occur before five years of age. Children born at very-low birthweight (VLBW), less than 1500 grams, are at 17.2 times higher odds of developing hepatoblastoma than children of normal birthweight. We aimed to analyze the cost-effectiveness of using an alpha-fetoprotein (AFP) assay screening regimen to improve early detection and treatment of hepatoblastoma between 3-48 months of age for VLBW children in the U.S.

METHODS From a U.S. societal perspective, we developed a decision tree to calculate discounted lifetime costs and monetary benefits in 2018 USD based on estimates in the published literature. These costs and quality-adjusted life years (QALYs) were used to calculate an incremental cost-effectiveness ratio (ICER) for the screening protocol compared to standard care at a willingness-to-pay threshold of $150,000/QALY. Future costs and benefits were discounted at a rate of 3% per year. Both univariate and multivariate probabilistic sensitivity analyses were performed to assess the effects of uncertainty in model parameters.

RESULTS : Over a lifetime horizon, a screening protocol for VLBW children increased the expected QALYs by 0.0011 per child at an additional cost of $141 per child compared to the standard of care. The expected ICER was $133,551/QALY. Results from both univariate and probabilistic sensitivity analyses did not substantively change this conclusion. For children diagnosed with hepatoblastoma, our model finds that the screening regimen is associated with 11.2% increase in survival, 3.31 increase in expected QALYs, and $286,097 decrease in expected cost.

CONCLUSIONS Screening with AFP is cost-effective for VLBW children, who are at an elevated risk for developing hepatoblastoma. An AFP screening regimen has the potential to identify cases of hepatoblastoma at an earlier stage, reducing treatment costs and improving morbidity and mortality outcomes.